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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
RO7239361
Placebo for RO7239361
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring muscular dystrophy, Duchenne's Muscular Dystrophy, DMD

Eligibility Criteria

6 Years - 11 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Neuromuscular Research Center
  • Stanford University
  • University of California Davis Medical Center
  • Yale University School of Medicine ; Pulmonary & Critical Care
  • University of Florida
  • Nemours Children's Hospital
  • Rare Disease Research, LLC
  • Rush University Medical Center - PPDS
  • University of Iowa
  • University of Kansas Medical Center
  • Kennedy Krieger Institute
  • University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
  • Saint Louis Children's Hospital
  • Las Vegas Clinic
  • Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
  • Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
  • Seattle Children's Hospital
  • Instituto centenario
  • Children's Hospital Westmead; Paediatrics & Child Health
  • Lady Cilento Children's Hospital; Neurosciences Department
  • Royal Children's Hospital
  • UZ Gent
  • London Health Sciences Centre; Children's Hospital; Pediatrics
  • Children'S Hospital of Eastern Ontario
  • Hospices Civils de Lyon
  • Hotel Dieu; Service Pharmacie Essais Cliniques
  • Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
  • Hopitaux Universitaires de Strasbourg
  • Universitatsklinikum Essen; Innere Klinik
  • Fondazione Serena Onlus - CENTRO CLINICO NEMO
  • Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
  • Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
  • Hyogo College of Medicine Hospital
  • Miyagi Children's Hospital
  • Shinshu University Hospital
  • National Hospital Organization Osaka Toneyama Medical Center
  • National Center of Neurology and Psychiatry
  • Leids Universitair Medisch Centrum
  • Radboud University Nijmegen Medical Centre; Ophthalmology
  • Hospital Sant Joan De Deu
  • Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
  • Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
  • Alder Hey Children s Hospital; Department of Pediatrics
  • UCL Institute of Child Health & Great Ormond Street Hospital for Children

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

RO7239361 Low Dose

RO7239361 High Dose

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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.
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).

Secondary Outcome Measures

Baseline Time for 4 Stair Climb
The time to complete the 4 stair climb was measured at 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).
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.
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 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.
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 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.
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).
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 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.
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).
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.
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.
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.
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.

Full Information

First Posted
January 27, 2017
Last Updated
November 25, 2020
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT03039686
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Completed
Study Start Date
July 6, 2017 (Actual)
Primary Completion Date
April 28, 2020 (Actual)
Study Completion Date
April 28, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
muscular dystrophy, Duchenne's Muscular Dystrophy, DMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
166 (Actual)

8. Arms, Groups, and Interventions

Arm Title
RO7239361 Low Dose
Arm Type
Experimental
Arm Description
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.
Arm Title
RO7239361 High Dose
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm 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.
Intervention Type
Drug
Intervention Name(s)
RO7239361
Intervention Description
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Intervention Type
Drug
Intervention Name(s)
Placebo for RO7239361
Intervention Description
Take placebo subcutaneously on specified days over a 48 week blinded period
Primary Outcome Measure Information:
Title
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
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
Title
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
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
Secondary Outcome Measure Information:
Title
Baseline Time for 4 Stair Climb
Description
The time to complete the 4 stair climb was measured at baseline.
Time Frame
Baseline
Title
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
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
Title
Baseline for the Time to Stand From Supine
Description
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Time Frame
Baseline
Title
Change From Baseline at Week 48 in Stand From Supine Velocity
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
Title
Baseline Time for 10 Meter Walk/Run
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
Title
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
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
Title
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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
Title
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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
Title
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
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
Title
Baseline for the 6 Minute Walk Distance (6MWD)
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
Title
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
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
Title
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
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
Title
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
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
Title
Number of Participants With Adverse Events (AEs)
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)
Title
Number of Participants With AEs Leading to Discontinuation
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)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
Neuromuscular Research Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85028
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Yale University School of Medicine ; Pulmonary & Critical Care
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Nemours Children's Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Rush University Medical Center - PPDS
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
Saint Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Las Vegas Clinic
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89145
Country
United States
Facility Name
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Instituto centenario
City
Buenos Aires
ZIP/Postal Code
C1204AAD
Country
Argentina
Facility Name
Children's Hospital Westmead; Paediatrics & Child Health
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Lady Cilento Children's Hospital; Neurosciences Department
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Facility Name
Royal Children's Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
London Health Sciences Centre; Children's Hospital; Pediatrics
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Children'S Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
Hospices Civils de Lyon
City
Lyon
ZIP/Postal Code
69004
Country
France
Facility Name
Hotel Dieu; Service Pharmacie Essais Cliniques
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
City
Paris Cedex 12
ZIP/Postal Code
75571
Country
France
Facility Name
Hopitaux Universitaires de Strasbourg
City
Strasbourg
ZIP/Postal Code
67091
Country
France
Facility Name
Universitatsklinikum Essen; Innere Klinik
City
Essen
ZIP/Postal Code
45122
Country
Germany
Facility Name
Fondazione Serena Onlus - CENTRO CLINICO NEMO
City
Milano
State/Province
Emilia-Romagna
ZIP/Postal Code
20162
Country
Italy
Facility Name
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
City
Rome
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Facility Name
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
City
Messina
State/Province
Sicilia
ZIP/Postal Code
98125
Country
Italy
Facility Name
Hyogo College of Medicine Hospital
City
Hyogo
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
Miyagi Children's Hospital
City
Miyagi
ZIP/Postal Code
989-3126
Country
Japan
Facility Name
Shinshu University Hospital
City
Nagano
ZIP/Postal Code
390-8621
Country
Japan
Facility Name
National Hospital Organization Osaka Toneyama Medical Center
City
Osaka
ZIP/Postal Code
560-8552
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
Leids Universitair Medisch Centrum
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
Radboud University Nijmegen Medical Centre; Ophthalmology
City
Nijmegen
ZIP/Postal Code
6525 EX
Country
Netherlands
Facility Name
Hospital Sant Joan De Deu
City
Esplugues De Llobregas
State/Province
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
City
Goeteborg
ZIP/Postal Code
41685
Country
Sweden
Facility Name
Alder Hey Children s Hospital; Department of Pediatrics
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
UCL Institute of Child Health & Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom

12. IPD Sharing Statement

Links:
URL
http://www.fda.gov/Safety/Recalls/
Description
FDA Safety Alerts and Recalls
URL
http://roche-duchenne-clinicaltrials.com
Description
Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne

Learn more about this trial

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

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