Clinical Trial to Evaluate the Efficacy, Safety, and Tole... | NCT03039686 | Trialant
NCT03039686
Sponsor
Hoffmann-La Roche
Status
Completed
Last Update Posted
Dec 21, 2020Actual
Enrollment
166Actual
Phase
Phase 2Phase 3
Conditions
Duchenne Muscular Dystrophy
Interventions
RO7239361
Placebo for RO7239361
Countries
United States
Argentina
Australia
Belgium
Canada
France
Germany
Italy
Japan
Netherlands
Spain
Sweden
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT03039686
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
CN001-016
Secondary IDs
ID
Type
Description
Link
2016-001654-18
EudraCT Number
WN40227
Other Identifier
Hoffman-La Roche
Brief Title
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Official Title
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Acronym
Not provided
Organization
Hoffmann-La RocheINDUSTRY
Status Module
Record Verification Date
Nov 2020
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jul 6, 2017Actual
Primary Completion Date
Apr 28, 2020Actual
Completion Date
Apr 28, 2020Actual
First Submitted Date
Jan 27, 2017
First Submission Date that Met QC Criteria
Jan 31, 2017
First Posted Date
Feb 1, 2017Estimated
Results Waived
Not provided
Results First Submitted Date
Oct 21, 2020
Results First Submitted that Met QC Criteria
Nov 25, 2020
Results First Posted Date
Dec 21, 2020Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Nov 25, 2020
Last Update Posted Date
Dec 21, 2020Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Hoffmann-La RocheINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
Detailed Description
Not provided
Conditions Module
Conditions
Duchenne Muscular Dystrophy
Keywords
muscular dystrophy
Duchenne's Muscular Dystrophy
DMD
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
166Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
RO7239361 Low Dose
Experimental
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.
Drug: RO7239361
RO7239361 High Dose
Experimental
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.
Drug: RO7239361
Placebo
Placebo Comparator
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.
Drug: Placebo for RO7239361
Interventions
Name
Type
Description
Arm Group Labels
Other Names
RO7239361
Drug
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
RO7239361 High Dose
RO7239361 Low Dose
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
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.
Baseline
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
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).
Baseline, Week 48
Secondary Outcomes
Measure
Description
Time Frame
Baseline Time for 4 Stair Climb
The time to complete the 4 stair climb was measured at baseline.
Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
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).
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Diagnosed with DMD by confirmed medical history and genetic testing
Able to walk without assistance
Minimum North Star Ambulatory Assessment score of 15 at screening
Able to walk up 4 stairs in 8 seconds or less
Weigh at least 15 kg (33 lbs)
Taking corticosteroids for DMD
Exclusion Criteria:
Any behavior or mental issue that will affect the ability to complete the required study procedures
Previously or currently taking medications like androgens or human growth hormone
Use of a ventilator during the day
Unable to have blood samples collected or receive an injection under the skin
Concomitant or previous participation at any time in a gene therapy study
Other protocol defined Inclusion/Exclusion Criteria could apply.
Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.
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.
Take placebo subcutaneously on specified days over a 48 week blinded period
Placebo
Baseline, Week 48
Baseline for the Time to Stand From Supine
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
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).
Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
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.
Baseline
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
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).
Baseline, Week 48
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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.
Baseline
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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).
Baseline, Week 48
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
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.
Baseline, Week 48
Baseline for the 6 Minute Walk Distance (6MWD)
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Baseline
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
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).
Baseline, Week 48
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
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.
Baseline, Week 48
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
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.
Baseline, Week 48
Number of Participants With Adverse Events (AEs)
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.
During DB period (48 weeks) and Whole study (up to approximately 34 months)
Number of Participants With AEs Leading to Discontinuation
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.
