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Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01559129
Recruitment Status : Terminated (Enrollment was stopped early (see limitations and caveats section).)
First Posted : March 21, 2012
Results First Posted : October 18, 2019
Last Update Posted : October 18, 2019
Sponsor:
Information provided by (Responsible Party):
Celgene

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Scleroderma, Systemic
Sclerosis, Systemic
Systemic Scleroderma
Systemic Sclerosis
Interstitial Lung Disease
Interventions Drug: Pomalidomide (CC-4047)
Drug: Placebo
Enrollment 23
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase.
Period Title: Treatment Phase
Started 12 11
Received Treatment 12 10
Completed 7 4
Not Completed 5 7
Reason Not Completed
Adverse Event             0             2
Progressive Disease             1             1
Lack of Efficacy             0             1
Study Terminated by Sponsor             3             2
Protocol Exclusion Criteria             1             0
Did Not Receive Study Drug             0             1
Period Title: Open-label Extension Period
Started 6 [1] 2 [2]
Completed 0 0
Not Completed 6 2
Reason Not Completed
Adverse Event             1             0
Study Terminated by Sponsor             5             2
[1]
One participant elected not to enter the Extension Phase.
[2]
Two participants elected not to enter the Extension Phase.
Arm/Group Title Placebo Pomalidomide Total
Hide Arm/Group Description Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase. Total of all reporting groups
Overall Number of Baseline Participants 12 11 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants 11 participants 23 participants
44.8  (13.77) 48.9  (9.84) 46.7  (11.97)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 11 participants 23 participants
< 65 years
10
  83.3%
10
  90.9%
20
  87.0%
≥ 65 years
2
  16.7%
1
   9.1%
3
  13.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 11 participants 23 participants
Female
10
  83.3%
10
  90.9%
20
  87.0%
Male
2
  16.7%
1
   9.1%
3
  13.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 11 participants 23 participants
Asian
0
   0.0%
2
  18.2%
2
   8.7%
Black or African American
0
   0.0%
1
   9.1%
1
   4.3%
Native Hawaiian or Other Pacific Islanders
1
   8.3%
0
   0.0%
1
   4.3%
White
10
  83.3%
8
  72.7%
18
  78.3%
Other
1
   8.3%
0
   0.0%
1
   4.3%
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 12 participants 11 participants 23 participants
30.64  (5.392) 27.61  (9.4) 29.19  (7.556)
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Hide Description An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event.
Time Frame From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized participants who received at least one dose of the study treatment (either pomalidomide or placebo).
Arm/Group Title Treatment Phase: Placebo Treatment Phase: Pomalidomide Extension Phase: Placebo/Pomalidomide Extension Phase: Pomalidomide/Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.
In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10 6 2
Measure Type: Number
Unit of Measure: participants
Any TEAE 12 9 5 2
Drug-related TEAE 8 6 3 1
Severe TEAE 1 4 0 0
Serious TEAE 1 4 0 1
Serious Drug-related TEAE 0 1 0 0
TEAE Leading to Drug Interruption 2 1 0 0
TEAE Leading to Drug Withdrawal 0 4 0 0
TEAE Leading to Death 0 0 0 0
2.Primary Outcome
Title Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Hide Description Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points.
Time Frame Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) consisted of all randomized participants who received at least one dose of study drug. Participants with a Baseline value and a Week 52 were included in the analysis.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 11 8
Mean (Standard Deviation)
Unit of Measure: percent predicted
-2.8  (4.02) -5.2  (5.29)
3.Primary Outcome
Title Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination
Hide Description Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate]), or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
Time Frame Baseline and Week 52 (or the Treatment Phase Early Termination visit)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and a post-baseline value at Week 52 or Early Termination
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 11 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
-3.7  (6.99) -2.7  (5.66)
4.Primary Outcome
Title Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
Time Frame Baseline and Week 52 (or Treatment Phase Early Termination visit)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and a Week 52 value
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.00  (0.18) 0.1  (0.29)
5.Secondary Outcome
Title Change From Baseline in Percent Predicted Forced Vital Capacity Over Time
Hide Description Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value).
