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Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy) (IIM)

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ClinicalTrials.gov Identifier: NCT02728752
Recruitment Status : Completed
First Posted : April 5, 2016
Results First Posted : April 21, 2021
Last Update Posted : April 21, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Dermatomyositis
Interventions Drug: Octagam 10%
Other: Placebo
Enrollment 95
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period. Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Period Title: First Period
Started 48 47
Completed 46 45
Not Completed 2 2
Period Title: Extension Period
Started 46 45
Completed 35 34
Not Completed 11 11
Arm/Group Title Placebo Octagam10% Total
Hide Arm/Group Description Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period. Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period. Total of all reporting groups
Overall Number of Baseline Participants 48 47 95
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 48 participants 47 participants 95 participants
51.35  (12.979) 54.04  (13.838) 52.68  (13.407)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants 47 participants 95 participants
Female
35
  72.9%
36
  76.6%
71
  74.7%
Male
13
  27.1%
11
  23.4%
24
  25.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants 47 participants 95 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
   2.1%
1
   2.1%
2
   2.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
3
   6.3%
2
   4.3%
5
   5.3%
White
43
  89.6%
44
  93.6%
87
  91.6%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   2.1%
0
   0.0%
1
   1.1%
1.Primary Outcome
Title Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS)
Hide Description Proportion of responders in the 2.0 g/kg Octagam 10% and placebo arms at Week 16 relative to baseline (Week 0). A responder being defined as a patient with an increase of ≥20 points on the Total Improvement Score (TIS, a scale from 0 to 100; 20-39 points being minimal improvement, 40-59 points being moderate improvement, and ≥60 points being major improvement
Time Frame At week 16
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 48 47
Measure Type: Count of Participants
Unit of Measure: Participants
21
  43.8%
37
  78.7%
2.Secondary Outcome
Title Proportion of TIS Responders by Improvement Category at Week 16
Hide Description

The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.

Primary: Total number of all patients who had at least minimal, moderate, or major response.

At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.

Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Only responders are displayed as the intent of the outcome measure is to present the proportion of responders. And patients not accounted for were non-responders.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Octagam 10%: Patients to be treated with Octagam 10%

Overall Number of Participants Analyzed 48 47
Measure Type: Count of Participants
Unit of Measure: Participants
Primary
21
  43.8%
37
  78.7%
At least moderate improvement
11
  22.9%
32
  68.1%
At least major improvement
4
   8.3%
15
  31.9%
3.Secondary Outcome
Title Proportion of TIS Responders by Improvement Category at Week 40
Hide Description

The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.

At Least Minimal: Total number of all patients who had minimal, moderate, or major response.

At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.

Time Frame 40 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Only responders are displayed as the intent of the outcome measure is to present the proportion of responders. And patients not accounted for were non-responders.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 46 45
Measure Type: Count of Participants
Unit of Measure: Participants
At least minimal improvement
32
  69.6%
32
  71.1%
At least moderate improvement
28
  60.9%
26
  57.8%
At least major improvement
14
  30.4%
17
  37.8%
4.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
Hide Description

The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed.

Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total activity score as well as for the total damage score the sub-scores will be added up. The score for total activity ranges from 0 to 100, for total damage from 0 to 32 points. Higher scores indicate greater disease activity or greater disease damage respectively.

Time Frame First 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: units on a scale
Total Activity Score -1.16  (7.000) -9.36  (10.542)
Total Damage Score -0.02  (0.771) -0.67  (1.871)
5.Secondary Outcome
Title Mean Change From End of First Period (Week 16) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
Hide Description

The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed

Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total activity score as well as for the total damage score the sub-scores will be added up. The score for total activity ranges from 0 to 100, for total damage from 0 to 32 points. Higher scores indicate greater disease activity or greater disease damage respectively.

Time Frame From week 16 to Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 33 34
Mean (Standard Deviation)
Unit of Measure: units on a scale
Total Activity Score -8.52  (11.344) -1.79  (4.169)
Total Damage Score -0.24  (0.969) 0.26  (2.220)
6.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: SF-36v2 Health Survey
Hide Description

The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.

SF-36 scores ranging from 0 to 100. 0 represented the lowest possible score (worst health state) and 100 represented the highest possible score (best health state), with scores in between representing the percentages of the total possible score achieved by respondents on a given scale.

Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Numbers of patients analyzed different due to patient discontinuations through study.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 43
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline to Week 16 Number Analyzed 43 participants 43 participants
2.27  (4.598) 7.06  (8.220)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
6.65  (8.258) 8.77  (10.930)
7.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Physician's Global Disease Activity
Hide Description 10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline to Week 16 Number Analyzed 43 participants 45 participants
-0.60  (1.815) -2.39  (1.987)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
-2.93  (1.888) -3.06  (1.817)
8.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Patient Global Disease Activity
Hide Description Patient's Global Disease Activity (10cm VAS assessing the overall activity of the patient's disease today from "No evidence of disease activity" to "Extremely active or severe disease activity", Disease Activity being active inflammation in the patient's muscles, skin, joints, intestines, heart, lungs or other parts of the body, which can improve when treated with medicines). 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline to Week 16 Number Analyzed 43 participants 45 participants
-1.11  (2.094) -2.19  (2.276)
Baseline to Week 40 Number Analyzed 35 participants 33 participants
-2.77  (2.133) -2.71  (2.651)
9.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: MMT-8
Hide Description Manual Muscle Testing - MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 - 150]. Higher score associated with better outcome.
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline to Week 16 Number Analyzed 43 participants 45 participants
3.21  (9.390) 14.38  (14.581)
Baseline to Week 40 Number Analyzed 35 participants 33 participants
12.00  (7.491) 20.09  (14.486)
10.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Health Assessment Questionnaire
Hide Description Health Assessment Questionnaire (HAQ); a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8). Assessment completed by patients. Lowest score 0 highest score 24. Higher score associated with worse outcome.
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline to Week 16 Number Analyzed 43 participants 45 participants
-0.16  (0.366) -0.56  (0.590)
Baseline to Week 40 Number Analyzed 34 participants 33 participants
-0.54  (0.524) -0.66  (0.805)
11.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Alanine Aminotransferase)
Hide Description [Not Specified]
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 42
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline to Week 16 Number Analyzed 43 participants 42 participants
-4.47  (29.796) -8.52  (18.474)
Baseline to Week 40 Number Analyzed 35 participants 33 participants
-4.06  (14.787) -7.15  (18.480)
12.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aspartate Aminotransferase)
Hide Description [Not Specified]
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 44
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline to Week 16 Number Analyzed 43 participants 44 participants
-3.07  (31.317) -7.82  (26.139)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
-2.20  (16.421) -7.76  (26.431)
13.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Lactate Dehydrogenase)
Hide Description [Not Specified]
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline to Week 16 Number Analyzed 43 participants 45 participants
-19.79  (142.012) -11.04  (165.252)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
-33.43  (63.651) -59.21  (92.801)
14.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aldolase)
Hide Description [Not Specified]
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 41 42
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline to Week 16 Number Analyzed 41 participants 42 participants
-2.51  (12.077) -0.48  (7.743)
Baseline to Week 40 Number Analyzed 35 participants 32 participants
-0.59  (7.839) -1.48  (3.644)
15.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Creatine Kinase)
Hide Description [Not Specified]
Time Frame From start of the trial till Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 44
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline to Week 16 Number Analyzed 43 participants 44 participants
-352.79  (2141.534) -169.20  (434.224)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
-56.54  (556.928) -169.38  (449.210)
16.Secondary Outcome
