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

• ClinicalTrials.gov Identifier: NCT01559129
• Recruitment Status: Terminated
• First Posted: March 21, 2012
• Results First Posted: October 18, 2019
• Last Update Posted: October 18, 2019
• Sponsor: Celgene
• 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

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Placebo	Pomalidomide
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.

Baseline Characteristics

Arm/Group Title		Placebo	Pomalidomide	Total
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
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)

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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.

Outcome Measure Data

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
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
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

Outcome Measure Data

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
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
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)

Outcome Measure Data

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
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
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)

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and a Week 52 value

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at the time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and a post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a Baseline value and post-baseline value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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

Outcome Measure Data

Analysis Population Description

FAS participants with available data

Arm/Group Title	Placebo	Pomalidomide
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
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).

Outcome Measure Data

Analysis Population Description

FAS participants with a value at each time point.

Arm/Group Title		Placebo	Pomalidomide
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
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.

Outcome Measure Data

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
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.

Adverse Events

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
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	--/--	--/--	--/--	--/--
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
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

Limitations and Caveats

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.

More Information

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

• Name/Title: Clinical Trial Disclosure
• Organization: Celgene Corporation
• Phone: 888-260-1599
• EMail: ClinicalTrialDisclosure@Celgene.com

Publications:

Hsu VM, Denton CP, Domsic RT, Furst DE, Rischmueller M, Stanislav M, Steen VD, Distler JHW, Korish S, Cooper A, Choi S, Schafer PH, Horan G, Hough DR. Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study. J Rheumatol. 2018 Mar;45(3):405-410. doi: 10.3899/jrheum.161040. Epub 2017 Nov 1.

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.

• 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