During DB period (48 weeks) and Whole study (up to approximately 34 months)
Palo Alto
California
94304
United States
University of California Davis Medical Center
Sacramento
California
95817
United States
Yale University School of Medicine ; Pulmonary & Critical Care
New Haven
Connecticut
06510
United States
University of Florida
Gainesville
Florida
32607
United States
Nemours Children's Hospital
Orlando
Florida
32827
United States
Rare Disease Research, LLC
Atlanta
Georgia
30318
United States
Rush University Medical Center - PPDS
Chicago
Illinois
60612
United States
University of Iowa
Iowa City
Iowa
52242
United States
University of Kansas Medical Center
Kansas City
Kansas
66160
United States
Kennedy Krieger Institute
Baltimore
Maryland
21205
United States
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
Worcester
Massachusetts
01655
United States
Saint Louis Children's Hospital
St Louis
Missouri
63110
United States
Las Vegas Clinic
Las Vegas
Nevada
89145
United States
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
Cincinnati
Ohio
45229
United States
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
Columbus
Ohio
43205
United States
Seattle Children's Hospital
Seattle
Washington
98105
United States
Instituto centenario
Buenos Aires
C1204AAD
Argentina
Children's Hospital Westmead; Paediatrics & Child Health
Westmead
New South Wales
2145
Australia
Lady Cilento Children's Hospital; Neurosciences Department
South Brisbane
Queensland
4101
Australia
Royal Children's Hospital
Parkville
Victoria
3052
Australia
UZ Gent
Ghent
9000
Belgium
London Health Sciences Centre; Children's Hospital; Pediatrics
London
Ontario
N6A 5W9
Canada
Children'S Hospital of Eastern Ontario
Ottawa
Ontario
K1H 8L1
Canada
Hospices Civils de Lyon
Lyon
69004
France
Hotel Dieu; Service Pharmacie Essais Cliniques
Nantes
44093
France
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
Paris
75571
France
Hopitaux Universitaires de Strasbourg
Strasbourg
67091
France
Universitatsklinikum Essen; Innere Klinik
Essen
45122
Germany
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano
Emilia-Romagna
20162
Italy
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
Rome
Lazio
00168
Italy
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
Messina
Sicily
98125
Italy
Hyogo College of Medicine Hospital
Hyōgo
663-8501
Japan
Miyagi Children's Hospital
Miyagi
989-3126
Japan
Shinshu University Hospital
Nagano
390-8621
Japan
National Hospital Organization Osaka Toneyama Medical Center
Osaka
560-8552
Japan
National Center of Neurology and Psychiatry
Tokyo
187-8551
Japan
Leids Universitair Medisch Centrum
Leiden
2333 ZA
Netherlands
Radboud University Nijmegen Medical Centre; Ophthalmology
Nijmegen
6525 EX
Netherlands
Hospital Sant Joan De Deu
Esplugues de Llobregas
Barcelona
08950
Spain
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
Valencia
46026
Spain
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
Gothenburg
41685
Sweden
Alder Hey Children s Hospital; Department of Pediatrics
Liverpool
L12 2AP
United Kingdom
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London
WC1N 1EH
United Kingdom
Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne
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.
FG002
RO7239361 High Dose
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.
FG00056 subjects
FG00155 subjects
FG00255 subjects
COMPLETED
FG00029 subjects
FG00126 subjects
FG00232 subjects
NOT COMPLETED
FG00027 subjects
FG00129 subjects
FG00223 subjects
Type
Comment
Reasons
Administrative Reason by Sponsor
FG00024 subjects
FG00125 subjects
FG00221 subjects
Subject Request to Discontinue Study Treatment
FG0001 subjects
FG0012 subjects
FG0020 subjects
Withdrawal by Subject
FG0002 subjects
FG0012 subjects
FG0021 subjects
Death
FG0000 subjects
FG0010 subjects
FG0021 subjects
Open Label Period
Type
Comment
Milestone Data
STARTED
FG0000 subjectsDouble-blind (DB) placebo participants switched to RO7239361 (RO) for the open label phase.
FG00138 subjects14 DB placebo participants switched to the RO Low Dose for the open-label phase.
FG00242 subjects13 DB placebo participants switched to the RO High Dose for the open-label phase.
Completed DB, Did Not Start OL
FG0002 subjects
FG0012 subjects
FG0023 subjects
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
NOT COMPLETED
FG0000 subjects
FG00138 subjects
FG00242 subjects
Type
Comment
Reasons
Administrative Reason by Sponsor
FG0000 subjects
FG00134 subjects
FG00241 subjects
Subject Request to Discontinue Study Treatment
FG000
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Placebo
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.
