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Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03039686
Recruitment Status : Completed
First Posted : February 1, 2017
Results First Posted : December 21, 2020
Last Update Posted : December 21, 2020
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Interventions Drug: RO7239361
Drug: Placebo for RO7239361
Enrollment 166
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Period Title: Double-blind Period
Started 56 55 55
Completed 29 26 32
Not Completed 27 29 23
Reason Not Completed
Administrative Reason by Sponsor             24             25             21
Subject Request to Discontinue Study Treatment             1             2             0
Withdrawal by Subject             2             2             1
Death             0             0             1
Period Title: Open Label Period
Started 0 [1] 38 [2] 42 [3]
Completed DB, Did Not Start OL 2 2 3
Completed 0 0 0
Not Completed 0 38 42
Reason Not Completed
Administrative Reason by Sponsor             0             34             41
Subject Request to Discontinue Study Treatment             0             1             0
Withdrawal by Subject             0             3             1
[1]
Double-blind (DB) placebo participants switched to RO7239361 (RO) for the open label phase.
[2]
14 DB placebo participants switched to the RO Low Dose for the open-label phase.
[3]
13 DB placebo participants switched to the RO High Dose for the open-label phase.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose Total
Hide Arm/Group Description Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Total of all reporting groups
Overall Number of Baseline Participants 56 55 55 166
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 56 participants 55 participants 55 participants 166 participants
8.4  (1.7) 8.5  (1.8) 8.4  (1.5) 8.5  (1.7)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Children (6-11 years) Number Analyzed 56 participants 55 participants 55 participants 166 participants
56 55 55 166
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 56 participants 55 participants 55 participants 166 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
56
 100.0%
55
 100.0%
55
 100.0%
166
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 56 participants 55 participants 55 participants 166 participants
Hispanic or Latino
7
  12.5%
5
   9.1%
6
  10.9%
18
  10.8%
Not Hispanic or Latino
43
  76.8%
41
  74.5%
43
  78.2%
127
  76.5%
Unknown or Not Reported
6
  10.7%
9
  16.4%
6
  10.9%
21
  12.7%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 56 participants 55 participants 55 participants 166 participants
Asian 9 7 7 23
Native Hawaiian or other Pacific Islander 0 0 1 1
White 42 45 45 132
Other 5 3 2 10
1.Primary Outcome
Title Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
Hide Description The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: score on a scale
23.1  (6.4) 24.5  (5.5) 22.7  (6.7)
2.Primary Outcome
Title Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Hide Description The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
-2.99  (0.65) -3.44  (0.67) -2.41  (0.64)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.45
Confidence Interval (2-Sided) 95%
-2.17 to 1.27
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.87
Estimation Comments Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age Interactive response system (IXRS) Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.58
Confidence Interval (2-Sided) 95%
-1.10 to 2.26
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.85
Estimation Comments Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
3.Secondary Outcome
Title Baseline Time for 4 Stair Climb
Hide Description The time to complete the 4 stair climb was measured at baseline.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: seconds (secs)
3.81  (1.55) 3.85  (1.61) 3.92  (1.91)
4.Secondary Outcome
Title Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Hide Description 4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: stairs/sec
-0.15  (0.07) -0.15  (0.07) -0.07  (0.07)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.00
Confidence Interval (2-Sided) 95%
-0.21 to 0.20
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.10
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.08
Confidence Interval (2-Sided) 95%
-0.12 to 0.27
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.10
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
5.Secondary Outcome
Title Baseline for the Time to Stand From Supine
Hide Description The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: secs
6.28  (4.75) 6.15  (4.07) 7.24  (9.22)
6.Secondary Outcome
Title Change From Baseline at Week 48 in Stand From Supine Velocity
Hide Description The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=28, Low Dose n=28, High Dose n=32.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: 1/sec
-0.05  (0.01) -0.02  (0.01) -0.02  (0.01)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.03
Confidence Interval (2-Sided) 95%
-0.00 to 0.06
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.02
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.03
Confidence Interval (2-Sided) 95%
-0.00 to 0.06
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.02
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
7.Secondary Outcome
Title Baseline Time for 10 Meter Walk/Run
Hide Description The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: secs