Title Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Extra-muscular Activity
Hide Description 10 cm VAS assessing extra-muscular activity from "No evidence of disease activity" to "Extremely active or severe disease activity". It encompasses an overall evaluation for the disease activity in all the extramuscular organ systems and excludes muscle disease activity. Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Time Frame From start of the trial until Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline to Week 16 Number Analyzed 43 participants 45 participants
-0.94  (2.287) -2.18  (2.134)
Baseline to Week 40 Number Analyzed 35 participants 34 participants
-2.64  (2.092) -2.67  (2.061)
17.Secondary Outcome
Title Mean TIS From Baseline (Week 0) to End of First Period (Week 16) and From Baseline (Week 0) to End of Extension Period (Week 40)
Hide Description The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.
Time Frame Up to 40 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Different numbers of patients analyzed in each group due to patient discontinuation.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 43 45
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 16 Number Analyzed 43 participants 45 participants
21.57  (20.185) 48.44  (24.444)
Week 40 Number Analyzed 35 participants 34 participants
51.07  (18.314) 55.44  (21.712)
18.Secondary Outcome
Title Time to Minimal, Moderate and Major Improvement in TIS
Hide Description When interpreting these time to event evaluations, one has to keep in mind that eventually all patients were treated with octagam 10%, so that differences in time to response for the Overall Period reflect this delayed start of treatment rather than the true difference between treatment with octagam 10% and placebo.
Time Frame Up to 40 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Number of participants based on number of responders. 95% confidence interval not available for all measurements.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 48 47
Median (95% Confidence Interval)
Unit of Measure: days
Time to Minimal Improvement - First Period Number Analyzed 23 participants 42 participants
115.0 [1] 
(84.0 to NA)
35.0
(29.0 to 58.0)
Time to Moderate Improvement - First Period Number Analyzed 11 participants 34 participants
NA [1] 
(NA to NA)
85.0
(58.0 to 113.0)
Time to Major Improvement - First Period Number Analyzed 5 participants 15 participants
NA [1] 
(NA to NA)
NA [1] 
(115.0 to NA)
Time to Minimal Improvement - From Start of First Treatment in Overall Period Number Analyzed 41 participants 47 participants
114.0
(84.0 to 195.0)
35.0
(29.0 to 58.0)
Time to Moderate Improvement - From Start of First Treatment in Overall Period Number Analyzed 36 participants 39 participants
197.0
(195.0 to 282.0)
85.0
(58.0 to 113.0)
Time to Major Improvement - From Start of First Treatment in Overall Period Number Analyzed 20 participants 25 participants
283.0 [1] 
(203.0 to NA)
283.0
(197.0 to 290.0)
[1]
Insufficient number of participants with events
19.Secondary Outcome
Title Time to Confirmed Deterioration in the First Period and Overall
Hide Description Confirmed deterioration is defined as disease worsening on 2 consecutive visits. Worsening criteria are defined as either Physician's Global Disease Activity worsening ≥2cm and Manual Muscle Testing worsening ≥20% or Extra-muscular Activity worsening ≥2cm or any 3 of the following scores worsening by ≥30%: Physician's Global Disease Activity, Manual Muscle Test, Extra-muscular Activity, Patient's Global Disease Activity or Health Assessment Questionnaire.
Time Frame Up to 40 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All patients analyzed and only those that had confirmed deterioration are presented.
Arm/Group Title Placebo Octagam10%
Hide Arm/Group Description:
Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
Overall Number of Participants Analyzed 3 1
Median (Full Range)
Unit of Measure: days
First Period Number Analyzed 3 participants 0 participants
58
(57 to 117)
Overall Period Number Analyzed 3 participants 1 participants
58
(57 to 117)
142 [1] 
(NA to NA)
[1]
Single participant
Time Frame 40 weeks
Adverse Event Reporting Description Note that the AE tables summarize data by the actual treatment at the time of the AE. An AE was considered to be associated with the most recent treatment administered, octagam 10% or placebo. Patients who were switched to the alternate treatment during the First Period were therefore considered to be 'at risk' for AEs in both treatment groups.
 