Time Frame Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at the time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: percent predicted
Week 12 Number Analyzed 10 participants 8 participants
-2.4  (3.07) -1.8  (3.39)
Week 24 Number Analyzed 10 participants 8 participants
-1.3  (3.33) 2.3  (12.58)
Week 36 Number Analyzed 9 participants 6 participants
-2.6  (2.48) -4.4  (3.97)
Week 64 Number Analyzed 6 participants 2 participants
-7.0  (4.29) -6.4  (4.49)
Week 76 Number Analyzed 2 participants 1 participants
-8.7  (10.34) -5.2 [1]   (NA)
Week 156 Number Analyzed 4 participants 2 participants
-6.7  (6.19) -5.9  (2.31)
[1]
Could not be calculated for one participant
6.Secondary Outcome
Title Change From Baseline in Modified Rodnan Skin Score Over Time
Hide Description Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate], or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
Time Frame Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and a post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 11 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
-1.8  (3.94) -0.3  (1.83)
Week 24 Number Analyzed 10 participants 8 participants
-3.6  (5.68) -2.0  (3.38)
Week 64 Number Analyzed 6 participants 2 participants
-4.3  (8.36) -4.0  (2.83)
Week 76 Number Analyzed 2 participants 1 participants
-8.5  (7.78) -7.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
-3.7  (8.52) -4.5  (3.54)
[1]
NA = Could not be calculated for one participant
7.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
Time Frame Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.2  (0.36) -0.1  (0.30)
Week 24 Number Analyzed 10 participants 8 participants
0.1  (0.23) -0.1  (0.20)
Week 64 Number Analyzed 6 participants 2 participants
0.0  (0.20) 0.4  (0.01)
Week 76 Number Analyzed 2 participants 1 participants
-0.1  (0.01) 0.5 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
0.0  (0.14) 0.0  (0.14)
[1]
NA = Could not be calculated for one participant
8.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.1  (0.36) -0.1  (0.51)
Week 24 Number Analyzed 10 participants 8 participants
-0.1  (0.27) 0.0  (0.47)
Week 52/Early Termination Number Analyzed 12 participants 10 participants
-0.1  (0.54) 0.1  (0.38)
Week 64 Number Analyzed 6 participants 2 participants
-0.2  (0.14) 0.2  (0.46)
Week 76 Number Analyzed 2 participants 1 participants
-0.5  (0.18) -0.2 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
0.0  (0.44) 0.1  (0.28)
[1]
NA = Could not be calculated for one participant
9.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.4  (0.56) 0.2  (1.21)
Week 24 Number Analyzed 10 participants 8 participants
0.0  (0.53) 0.3  (0.80)
Week 52/Early Termination Number Analyzed 12 participants 10 participants
0.0  (0.55) 0.2  (1.07)
Week 64 Number Analyzed 6 participants 2 participants
-0.2  (0.70) 1.0  (1.41)
Week 76 Number Analyzed 2 participants 1 participants
0.0  (0.00) 3.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
-0.3  (0.30) 0.9  (1.24)
[1]
NA = Could not be calculated for one participant
10.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.0  (0.00) 0.0  (0.00)
Week 24 Number Analyzed 10 participants 8 participants
0.2  (0.42) 0.0  (0.00)
Week 52/Early Termination Number Analyzed 12 participants 9 participants
0.0  (0.00) 0.1  (0.33)
Week 64 Number Analyzed 6 participants 2 participants
0.2  (0.41) 0.0  (0.00)
Week 76 Number Analyzed 2 participants 1 participants
0.0  (0.00) 0.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
0.2  (0.41) 0.0  (0.00)
[1]
NA = Could not be calculated for one participant
11.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 [none] to 3 [5-7 days/week] and one question about the presence of watery stools (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.4  (0.57) -0.2  (0.65)
Week 24 Number Analyzed 10 participants 8 participants
0.1  (0.21) -0.3  (0.46)
Week 52/Early Termination Number Analyzed 12 participants 10 participants
0.3  (0.34) 0.0  (0.75)
Week 64 Number Analyzed 6 participants 2 participants
0.1  (0.38) 0.5  (0.71)
Week 76 Number Analyzed 2 participants 1 participants
0.0  (0.00) -0.5 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
0.3  (0.42) -0.8  (0.35)
[1]
NA = Could not be calculated for one participant
12.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 11 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 9 participants 8 participants
0.3  (0.49) 0.0  (0.28)
Week 24 Number Analyzed 9 participants 8 participants
0.1  (0.30) -0.1  (0.20)
Week 52/Early termination Number Analyzed 11 participants 10 participants
0.0  (0.25) 0.2  (0.45)