5.38  (1.48) 5.51  (1.68) 5.68  (2.30)
8.Secondary Outcome
Title Change From Baseline at Week 48 in 10 M Walk/Run Velocity
Hide Description The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=31.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: m/sec
-0.23  (0.06) -0.14  (0.07) -0.23  (0.06)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.09
Confidence Interval (2-Sided) 95%
-0.08 to 0.27
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.09
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
-0.17 to 0.18
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.09
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
9.Secondary Outcome
Title Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Hide Description The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: score on a scale
85.59  (10.21) 86.54  (9.52) 84.47  (14.76)
10.Secondary Outcome
Title Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Hide Description The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=31, Low Dose n=29, High Dose n=34.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
-5.47  (1.79) -7.47  (1.83) -4.51  (1.77)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -2.00
Confidence Interval (2-Sided) 95%
-6.76 to 2.77
Parameter Dispersion
Type: Standard Error of the Mean
Value: 2.41
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.96
Confidence Interval (2-Sided) 95%
-3.71 to 5.63
Parameter Dispersion
Type: Standard Error of the Mean
Value: 2.36
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
11.Secondary Outcome
Title Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Hide Description Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Number analyzed is the number of participants with data available for analyses at the given timepoint.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: kilogram (kg)
Baseline: Knee Extenders Number Analyzed 56 participants 55 participants 54 participants
5.58  (2.87) 6.25  (3.63) 5.76  (3.53)
Change from Baseline at Week 48: Knee Extenders Number Analyzed 30 participants 28 participants 33 participants
-1.19  (2.13) -0.47  (2.43) -0.88  (2.97)
Baseline: Knee Flexors Number Analyzed 56 participants 55 participants 54 participants
5.04  (2.58) 5.70  (3.08) 5.04  (2.72)
Change from Baseline at Week 48: Knee Flexors Number Analyzed 30 participants 28 participants 33 participants
0.15  (2.24) 0.08  (2.53) -0.13  (2.29)
12.Secondary Outcome
Title Baseline for the 6 Minute Walk Distance (6MWD)
Hide Description The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Mean (Standard Deviation)
Unit of Measure: meters (m)
388.33  (69.59) 399.73  (68.35) 370.73  (93.35)
13.Secondary Outcome
Title Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
Hide Description The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=29, Low Dose n=25, High Dose n=31.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55
Least Squares Mean (Standard Error)
Unit of Measure: meters (m)
-41.3  (8.7) -39.6  (9.0) -30.0  (8.7)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.7
Confidence Interval (2-Sided) 95%
-21.1 to 24.6
Parameter Dispersion
Type: Standard Error of the Mean
Value: 11.5
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, RO7239361 High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 11.3
Confidence Interval (2-Sided) 95%
-11.0 to 33.6
Parameter Dispersion
Type: Standard Error of the Mean
Value: 11.3
Estimation Comments Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
14.Secondary Outcome
Title Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
Hide Description The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Included in the analysis are only those subjects for whom an efficacy assessment was completed at Week 48.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 36 37 31
Measure Type: Number
Unit of Measure: percentage of participants
Very much improved 0 0 0
Much improved 5.6 2.7 3.2
Minimally improved 13.9 13.5 19.4
No change 58.3 54.1 51.6
Minimally worse 16.7 18.9 22.6
Much worse 5.6 10.8 3.2
Very much worse 0 0 0
15.Secondary Outcome
Title Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Hide Description Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Arm/Group Title Placebo RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 17 19 15
Mean (Standard Deviation)
Unit of Measure: m/sec
Baseline Number Analyzed 17 participants 19 participants 15 participants
1.69  (0.33) 1.54  (0.35) 1.57  (0.46)
Change from Baseline at Week 48 Number Analyzed 5 participants 7 participants 4 participants
-0.25  (0.39) -0.22  (0.22) -0.28  (0.29)
16.Secondary Outcome
Title Number of Participants With Adverse Events (AEs)
Hide Description An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
Time Frame During DB period (48 weeks) and Whole study (up to approximately 34 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.
Arm/Group Title Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB RO7239361 Low Dose RO7239361 High Dose Whole Study
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55 69 68
Measure Type: Number
Unit of Measure: participants
Double-Blind Period Number Analyzed 56 participants 55 participants 55 participants 0 participants 0 participants
46 48 49
Whole Study Number Analyzed 0 participants 0 participants 0 participants 69 participants 68 participants
57 56
17.Secondary Outcome
Title Number of Participants With AEs Leading to Discontinuation
Hide Description An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.