Arm/Group Title First Period Placebo Overall Period Octagam
Hide Arm/Group Description Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but with not further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period. Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.
All-Cause Mortality
First Period Placebo Overall Period Octagam
Affected / at Risk (%) Affected / at Risk (%)
Total   0/48 (0.00%)      0/95 (0.00%)    
Hide Serious Adverse Events
First Period Placebo Overall Period Octagam
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/48 (4.17%)      14/95 (14.74%)    
Cardiac disorders     
Sinus tachycardia  1  1/48 (2.08%)  2 0/95 (0.00%)  0
Ventricular extrasystoles  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Cardiac failure congestive  1  0/48 (0.00%)  0 1/95 (1.05%)  1
General disorders     
Condition aggravated  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Infections and infestations     
Sepsis  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Tropical spastic paresis  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Atypical pneumonia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Escherichia bacteraemia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pneumonia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Musculoskeletal and connective tissue disorders     
Muscle spasms  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Squamous cell carcinoma  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Nervous system disorders     
Cerebral infarction  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Cerebrovascular accident  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hypoaesthesia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Loss of consciousness  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Renal and urinary disorders     
Acute kidney injury  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Respiratory, thoracic and mediastinal disorders     
Dyspnoea  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pulmonary embolism  1  0/48 (0.00%)  0 3/95 (3.16%)  3
Acute respiratory failure  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Vascular disorders     
Hypertension  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Deep vein thrombosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
1
Term from vocabulary, MedDRA (Unspecified)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
First Period Placebo Overall Period Octagam
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   28/48 (58.33%)      84/95 (88.42%)    
Blood and lymphatic system disorders     
Lymphopenia  1  1/48 (2.08%)  1 2/95 (2.11%)  2
Anaemia  1  0/48 (0.00%)  0 3/95 (3.16%)  3
Haemolysis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Thrombocytosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Cardiac disorders     
Atrioventricular block first degree  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Bradycardia  1  2/48 (4.17%)  2 1/95 (1.05%)  2
Mitral valve calcification  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Palpitations  1  2/48 (4.17%)  2 1/95 (1.05%)  1
Sinus tachycardia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tachycardia  1  2/48 (4.17%)  2 5/95 (5.26%)  6
Ventricular extrasystoles  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pulmonary valve incompetence  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Ear and labyrinth disorders     
Deafness  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Vertigo  1  1/48 (2.08%)  1 2/95 (2.11%)  2
Endocrine disorders     
Adrenal insufficiency  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hyperthyroidism  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Eye disorders     
Cataract  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Conjunctival haemorrhage  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Eye pruritus  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Ocular hyperaemia  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Vision blurred  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Eye disorder  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Gastrointestinal disorders     
Abdominal pain  1  1/48 (2.08%)  1 2/95 (2.11%)  2
Abdominal pain upper  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Constipation  1  0/48 (0.00%)  0 5/95 (5.26%)  5
Diarrhoea  1  2/48 (4.17%)  2 4/95 (4.21%)  7
Dry mouth  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Faecal incontinence  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Haemorrhoids  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hypoaesthesia oral  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Nausea  1  2/48 (4.17%)  2 18/95 (18.95%)  30
Oral pain  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Vomiting  1  0/48 (0.00%)  0 9/95 (9.47%)  12
Dysphagia  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Frequent bowel movements  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Gastritis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
General disorders     
Asthenia  1  1/48 (2.08%)  1 3/95 (3.16%)  7
Catheter site erythema  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Chest discomfort  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Chest pain  1  3/48 (6.25%)  3 3/95 (3.16%)  3
Chills  1  1/48 (2.08%)  2 7/95 (7.37%)  12
Condition aggravated  1  9/48 (18.75%)  22 9/95 (9.47%)  13
Extravastation  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Fatigue  1  4/48 (8.33%)  4 5/95 (5.26%)  6
Feeling cold  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Infusion site bruising  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Injection site pain  1  0/48 (0.00%)  0 2/95 (2.11%)  4
Malaise  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Oedema peripheral  1  0/48 (0.00%)  0 3/95 (3.16%)  3
Pain  1  0/48 (0.00%)  0 2/95 (2.11%)  3
Peripheral swelling  1  1/48 (2.08%)  1 3/95 (3.16%)  3
Pyrexia  1  3/48 (6.25%)  3 17/95 (17.89%)  26
Influenza like illness  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infusion site extravasation  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infusion site pain  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infusion site reaction  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infusion site swelling  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Injection site erythema  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Injection site induration  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Local swelling  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Nodule  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infusion site erythema  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hepatobiliary disorders     
Hepatic steatosis  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Cholecystitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Cholelithiasis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Immune system disorders     
Hypersensitivity  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Infections and infestations     
Asymptomatic bacteriuria  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Bronchitis  1  2/48 (4.17%)  2 3/95 (3.16%)  3
Cellulitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Conjunctivitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Gastroenteritis viral  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Influenza  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Localised infection  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Nasopharyngitis  1  1/48 (2.08%)  1 6/95 (6.32%)  8
Rash pustular  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tooth abscess  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Upper respiratory tract infection  1  2/48 (4.17%)  2 3/95 (3.16%)  4
Cystitis  1  0/48 (0.00%)  0 2/95 (2.11%)  3
Diverticulitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Herpes zoster  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Lyme disease  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pharyngitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pneumonia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pyelonephritis acute  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Sinusitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tooth infection  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tracheitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Urinary tract infection  1  0/48 (0.00%)  0 2/95 (2.11%)  3