BG001
RO7239361 Low Dose
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.
BG002
RO7239361 High Dose
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.
BG003
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00056
BG00155
BG00255
BG003166
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG0008.4± 1.7
BG0018.5± 1.8
BG0028.4± 1.5
BG003
Age, Customized
Number
participants
Title
Denominators
Categories
Children (6-11 years)
Title
Measurements
BG00056
BG00155
BG002
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0000
BG0010
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0007
BG0015
BG002
Race/Ethnicity, Customized
Number
participants
Title
Denominators
Categories
Asian
Title
Measurements
BG0009
BG0017
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
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.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
score on a scale
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG00023.1± 6.4
OG00124.5± 5.5
OG00222.7± 6.7
Primary
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
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).
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.
Posted
Least Squares Mean
Standard Error
score on a scale
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
Secondary
Baseline Time for 4 Stair Climb
The time to complete the 4 stair climb was measured at baseline.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
seconds (secs)
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
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.
Secondary
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
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).
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.
Posted
Least Squares Mean
Standard Error
stairs/sec
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
Secondary
Baseline for the Time to Stand From Supine
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
secs
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
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.
Secondary
Change From Baseline at Week 48 in Stand From Supine Velocity
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).
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.
Posted
Least Squares Mean
Standard Error
1/sec
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Baseline Time for 10 Meter Walk/Run
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.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
secs
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
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.
Secondary
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
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).
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.
Posted
Least Squares Mean
Standard Error
m/sec
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
score on a scale
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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).
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.
Posted
Least Squares Mean
Standard Error
score on a scale
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
Secondary
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
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.
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.
Posted
Mean
Standard Deviation
kilogram (kg)
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Baseline for the 6 Minute Walk Distance (6MWD)
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
meters (m)
Baseline
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
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.
Secondary
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
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).
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.
Posted
Least Squares Mean
Standard Error
meters (m)
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
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.
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.
Posted
Number
percentage of participants
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
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.
Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.
Posted
Mean
Standard Deviation
m/sec
Baseline, Week 48
ID
Title
Description
OG000
Placebo
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.
OG001
RO7239361 Low Dose
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.
OG002
RO7239361 High Dose
Secondary
Number of Participants With Adverse Events (AEs)
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.
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.
Posted
Number
participants
During DB period (48 weeks) and Whole study (up to approximately 34 months)
ID
Title
Description
OG000
Placebo DB
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.
OG001
RO7239361 Low Dose DB
Participants received low dose RO7239361 SC on specified days of the 48-week DB period.
OG002
RO7239361 High Dose DB
Secondary
Number of Participants With AEs Leading to Discontinuation
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.
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.
Posted
Number
participants
During DB period (48 weeks) and Whole study (up to approximately 34 months)
ID
Title
Description
OG000
Placebo DB
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.
OG001
RO7239361 Low Dose DB
Participants received low dose RO7239361 SC on specified days of the 48-week DB period.
OG002
Time Frame
Up to approximately 34 months
Description
Safety population included all enrolled participants who received at least 1 dose of study therapy.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Placebo DB
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.
0
56
3
56
43
56
EG001
RO7239361 Low Dose DB
Participants received low dose RO7239361 SC on specified days of the 48-week DB period.
0
55
2
55
43
55
EG002
RO7239361 High Dose DB
Participants received high dose RO7239361 SC on specified days of the 48-week DB period.
1
55
4
55
47
55
EG003
Placebo, Then RO7239361 Low Dose OL
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.
0
14
0
14
8
14
EG004
Placebo, Then RO7239361 High Dose OL
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.
0
13
0
13
7
13
EG005
RO7239361 Low Dose, Then RO7239361 Low Dose OL
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.