Week 64 Number Analyzed 5 participants 2 participants
0.0  (0.18) 0.3  (0.35)
Week 76 Number Analyzed 1 participants 1 participants
0.2 [1]   (NA) 0.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 5 participants 2 participants
0.0  (0.25) -0.2  (0.23)
[1]
NA = Could not be calculated for one participant
13.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
0.2  (0.40) -0.4  (0.80)
Week 24 Number Analyzed 10 participants 8 participants
0.1  (0.16) -0.3  (0.56)
Week 52/Early Termination Number Analyzed 12 participants 10 participants
0.1  (0.25) -0.1  (0.42)
Week 64 Number Analyzed 6 participants 2 participants
0.0  (0.36) 0.2  (0.00)
Week 76 Number Analyzed 2 participants 1 participants
0.0  (0.00) 0.3 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
-0.1  (0.15) 0.0  (0.00)
[1]
NA = Could not be calculated for one participant
14.Secondary Outcome
Title Change From Baseline in UCLA SCTC GIT 2.0 Constipation Subscale Score Over Time
Hide Description The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 [no days] to 3 [5-7 days/week] and one question about the presence of stools becoming harder (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a Baseline value and post-baseline value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 12 Number Analyzed 10 participants 8 participants
-0.1  (0.36) 0.2  (0.98)
Week 24 Number Analyzed 10 participants 8 participants
-0.2  (0.31) 0.3  (0.81)
Week 52/Early Termination Number Analyzed 12 participants 10 participants
0.0  (0.25) 0.3  (0.51)
Week 64 Number Analyzed 6 participants 2 participants
0.0  (0.60) 0.1  (0.18)
Week 76 Number Analyzed 2 participants 1 participants
0.0  (0.00) 0.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
-0.2  (0.20) 0.1  (0.18)
[1]
NA = Could not be calculated for one participant
15.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 12
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Count of Participants
Unit of Measure: Participants
Major deterioration
0
   0.0%
0
   0.0%
Moderate deterioration
1
  10.0%
1
  14.3%
Minor deterioration
0
   0.0%
1
  14.3%
No change
7
  70.0%
3
  42.9%
Minor improvement
2
  20.0%
1
  14.3%
Moderate improvement
0
   0.0%
0
   0.0%
Major improvement
0
   0.0%
0
   0.0%
Further impairment for other reasons
0
   0.0%
1
  14.3%
16.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 24
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Number
Unit of Measure: Participants
Major deterioration 1 0
Moderate deterioration 0 2
Minor deterioration 1 1
No change 4 2
Minor improvement 3 1
Moderate improvement 1 0
Major improvement 0 0
Further impairment for other reasons 0 1
17.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 52/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 52 or at the Treatment Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 4
Minor deterioration 1 1
No change 8 4
Minor improvement 3 1
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
18.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 64
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 1
Moderate deterioration 0 1
Minor deterioration 1 0
No change 4 0
Minor improvement 1 0
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
19.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 76
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 76
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 2 1
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 1
Minor deterioration 1 0
No change 0 0
Minor improvement 1 0
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
20.Secondary Outcome
Title Change From Baseline in Dyspnea Functional Impairment at Week 156/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
Time Frame Week 156 or the Extension Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 1
Minor deterioration 2 1
No change 2 0
Minor improvement 1 0
Moderate improvement 1 0
Major improvement 0 0
Further impairment for other reasons 0 0
21.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 12
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 0
Minor deterioration 2 1
No change 5 5
Minor improvement 3 1
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
22.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 24
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 1 1
Minor deterioration 2 1
No change 5 2
Minor improvement 2 3
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
23.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 52/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 52 or at the Treatment Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 3