Time Frame During DB period (48 weeks) and Whole study (up to approximately 34 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.
Arm/Group Title Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB RO7239361 Low Dose RO7239361 High Dose
Hide Arm/Group Description:
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period.
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Overall Number of Participants Analyzed 56 55 55 69 68
Measure Type: Number
Unit of Measure: participants
Double-Blind Period Number Analyzed 56 participants 55 participants 55 participants 0 participants 0 participants
0 0 0
Whole Study Number Analyzed 0 participants 0 participants 0 participants 69 participants 68 participants
0 0
Time Frame Up to approximately 34 months
Adverse Event Reporting Description Safety population included all enrolled participants who received at least 1 dose of study therapy.
 
Arm/Group Title Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB Placebo, Then RO7239361 Low Dose OL Placebo, Then RO7239361 High Dose OL RO7239361 Low Dose, Then RO7239361 Low Dose OL RO7239361 High Dose, Then RO7239361 High Dose OL
Hide Arm/Group Description Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Participants received matching placebo solution SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label (OL) period followed by 24 weeks of follow-up. Participants received matching placebo solution SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up
All-Cause Mortality
Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB Placebo, Then RO7239361 Low Dose OL Placebo, Then RO7239361 High Dose OL RO7239361 Low Dose, Then RO7239361 Low Dose OL RO7239361 High Dose, Then RO7239361 High Dose OL
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/56 (0.00%)      0/55 (0.00%)      1/55 (1.82%)      0/14 (0.00%)      0/13 (0.00%)      0/24 (0.00%)      0/29 (0.00%)    
Hide Serious Adverse Events
Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB Placebo, Then RO7239361 Low Dose OL Placebo, Then RO7239361 High Dose OL RO7239361 Low Dose, Then RO7239361 Low Dose OL RO7239361 High Dose, Then RO7239361 High Dose OL
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/56 (5.36%)      2/55 (3.64%)      4/55 (7.27%)      0/14 (0.00%)      0/13 (0.00%)      0/24 (0.00%)      0/29 (0.00%)    
Cardiac disorders               
Cardiac arrest † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Myocarditis † 1  1/56 (1.79%)  1 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Supraventricular tachycardia † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
General disorders               
Influenza like illness † 1  1/56 (1.79%)  1 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Hepatobiliary disorders               
Hyperbilirubinaemia † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Infections and infestations               
Gastrointestinal viral infection † 1  1/56 (1.79%)  1 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Viral infection † 1  0/56 (0.00%)  0 1/55 (1.82%)  1 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Injury, poisoning and procedural complications               
Humerus fracture † 1  0/56 (0.00%)  0 1/55 (1.82%)  1 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Overdose † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Metabolism and nutrition disorders               
Hypocalcaemia † 1  1/56 (1.79%)  1 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
1
Term from vocabulary, MedDRA 23.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo DB RO7239361 Low Dose DB RO7239361 High Dose DB Placebo, Then RO7239361 Low Dose OL Placebo, Then RO7239361 High Dose OL RO7239361 Low Dose, Then RO7239361 Low Dose OL RO7239361 High Dose, Then RO7239361 High Dose OL
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   43/56 (76.79%)      43/55 (78.18%)      47/55 (85.45%)      8/14 (57.14%)      7/13 (53.85%)      13/24 (54.17%)      12/29 (41.38%)    