0
24
0
24
13
24
EG006
RO7239361 High Dose, Then RO7239361 High Dose OL
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
0
29
0
29
12
29
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Cardiac arrest
Cardiac disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0021 events1 affected55 at risk
EG0030 events0 affected14 at risk
EG0040 events0 affected13 at risk
EG0050 events0 affected24 at risk
EG0060 events0 affected29 at risk
Myocarditis
Cardiac disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Supraventricular tachycardia
Cardiac disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Influenza like illness
General disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Hyperbilirubinaemia
Hepatobiliary disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Gastrointestinal viral infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Viral infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0011 events1 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Humerus fracture
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0011 events1 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Overdose
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Hypocalcaemia
Metabolism and nutrition disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Ear pain
Ear and labyrinth disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0011 events1 affected55 at risk
EG0027 events4 affected55 at risk
EG0030 events0 affected14 at risk
EG0040 events0 affected13 at risk
EG0050 events0 affected24 at risk
EG0060 events0 affected29 at risk
Abdominal discomfort
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0002 events1 affected56 at risk
EG0011 events1 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0012 events2 affected55 at risk
EG0025 events3 affected55 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0004 events3 affected56 at risk
EG0015 events4 affected55 at risk
EG00212 events7 affected55 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0005 events4 affected56 at risk
EG0014 events4 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0003 events3 affected56 at risk
EG00113 events10 affected55 at risk
EG0028 events4 affected55 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0011 events1 affected55 at risk
EG0024 events3 affected55 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0012 events2 affected55 at risk
EG0029 events5 affected55 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 23.0
Systematic Assessment
EG0006 events6 affected56 at risk
EG00114 events8 affected55 at risk
EG00211 events6 affected55 at risk
EG003
Fatigue
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0014 events4 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Gait inability
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Injection site bruising
General disorders
MedDRA 23.0
Systematic Assessment
EG0003 events2 affected56 at risk
EG0014 events4 affected55 at risk
EG0024 events4 affected55 at risk
EG003
Injection site erythema
General disorders
MedDRA 23.0
Systematic Assessment
EG00025 events8 affected56 at risk
EG00143 events11 affected55 at risk
EG00238 events12 affected55 at risk
EG003
Injection site induration
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Injection site oedema
General disorders
MedDRA 23.0
Systematic Assessment
EG0007 events3 affected56 at risk
EG0019 events2 affected55 at risk
EG0029 events1 affected55 at risk
EG003
Injection site pruritus
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0012 events2 affected55 at risk
EG0026 events2 affected55 at risk
EG003
Injection site reaction
General disorders
MedDRA 23.0
Systematic Assessment
EG0003 events2 affected56 at risk
EG00129 events4 affected55 at risk
EG00233 events2 affected55 at risk
EG003
Injection site swelling
General disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG00131 events4 affected55 at risk
EG0025 events3 affected55 at risk
EG003
Localised oedema
General disorders
MedDRA 23.0
Systematic Assessment
EG0005 events3 affected56 at risk
EG0010 events0 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Pyrexia
General disorders
MedDRA 23.0
Systematic Assessment
EG0008 events8 affected56 at risk
EG00113 events9 affected55 at risk
EG0029 events8 affected55 at risk
EG003
Ear infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0014 events4 affected55 at risk
EG0022 events1 affected55 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0002 events1 affected56 at risk
EG0011 events1 affected55 at risk
EG0025 events3 affected55 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0011 events1 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Hordeolum
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0012 events2 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Influenza
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0003 events3 affected56 at risk