Minor deterioration 1 1
No change 8 6
Minor improvement 2 0
Moderate improvement 0 0
Major improvement 1 0
Further impairment for other reasons 0 0
24.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 64
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 1
Moderate deterioration 0 0
Minor deterioration 2 0
No change 3 0
Minor improvement 1 1
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
25.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 76
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 76
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 2 1
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 0
Minor deterioration 1 0
No change 1 0
Minor improvement 0 1
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
26.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Task at Week 156/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
Time Frame Week 156 or the Extension Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 1 0
Minor deterioration 1 1
No change 2 0
Minor improvement 2 1
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
27.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 12
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 1 0
Minor deterioration 2 2
No change 4 4
Minor improvement 2 1
Moderate improvement 1 0
Major improvement 0 0
Further impairment for other reasons 0 0
28.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 24
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 10 7
Measure Type: Number
Unit of Measure: Participants
Major deterioration 1 1
Moderate deterioration 0 1
Minor deterioration 1 0
No change 4 3
Minor improvement 3 2
Moderate improvement 1 0
Major improvement 0 0
Further impairment for other reasons 0 0
29.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 52/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 52 or at the Treatment Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 10
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 3
Minor deterioration 1 1
No change 7 6
Minor improvement 3 0
Moderate improvement 1 0
Major improvement 0 0
Further impairment for other reasons 0 0
30.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 64
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 1
Moderate deterioration 0 0
Minor deterioration 3 0
No change 2 1
Minor improvement 1 0
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
31.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 76
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 76
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 2 1
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 0 0
Minor deterioration 1 0
No change 0 1
Minor improvement 1 0
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
32.Secondary Outcome
Title Change From Baseline in Dyspnea Magnitude of Effort at Week 156/Early Termination
Hide Description The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
Time Frame Week 156 or at the Extension Phase Early Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with available data
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 6 2
Measure Type: Number
Unit of Measure: Participants
Major deterioration 0 0
Moderate deterioration 1 0
Minor deterioration 2 1
No change 2 1
Minor improvement 1 0
Moderate improvement 0 0
Major improvement 0 0
Further impairment for other reasons 0 0
33.Secondary Outcome
Title Oxygen Saturation Over Time
Hide Description Oxygen saturation was measured by pulse oximetry.
Time Frame Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).
Hide Outcome Measure Data
Hide Analysis Population Description
FAS participants with a value at each time point.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 12 9
Mean (Standard Deviation)
Unit of Measure: percent saturation
Baseline Number Analyzed 12 participants 9 participants
97.5  (2.07) 96.8  (1.92)
Week 12 Number Analyzed 10 participants 8 participants
97.1  (2.18) 97.4  (1.51)
Week 24 Number Analyzed 10 participants 8 participants
97.6  (1.65) 96.5  (4.31)
Week 52/Early Termination Number Analyzed 12 participants 9 participants
96.8  (2.18) 96.8  (1.86)
Week 64 Number Analyzed 6 participants 2 participants
96.3  (2.94) 94.0  (2.83)
Week 76 Number Analyzed 2 participants 1 participants
98.5  (0.71) 97.0 [1]   (NA)
Week 156/Early Termination Number Analyzed 6 participants 2 participants
95.8  (5.12) 97.5  (0.71)
[1]
NA = Could not be calculated for one participant
34.Secondary Outcome
Title Pharmacokinetic Parameters of Pomalidomide in Plasma
Hide Description Pharmacokinetic (PK) analyses were not conducted as there were too few participants with available data.
