- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03039686
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
November 25, 2020 updated by: Hoffmann-La Roche
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
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).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
166
Phase
- Phase 2
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Buenos Aires, Argentina, C1204AAD
- Instituto Centenario
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New South Wales
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Westmead, New South Wales, Australia, 2145
- Children's Hospital Westmead; Paediatrics & Child Health
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Queensland
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South Brisbane, Queensland, Australia, 4101
- Lady Cilento Children's Hospital; Neurosciences Department
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Victoria
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Parkville, Victoria, Australia, 3052
- Royal Children's Hospital
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Gent, Belgium, 9000
- UZ Gent
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Ontario
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London, Ontario, Canada, N6A 5W9
- London Health Sciences Centre; Children's Hospital; Pediatrics
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Ottawa, Ontario, Canada, K1H 8L1
- Children's Hospital of Eastern Ontario
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Lyon, France, 69004
- Hospices Civils de Lyon
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Nantes, France, 44093
- Hotel Dieu; Service Pharmacie Essais Cliniques
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Paris Cedex 12, France, 75571
- Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
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Strasbourg, France, 67091
- Hopitaux Universitaires de Strasbourg
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Essen, Germany, 45122
- Universitatsklinikum Essen; Innere Klinik
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Emilia-Romagna
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Milano, Emilia-Romagna, Italy, 20162
- Fondazione Serena Onlus - Centro Clinico Nemo
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Lazio
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Rome, Lazio, Italy, 00168
- Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
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Sicilia
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Messina, Sicilia, Italy, 98125
- Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
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Hyogo, Japan, 663-8501
- Hyogo College of Medicine Hospital
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Miyagi, Japan, 989-3126
- Miyagi Children's Hospital
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Nagano, Japan, 390-8621
- Shinshu University Hospital
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Osaka, Japan, 560-8552
- National Hospital Organization Osaka Toneyama Medical Center
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Tokyo, Japan, 187-8551
- National Center of Neurology and Psychiatry
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Leiden, Netherlands, 2333 ZA
- Leids Universitair Medisch Centrum
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Nijmegen, Netherlands, 6525 EX
- Radboud University Nijmegen Medical Centre; Ophthalmology
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Valencia, Spain, 46026
- Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
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Barcelona
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Esplugues De Llobregas, Barcelona, Spain, 08950
- Hospital Sant Joan de Déu
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Goeteborg, Sweden, 41685
- Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin
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Liverpool, United Kingdom, L12 2AP
- Alder Hey Children s Hospital; Department of Pediatrics
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London, United Kingdom, WC1N 1EH
- UCL Institute of Child Health & Great Ormond Street Hospital for Children
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Arizona
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Phoenix, Arizona, United States, 85028
- Neuromuscular Research Center
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California
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Palo Alto, California, United States, 94304
- Stanford University
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Sacramento, California, United States, 95817
- University of California Davis Medical Center
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Connecticut
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New Haven, Connecticut, United States, 06510
- Yale University School of Medicine ; Pulmonary & Critical Care
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Florida
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Gainesville, Florida, United States, 32607
- University of Florida
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Orlando, Florida, United States, 32827
- Nemours Children's Hospital
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Georgia
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Atlanta, Georgia, United States, 30318
- Rare Disease Research, LLC
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Illinois
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Chicago, Illinois, United States, 60612
- Rush University Medical Center - PPDS
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa
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Kansas
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Kansas City, Kansas, United States, 66160
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, United States, 21205
- Kennedy Krieger Institute
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Massachusetts
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Worcester, Massachusetts, United States, 01655
- University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
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Missouri
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Saint Louis, Missouri, United States, 63110
- Saint Louis Children's Hospital
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Nevada
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Las Vegas, Nevada, United States, 89145
- Las Vegas Clinic
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
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Columbus, Ohio, United States, 43205
- Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years to 11 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: 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.
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Take RO7239361 subcutaneously on specified days over a 48 week blinded period
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Experimental: 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.
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Take RO7239361 subcutaneously on specified days over a 48 week blinded period
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Placebo Comparator: 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.
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Take placebo subcutaneously on specified days over a 48 week blinded period
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
Time Frame: Baseline
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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.
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Baseline
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Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Baseline Time for 4 Stair Climb
Time Frame: Baseline
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The time to complete the 4 stair climb was measured at baseline.
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Baseline
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Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Baseline for the Time to Stand From Supine
Time Frame: Baseline
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The time required for a participant to stand from supine position.
A longer time reflects a worse outcome.
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Baseline
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Change From Baseline at Week 48 in Stand From Supine Velocity
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Baseline Time for 10 Meter Walk/Run
Time Frame: Baseline
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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.
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Baseline
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Change From Baseline at Week 48 in 10 M Walk/Run Velocity
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time Frame: Baseline
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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.
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Baseline
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Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Time Frame: Baseline, Week 48
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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.
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Baseline, Week 48
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Baseline for the 6 Minute Walk Distance (6MWD)
Time Frame: Baseline
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The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes.
A longer distance reflects a better outcome.
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Baseline
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Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
Time Frame: Baseline, Week 48
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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).
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Baseline, Week 48
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Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
Time Frame: Baseline, Week 48
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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.
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Baseline, Week 48
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Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Time Frame: Baseline, Week 48
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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.
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Baseline, Week 48
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Number of Participants With Adverse Events (AEs)
Time Frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
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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.
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During DB period (48 weeks) and Whole study (up to approximately 34 months)
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Number of Participants With AEs Leading to Discontinuation
Time Frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
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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.
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During DB period (48 weeks) and Whole study (up to approximately 34 months)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 6, 2017
Primary Completion (Actual)
April 28, 2020
Study Completion (Actual)
April 28, 2020
Study Registration Dates
First Submitted
January 27, 2017
First Submitted That Met QC Criteria
January 31, 2017
First Posted (Estimate)
February 1, 2017
Study Record Updates
Last Update Posted (Actual)
December 21, 2020
Last Update Submitted That Met QC Criteria
November 25, 2020
Last Verified
November 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CN001-016
- 2016-001654-18 (EudraCT Number)
- WN40227 (Other Identifier: Hoffman-La Roche)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Clinical Trials on RO7239361
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