EG0016 events6 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG00017 events13 affected56 at risk
EG00117 events13 affected55 at risk
EG00216 events13 affected55 at risk
EG003
Pharyngitis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0002 events2 affected56 at risk
EG0012 events2 affected55 at risk
EG0023 events3 affected55 at risk
EG003
Rhinitis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0015 events3 affected55 at risk
EG0027 events4 affected55 at risk
EG003
Sinusitis
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0010 events0 affected55 at risk
EG0024 events3 affected55 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA 23.0
Systematic Assessment
EG0008 events6 affected56 at risk
EG0015 events4 affected55 at risk
EG00216 events7 affected55 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0002 events2 affected56 at risk
EG0017 events5 affected55 at risk
EG0025 events4 affected55 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG00011 events5 affected56 at risk
EG0011 events1 affected55 at risk
EG0026 events5 affected55 at risk
EG003
Gadolinium deposition disease
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Ligament sprain
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0004 events4 affected56 at risk
EG0012 events1 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Skin abrasion
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0003 events2 affected56 at risk
EG00112 events3 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Thermal burn
Injury, poisoning and procedural complications
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Bone density decreased
Investigations
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Glutamate dehydrogenase increased
Investigations
MedDRA 23.0
Systematic Assessment
EG0002 events2 affected56 at risk
EG0011 events1 affected55 at risk
EG0023 events3 affected55 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0006 events6 affected56 at risk
EG0018 events7 affected55 at risk
EG0026 events5 affected55 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0004 events4 affected56 at risk
EG0019 events7 affected55 at risk
EG0022 events2 affected55 at risk
EG003
Mobility decreased
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Muscle spasms
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0017 events2 affected55 at risk
EG0026 events2 affected55 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 23.0
Systematic Assessment
EG0003 events2 affected56 at risk
EG0016 events4 affected55 at risk
EG00211 events8 affected55 at risk
EG003
Headache
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG00028 events9 affected56 at risk
EG00160 events14 affected55 at risk
EG00242 events10 affected55 at risk
EG003
Migraine
Nervous system disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG00110 events1 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG00010 events10 affected56 at risk
EG00111 events8 affected55 at risk
EG00213 events7 affected55 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0009 events3 affected56 at risk
EG00115 events3 affected55 at risk
EG00217 events6 affected55 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0013 events3 affected55 at risk
EG0023 events3 affected55 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0013 events3 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 23.0
Systematic Assessment
EG0001 events1 affected56 at risk
EG0017 events4 affected55 at risk
EG0023 events3 affected55 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0004 events3 affected56 at risk
EG0012 events2 affected55 at risk
EG0021 events1 affected55 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0009 events4 affected56 at risk
EG0015 events4 affected55 at risk
EG0029 events7 affected55 at risk
EG003
Rash macular
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0000 events0 affected56 at risk
EG0010 events0 affected55 at risk
EG0020 events0 affected55 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA 23.0
Systematic Assessment
EG0004 events3 affected56 at risk
EG0012 events2 affected55 at risk
EG0024 events3 affected55 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
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.
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Browse Leaves
Not provided
Browse Branches
Not provided
0 subjects
FG0011 subjects
FG0020 subjects
Withdrawal by Subject
FG0000 subjects
FG0013 subjects
FG0021 subjects
8.5
± 1.7
55
BG003166
0
BG0030
Male
BG00056
BG00155
BG00255
BG003166
6
BG00318
Not Hispanic or Latino
BG00043
BG00141
BG00243
BG003127
Unknown or Not Reported
BG0006
BG0019
BG0026
BG00321
7
BG00323
Native Hawaiian or other Pacific Islander
Title
Measurements
BG0000
BG0010
BG0021
BG0031
White
Title
Measurements
BG00042
BG00145
BG00245
BG003132
Other
Title
Measurements
BG0005
BG0013
BG0022
BG00310
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.