Time Frame Day 1 and week 4 pre-dose and up to 24 hours post-dose.
Hide Outcome Measure Data
Hide Analysis Population Description
PK analyses were not conducted as there were too few participants with available data. No data was analyzed.
Arm/Group Title Placebo Pomalidomide
Hide Arm/Group Description:
Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.
Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and Pomalidomide.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment Phase: Placebo Treatment Phase: Pomalidomide Extension Phase: Placebo/Pomalidomide Extension Phase: Pomalidomide/Pomalidomide
Hide Arm/Group Description Participants received placebo orally once a day for 52 weeks during the treatment phase. Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years. Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
All-Cause Mortality
Treatment Phase: Placebo Treatment Phase: Pomalidomide Extension Phase: Placebo/Pomalidomide Extension Phase: Pomalidomide/Pomalidomide
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
Treatment Phase: Placebo Treatment Phase: Pomalidomide Extension Phase: Placebo/Pomalidomide Extension Phase: Pomalidomide/Pomalidomide
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/12 (8.33%)   4/10 (40.00%)   0/6 (0.00%)   1/2 (50.00%) 
Infections and infestations         
Pneumonia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Sepsis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Upper respiratory tract infection  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Renal and urinary disorders         
Renal failure  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Acute respiratory failure  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Chronic respiratory failure  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Pulmonary embolism  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Pulmonary hypertension  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment Phase: Placebo Treatment Phase: Pomalidomide Extension Phase: Placebo/Pomalidomide Extension Phase: Pomalidomide/Pomalidomide
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   12/12 (100.00%)   9/10 (90.00%)   5/6 (83.33%)   2/2 (100.00%) 
Blood and lymphatic system disorders         
Anaemia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Splenomegaly  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Cardiac disorders         
Palpitations  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Ear and labyrinth disorders         
Tinnitus  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Eye disorders         
Conjunctivitis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Gastrointestinal disorders         
Abdominal pain upper  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Barrett's oesophagus  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Colonic polyp  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Constipation  1  1/12 (8.33%)  3/10 (30.00%)  0/6 (0.00%)  0/2 (0.00%) 
Diarrhoea  1  3/12 (25.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dry mouth  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dyspepsia  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Gastric polyps  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Gastrooesophageal reflux disease  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  1/2 (50.00%) 
Hiatus hernia  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Mouth ulceration  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Mucous stools  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Nausea  1  2/12 (16.67%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
General disorders         
Chills  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Device breakage  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Influenza like illness  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Non-cardiac chest pain  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Oedema  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Oedema peripheral  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Pain  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  1/2 (50.00%) 
Pyrexia  1  0/12 (0.00%)  1/10 (10.00%)  1/6 (16.67%)  0/2 (0.00%) 
Hepatobiliary disorders         
Biliary colic  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Cholecystitis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Cholelithiasis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hepatic steatosis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Immune system disorders         
Contrast media allergy  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Seasonal allergy  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Infections and infestations         
Bronchitis  1  0/12 (0.00%)  2/10 (20.00%)  0/6 (0.00%)  0/2 (0.00%) 
Diverticulitis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Folliculitis  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Gastroenteritis  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Infected skin ulcer  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Influenza  1  2/12 (16.67%)  0/10 (0.00%)  2/6 (33.33%)  0/2 (0.00%) 
Laryngitis viral  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Localised infection  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Lower respiratory tract infection  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Tooth abscess  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Upper respiratory tract infection  1  3/12 (25.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Urinary tract infection  1  2/12 (16.67%)  0/10 (0.00%)  2/6 (33.33%)  0/2 (0.00%) 
Viral upper respiratory tract infection  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Injury, poisoning and procedural complications         
Contusion  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Fall  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Ligament sprain  1  0/12 (0.00%)  2/10 (20.00%)  0/6 (0.00%)  0/2 (0.00%) 
Procedural pain  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Investigations         