Ear and labyrinth disorders               
Ear pain † 1  1/56 (1.79%)  1 1/55 (1.82%)  1 4/55 (7.27%)  7 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Gastrointestinal disorders               
Abdominal discomfort † 1  1/56 (1.79%)  2 1/55 (1.82%)  1 1/55 (1.82%)  1 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Abdominal pain † 1  1/56 (1.79%)  1 2/55 (3.64%)  2 3/55 (5.45%)  5 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Abdominal pain upper † 1  3/56 (5.36%)  4 4/55 (7.27%)  5 7/55 (12.73%)  12 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 1/29 (3.45%)  2
Constipation † 1  4/56 (7.14%)  5 4/55 (7.27%)  4 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 0/29 (0.00%)  0
Diarrhoea † 1  3/56 (5.36%)  3 10/55 (18.18%)  13 4/55 (7.27%)  8 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 2/29 (6.90%)  2
Dyspepsia † 1  0/56 (0.00%)  0 1/55 (1.82%)  1 3/55 (5.45%)  4 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Nausea † 1  1/56 (1.79%)  1 2/55 (3.64%)  2 5/55 (9.09%)  9 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 4/29 (13.79%)  4
Vomiting † 1  6/56 (10.71%)  6 8/55 (14.55%)  14 6/55 (10.91%)  11 0/14 (0.00%)  0 0/13 (0.00%)  0 3/24 (12.50%)  4 2/29 (6.90%)  2
General disorders               
Fatigue † 1  0/56 (0.00%)  0 4/55 (7.27%)  4 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 1/29 (3.45%)  1
Gait inability † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site bruising † 1  2/56 (3.57%)  3 4/55 (7.27%)  4 4/55 (7.27%)  4 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site erythema † 1  8/56 (14.29%)  25 11/55 (20.00%)  43 12/55 (21.82%)  38 0/14 (0.00%)  0 3/13 (23.08%)  17 2/24 (8.33%)  6 1/29 (3.45%)  1
Injection site induration † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site oedema † 1  3/56 (5.36%)  7 2/55 (3.64%)  9 1/55 (1.82%)  9 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site pruritus † 1  0/56 (0.00%)  0 2/55 (3.64%)  2 2/55 (3.64%)  6 0/14 (0.00%)  0 1/13 (7.69%)  12 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site reaction † 1  2/56 (3.57%)  3 4/55 (7.27%)  29 2/55 (3.64%)  33 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Injection site swelling † 1  0/56 (0.00%)  0 4/55 (7.27%)  31 3/55 (5.45%)  5 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 1/29 (3.45%)  1
Localised oedema † 1  3/56 (5.36%)  5 0/55 (0.00%)  0 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Pyrexia † 1  8/56 (14.29%)  8 9/55 (16.36%)  13 8/55 (14.55%)  9 2/14 (14.29%)  2 1/13 (7.69%)  1 3/24 (12.50%)  3 1/29 (3.45%)  1
Infections and infestations               
Ear infection † 1  0/56 (0.00%)  0 4/55 (7.27%)  4 1/55 (1.82%)  2 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Gastroenteritis † 1  1/56 (1.79%)  2 1/55 (1.82%)  1 3/55 (5.45%)  5 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Gastroenteritis viral † 1  1/56 (1.79%)  1 1/55 (1.82%)  1 1/55 (1.82%)  1 1/14 (7.14%)  1 0/13 (0.00%)  0 1/24 (4.17%)  1 0/29 (0.00%)  0
Hordeolum † 1  0/56 (0.00%)  0 2/55 (3.64%)  2 0/55 (0.00%)  0 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 1/29 (3.45%)  1
Influenza † 1  3/56 (5.36%)  3 6/55 (10.91%)  6 1/55 (1.82%)  1 0/14 (0.00%)  0 0/13 (0.00%)  0 2/24 (8.33%)  2 0/29 (0.00%)  0
Nasopharyngitis † 1  13/56 (23.21%)  17 13/55 (23.64%)  17 13/55 (23.64%)  16 2/14 (14.29%)  2 0/13 (0.00%)  0 0/24 (0.00%)  0 2/29 (6.90%)  2
Pharyngitis † 1  2/56 (3.57%)  2 2/55 (3.64%)  2 3/55 (5.45%)  3 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Rhinitis † 1  1/56 (1.79%)  1 3/55 (5.45%)  5 4/55 (7.27%)  7 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Sinusitis † 1  1/56 (1.79%)  1 0/55 (0.00%)  0 3/55 (5.45%)  4 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Upper respiratory tract infection † 1  6/56 (10.71%)  8 4/55 (7.27%)  5 7/55 (12.73%)  16 1/14 (7.14%)  1 1/13 (7.69%)  1 0/24 (0.00%)  0 2/29 (6.90%)  2