Injury, poisoning and procedural complications     
Contusion  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Fall  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Foot fracture  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tendon injury  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tendon rupture  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Overdose  1  2/48 (4.17%)  2 0/95 (0.00%)  0
Joint injury  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Rib fracture  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Investigations     
Alanine aminotransferase increased  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Aspartate aminotransferase increased  1  2/48 (4.17%)  2 1/95 (1.05%)  1
Blood glucose increased  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Blood lactate dehydrogenase increased  1  2/48 (4.17%)  2 0/95 (0.00%)  0
Blood potassium increased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Blood pressure decreased  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Blood pressure diastolic increased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Blood pressure increased  1  3/48 (6.25%)  4 4/95 (4.21%)  8
Blood pressure systolic increased  1  0/48 (0.00%)  0 3/95 (3.16%)  3
Blood urea increased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Body temperature increased  1  0/48 (0.00%)  0 5/95 (5.26%)  8
Coombs test positive  1  0/48 (0.00%)  0 5/95 (5.26%)  5
Haemoglobin decreased  1  1/48 (2.08%)  1 4/95 (4.21%)  5
Lymphocyte count decreased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Neutrophil count decreased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Weight decreased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
White blood cell count decreased  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Blood creatinine increased  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Cardiac murmur  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Free haemoglobin present  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Heart rate increased  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Muscle enzyme increased  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Metabolism and nutrition disorders     
Diabetes mellitus inadequate control  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hypokalaemia  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Lactic acidosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Decreased appetite  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hyperglycaemia  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Hyperlipidaemia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Hyponatraemia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Malnutrition  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Musculoskeletal and connective tissue disorders     
Arthralgia  1  4/48 (8.33%)  5 7/95 (7.37%)  8
Back pain  1  1/48 (2.08%)  1 4/95 (4.21%)  6
Bursitis  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Joint swelling  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Muscle spasms  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Muscle tightness  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Muscular weakness  1  0/48 (0.00%)  0 3/95 (3.16%)  3
Musculoskeletal chest pain  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Myalgia  1  0/48 (0.00%)  0 9/95 (9.47%)  12
Musculoskeletal pain  1  1/48 (2.08%)  1 2/95 (2.11%)  3
Myopathy  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Arthritis  1  0/48 (0.00%)  0 2/95 (2.11%)  2
Flank pain  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Intervertebral disc protrusion  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Muscle fatigue  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Osteoarthritis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pain in extremity  1  0/48 (0.00%)  0 6/95 (6.32%)  9
Spinal osteoarthritis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tendonitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tenosynovitis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Thyroid neoplasm  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Skin papilloma  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Nervous system disorders     
Basilar artery stenosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Dizziness  1  3/48 (6.25%)  3 5/95 (5.26%)  6
Headache  1  4/48 (8.33%)  10 46/95 (48.42%)  102
Hypoaesthesia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Paraesthesia  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Somnolence  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Tremor  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Psychiatric disorders     
Insomnia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Depression  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Renal and urinary disorders     
Acute kidney injury  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Urinary retention  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Urine abnormality  1  0/48 (0.00%)  0 1/95 (1.05%)  7
Dysuria  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pollakiuria  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Respiratory, thoracic and mediastinal disorders     
Dysphonia  1  2/48 (4.17%)  2 0/95 (0.00%)  0
Dyspnoea  1  1/48 (2.08%)  1 5/95 (5.26%)  5
Epistaxis  1  1/48 (2.08%)  1 2/95 (2.11%)  2
Oropharyngeal pain  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Throat irritation  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Pulmonary hypertension  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Cough  1  0/48 (0.00%)  0 4/95 (4.21%)  4
Pulmonary congestion  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pulmonary fibrosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Rhinorrhoea  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Tachypnoea  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Skin and subcutaneous tissue disorders     
Diffuse alopecia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Erythema  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Night sweats  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Pruritus  1  2/48 (4.17%)  3 1/95 (1.05%)  1
Rash  1  1/48 (2.08%)  1 6/95 (6.32%)  6
Telangiectasia  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Alopecia  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Dermatitis allergic  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Poikiloderma  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Pruritus generalised  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Rash generalised  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Skin burning sensation  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Skin lesion  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Urticaria  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Urticaria contact  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Social circumstances     
Menopause  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Postmenopause  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Vascular disorders     
Deep vein thrombosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
Essential hypertension  1  1/48 (2.08%)  1 0/95 (0.00%)  0
Hot flush  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Hypertension  1  3/48 (6.25%)  3 5/95 (5.26%)  6
Hypotension  1  1/48 (2.08%)  1 1/95 (1.05%)  1
Peripheral coldness  1  0/48 (0.00%)  0 1/95 (1.05%)  2
Brachiocephalic arteriosclerosis  1  0/48 (0.00%)  0 1/95 (1.05%)  1
1
Term from vocabulary, MedDRA (Unspecified)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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 Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mikaela Raymond
Organization: CRMG
Phone: 866-337-1868
EMail: ctgov@clinicalresearchmgt.com
Layout table for additonal information
Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02728752    
Other Study ID Numbers: GAM10-08
First Submitted: March 11, 2016
First Posted: April 5, 2016
Results First Submitted: November 2, 2020
Results First Posted: April 21, 2021
Last Update Posted: April 21, 2021