OG002
RO7239361 High Dose
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-2.99± 0.65
OG001-3.44± 0.67
OG002-2.41± 0.64
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
-0.45
Standard Error of the Mean
0.87
2-Sided
95
-2.17
1.27
Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age Interactive response system (IXRS) Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
0.58
Standard Error of the Mean
0.85
2-Sided
95
-1.10
2.26
Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG0003.81± 1.55
OG0013.85± 1.61
OG0023.92± 1.91
RO7239361 High Dose
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-0.15± 0.07
OG001-0.15± 0.07
OG002-0.07± 0.07
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
-0.00
Standard Error of the Mean
0.10
2-Sided
95
-0.21
0.20
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
0.08
Standard Error of the Mean
0.10
2-Sided
95
-0.12
0.27
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG0006.28± 4.75
OG0016.15± 4.07
OG0027.24± 9.22
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-0.05± 0.01
OG001-0.02± 0.01
OG002-0.02± 0.01
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
0.03
Standard Error of the Mean
0.02
2-Sided
95
-0.00
0.06
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
0.03
Standard Error of the Mean
0.02
2-Sided
95
-0.00
0.06
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG0005.38± 1.48
OG0015.51± 1.68
OG0025.68± 2.30
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-0.23± 0.06
OG001-0.14± 0.07
OG002-0.23± 0.06
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
0.09
Standard Error of the Mean
0.09
2-Sided
95
-0.08
0.27
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
0.00
Standard Error of the Mean
0.09
2-Sided
95
-0.17
0.18
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG00085.59± 10.21
OG00186.54± 9.52
OG00284.47± 14.76
OG002
RO7239361 High Dose
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-5.47± 1.79
OG001-7.47± 1.83
OG002-4.51± 1.77
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
-2.00
Standard Error of the Mean
2.41
2-Sided
95
-6.76
2.77
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
0.96
Standard Error of the Mean
2.36
2-Sided
95
-3.71
5.63
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Baseline: Knee Extenders
ParticipantsOG00056
ParticipantsOG00155
ParticipantsOG00254
Title
Measurements
OG0005.58± 2.87
OG0016.25± 3.63
OG0025.76± 3.53
Change from Baseline at Week 48: Knee Extenders
ParticipantsOG00030
ParticipantsOG00128
ParticipantsOG00233
Title
Measurements
OG000
Baseline: Knee Flexors
ParticipantsOG00056
ParticipantsOG00155
ParticipantsOG00254
Title
Measurements
OG000
Change from Baseline at Week 48: Knee Flexors
ParticipantsOG00030
ParticipantsOG00128
ParticipantsOG00233
Title
Measurements
OG000
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000388.33± 69.59
OG001399.73± 68.35
OG002370.73± 93.35
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
Title
Denominators
Categories
Title
Measurements
OG000-41.3± 8.7
OG001-39.6± 9.0
OG002-30.0± 8.7
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean Difference (Final Values)
1.7
Standard Error of the Mean
11.5
2-Sided
95
-21.1
24.6
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
OG000
OG002
Mean Difference (Final Values)
11.3
Standard Error of the Mean
11.3
2-Sided
95
-11.0
33.6
Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.
Superiority
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.
Units
Counts
Participants
OG00036
OG00137
OG00231
Title
Denominators
Categories
Very much improved
Title
Measurements
OG0000
OG0010
OG0020
Much improved
Title
Measurements
OG0005.6
OG0012.7
OG0023.2
Minimally improved
Title
Measurements
OG00013.9
OG00113.5
OG00219.4
No change
Title
Measurements
OG00058.3
OG00154.1
OG00251.6
Minimally worse
Title
Measurements
OG00016.7
OG00118.9
OG00222.6
Much worse
Title
Measurements
OG0005.6
OG00110.8
OG0023.2
Very much worse
Title
Measurements
OG0000
OG0010
OG0020
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.
Units
Counts
Participants
OG00017
OG00119
OG00215
Title
Denominators
Categories
Baseline
ParticipantsOG00017
ParticipantsOG00119
ParticipantsOG00215
Title
Measurements
OG0001.69± 0.33
OG0011.54± 0.35
OG0021.57± 0.46
Change from Baseline at Week 48
ParticipantsOG0005
ParticipantsOG0017
ParticipantsOG0024
Title
Measurements
OG000
Participants received high dose RO7239361 SC on specified days of the 48-week DB period.
OG003
RO7239361 Low Dose
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.
OG004
RO7239361 High Dose Whole Study
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.
Units
Counts
Participants
OG00056
OG00155
OG00255
OG00369
OG00468
Title
Denominators
Categories
Double-Blind Period
ParticipantsOG00056
ParticipantsOG00155
ParticipantsOG00255
ParticipantsOG0030
ParticipantsOG0040
Title
Measurements
OG00046
OG00148
OG00249
Whole Study
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0020
ParticipantsOG00369
RO7239361 High Dose DB
Participants received high dose RO7239361 SC on specified days of the 48-week DB period.
OG003
RO7239361 Low Dose
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.
OG004
RO7239361 High Dose
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.