Injury, poisoning and procedural complications               
Contusion † 1  2/56 (3.57%)  2 5/55 (9.09%)  7 4/55 (7.27%)  5 0/14 (0.00%)  0 1/13 (7.69%)  2 0/24 (0.00%)  0 2/29 (6.90%)  2
Fall † 1  5/56 (8.93%)  11 1/55 (1.82%)  1 5/55 (9.09%)  6 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 1/29 (3.45%)  1
Gadolinium deposition disease † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Ligament sprain † 1  4/56 (7.14%)  4 1/55 (1.82%)  2 1/55 (1.82%)  1 2/14 (14.29%)  2 0/13 (0.00%)  0 3/24 (12.50%)  3 1/29 (3.45%)  1
Skin abrasion † 1  2/56 (3.57%)  3 3/55 (5.45%)  12 0/55 (0.00%)  0 1/14 (7.14%)  3 0/13 (0.00%)  0 1/24 (4.17%)  4 1/29 (3.45%)  1
Thermal burn † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Investigations               
Bone density decreased † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Glutamate dehydrogenase increased † 1  2/56 (3.57%)  2 1/55 (1.82%)  1 3/55 (5.45%)  3 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Musculoskeletal and connective tissue disorders               
Arthralgia † 1  6/56 (10.71%)  6 7/55 (12.73%)  8 5/55 (9.09%)  6 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 1/29 (3.45%)  1
Back pain † 1  4/56 (7.14%)  4 7/55 (12.73%)  9 2/55 (3.64%)  2 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 1/29 (3.45%)  1
Mobility decreased † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Muscle spasms † 1  1/56 (1.79%)  1 2/55 (3.64%)  7 2/55 (3.64%)  6 1/14 (7.14%)  1 2/13 (15.38%)  2 0/24 (0.00%)  0 1/29 (3.45%)  1
Pain in extremity † 1  2/56 (3.57%)  3 4/55 (7.27%)  6 8/55 (14.55%)  11 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Nervous system disorders               
Headache † 1  9/56 (16.07%)  28 14/55 (25.45%)  60 10/55 (18.18%)  42 1/14 (7.14%)  3 0/13 (0.00%)  0 2/24 (8.33%)  9 2/29 (6.90%)  4
Migraine † 1  0/56 (0.00%)  0 1/55 (1.82%)  10 0/55 (0.00%)  0 1/14 (7.14%)  1 0/13 (0.00%)  0 1/24 (4.17%)  3 0/29 (0.00%)  0
Respiratory, thoracic and mediastinal disorders               
Cough † 1  10/56 (17.86%)  10 8/55 (14.55%)  11 7/55 (12.73%)  13 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 1/29 (3.45%)  2
Epistaxis † 1  3/56 (5.36%)  9 3/55 (5.45%)  15 6/55 (10.91%)  17 0/14 (0.00%)  0 1/13 (7.69%)  1 0/24 (0.00%)  0 0/29 (0.00%)  0
Nasal congestion † 1  1/56 (1.79%)  1 3/55 (5.45%)  3 3/55 (5.45%)  3 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 2/29 (6.90%)  2
Productive cough † 1  0/56 (0.00%)  0 3/55 (5.45%)  3 0/55 (0.00%)  0 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Rhinorrhoea † 1  1/56 (1.79%)  1 4/55 (7.27%)  7 3/55 (5.45%)  3 1/14 (7.14%)  2 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Skin and subcutaneous tissue disorders               
Erythema † 1  3/56 (5.36%)  4 2/55 (3.64%)  2 1/55 (1.82%)  1 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Rash † 1  4/56 (7.14%)  9 4/55 (7.27%)  5 7/55 (12.73%)  9 0/14 (0.00%)  0 0/13 (0.00%)  0 1/24 (4.17%)  1 1/29 (3.45%)  1
Rash macular † 1  0/56 (0.00%)  0 0/55 (0.00%)  0 0/55 (0.00%)  0 1/14 (7.14%)  1 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
Urticaria † 1  3/56 (5.36%)  4 2/55 (3.64%)  2 3/55 (5.45%)  4 0/14 (0.00%)  0 0/13 (0.00%)  0 0/24 (0.00%)  0 0/29 (0.00%)  0
1
Term from vocabulary, MedDRA 23.0
†
Indicates events were collected by systematic assessment
[Not Specified]
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.
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800 821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03039686    
Other Study ID Numbers: CN001-016
2016-001654-18 ( EudraCT Number )
WN40227 ( Other Identifier: Hoffman-La Roche )
First Submitted: January 27, 2017
First Posted: February 1, 2017
Results First Submitted: October 21, 2020
Results First Posted: December 21, 2020
Last Update Posted: December 21, 2020