Alanine aminotransferase increased  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Aspartate aminotransferase increased  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Blood creatinine increased  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Blood iron decreased  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Blood lactate dehydrogenase increased  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
C-reactive protein increased  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Heart rate increased  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hepatic enzyme increased  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
White blood cell count increased  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Metabolism and nutrition disorders         
Hyperglycaemia  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Hypomagnesaemia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hypophosphataemia  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Iron deficiency  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Vitamin D deficiency  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  4/12 (33.33%)  4/10 (40.00%)  0/6 (0.00%)  0/2 (0.00%) 
Joint swelling  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Muscle spasms  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Musculoskeletal chest pain  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Myalgia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Pain in jaw  1  0/12 (0.00%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Synovitis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Tendon disorder  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Tendon pain  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Seborrhoeic keratosis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Nervous system disorders         
Ageusia  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dizziness  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Headache  1  3/12 (25.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hypoaesthesia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Migraine  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Psychiatric disorders         
Anxiety  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Depressed mood  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Insomnia  1  1/12 (8.33%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Renal and urinary disorders         
Dysuria  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Glycosuria  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Renal failure  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Reproductive system and breast disorders         
Amenorrhoea  1  1/12 (8.33%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Nipple pain  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Ovarian cyst ruptured  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  1/12 (8.33%)  1/10 (10.00%)  1/6 (16.67%)  0/2 (0.00%) 
Dysphonia  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dyspnoea  1  2/12 (16.67%)  0/10 (0.00%)  0/6 (0.00%)  1/2 (50.00%) 
Oropharyngeal pain  1  2/12 (16.67%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Productive cough  1  0/12 (0.00%)  0/10 (0.00%)  2/6 (33.33%)  0/2 (0.00%) 
Pulmonary hypertension  1  1/12 (8.33%)  0/10 (0.00%)  1/6 (16.67%)  1/2 (50.00%) 
Rhinorrhoea  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Sinus congestion  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Skin and subcutaneous tissue disorders         
Alopecia  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Butterfly rash  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dermatitis acneiform  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Digital ulcer  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Dry skin  1  1/12 (8.33%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Eczema  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hyperhidrosis  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Hyperkeratosis  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Macule  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Night sweats  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Papule  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Rash  1  1/12 (8.33%)  2/10 (20.00%)  0/6 (0.00%)  0/2 (0.00%) 
Rash maculo-papular  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Rash papular  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Rash pruritic  1  0/12 (0.00%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Rosacea  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Seborrhoeic dermatitis  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Skin ulcer  1  2/12 (16.67%)  0/10 (0.00%)  2/6 (33.33%)  0/2 (0.00%) 
Toxic skin eruption  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Social circumstances         
Menopause  1  1/12 (8.33%)  0/10 (0.00%)  0/6 (0.00%)  0/2 (0.00%) 
Vascular disorders         
Hot flush  1  1/12 (8.33%)  0/10 (0.00%)  1/6 (16.67%)  0/2 (0.00%) 
Hypertension  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Raynaud's phenomenon  1  0/12 (0.00%)  1/10 (10.00%)  0/6 (0.00%)  0/2 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
Based upon interim analysis data, the study did not meet its primary endpoints for subjects who had completed blinded treatment. The IDMC recommended the study be stopped due to lack of efficacy and the sponsor agreed with this recommendation.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission or defer publication to permit patent applications.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trial Disclosure
Organization: Celgene Corporation
Phone: 888-260-1599
EMail: ClinicalTrialDisclosure@Celgene.com
Publications:
Hsu V, et al. A Phase 2 Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness in Subjects with Systemic Sclerosis with Interstitial Lung Disease. Presented at the 2016 ACR/ARHP Annual Meeting, November 11-16, 2016, Washington, DC. Abstract No. 823.
Layout table for additonal information
Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT01559129    
Other Study ID Numbers: CC-4047-SSC-001
2010-023047-15 ( EudraCT Number )
First Submitted: March 19, 2012
First Posted: March 21, 2012
Results First Submitted: November 3, 2017
Results First Posted: October 18, 2019
Last Update Posted: October 18, 2019