Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT03039686 |
Recruitment Status :
Recruiting
First Posted : February 1, 2017
Last Update Posted : November 16, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: RO7239361 Drug: Placebo for RO7239361 | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 159 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | July 6, 2017 |
Estimated Primary Completion Date : | December 30, 2020 |
Estimated Study Completion Date : | December 17, 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: RO7239361, dose 1
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
|
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period |
Experimental: RO7239361, dose 2
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
|
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period |
Placebo Comparator: Placebo
Placebo solution taken subcutaneously on specified days over a 48 week blinded period
|
Drug: Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period |
- Change from baseline in the North Star Ambulatory Assessment (NSAA) total score in RO7239361 treated participants. [ Time Frame: 48 Week ]
- Change from baseline in the in the North Star Ambulatory Assessment (NSAA) total score in placebo treated participants. [ Time Frame: 48 Week ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in the 4 Stair Climb Velocity (4SCV) Assessment [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Stand from supine velocity [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 10 M walk/run velocity [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in PODCI transfers and basic mobility subscale [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Proximal lower extremity flexor (knee extension and knee flexion) strength, measured using manual myometry [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 6 Minute Walk Distance (6MWD) [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Clinical Global Impression of Change (CGI-C) rating. [ Time Frame: 48 weeks ]
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 95th percentile stride velocity [ Time Frame: 48 weeks ]As recorded with the ActiMyo device in a subset of the overall study population.

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Ages Eligible for Study: | 6 Years to 11 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03039686
Contact: Reference Study ID Number: WN40227 www.roche.com/about_roche/roche_worldwide.htm | 888-662-6728 (U.S. and Canada) | global-roche-genentech-trials@gene.com |

United States, Arizona | |
Neuromuscular Research Center | Recruiting |
Phoenix, Arizona, United States, 85028 | |
United States, Arkansas | |
Arkansas Children's Hospital; Pediatrics | Not yet recruiting |
Little Rock, Arkansas, United States, 72202-3591 | |
United States, California | |
David Geffen School of Medicine at UCLA; Clinical Trials Contract Unit | Recruiting |
Los Angeles, California, United States, 90095 | |
Stanford University | Recruiting |
Palo Alto, California, United States, 94304 | |
University of California Davis Medical Center | Recruiting |
Sacramento, California, United States, 95817 | |
United States, Connecticut | |
Yale University School of Medicine ; Pulmonary & Critical Care | Recruiting |
New Haven, Connecticut, United States, 06510 | |
United States, Florida | |
University of Florida | Recruiting |
Gainesville, Florida, United States, 32607 | |
Nemours Children's Hospital | Recruiting |
Orlando, Florida, United States, 32827 | |
United States, Georgia | |
Rare Disease Research, LLC | Recruiting |
Atlanta, Georgia, United States, 30318 | |
United States, Illinois | |
Rush University Medical Center - PPDS | Recruiting |
Chicago, Illinois, United States, 60612 | |
United States, Iowa | |
University of Iowa | Recruiting |
Iowa City, Iowa, United States, 52242 | |
United States, Kansas | |
University of Kansas Medical Center | Recruiting |
Kansas City, Kansas, United States, 66160 | |
United States, Maryland | |
Kennedy Krieger Institute | Recruiting |
Baltimore, Maryland, United States, 21205 | |
United States, Massachusetts | |
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology | Not yet recruiting |
Worcester, Massachusetts, United States, 01655 | |
United States, Missouri | |
Saint Louis Children's Hospital | Recruiting |
Saint Louis, Missouri, United States, 63110 | |
United States, Nevada | |
Las Vegas Clinic | Recruiting |
Las Vegas, Nevada, United States, 89145 | |
United States, Ohio | |
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital | Recruiting |
Columbus, Ohio, United States, 43205 | |
United States, Pennsylvania | |
Children'S Hospital of Philadelphia | Not yet recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Washington | |
Seattle Children's Hospital | Recruiting |
Seattle, Washington, United States, 98105 | |
Argentina | |
Instituto centenario | Recruiting |
Buenos Aires, Argentina, C1204AAD | |
Australia, New South Wales | |
Children's Hospital Westmead; Paediatrics & Child Health | Recruiting |
Westmead, New South Wales, Australia, 2145 | |
Australia, Queensland | |
Lady Cilento Children's Hospital; Neurosciences Department | Recruiting |
South Brisbane, Queensland, Australia, 4101 | |
Australia, Victoria | |
Royal Children's Hospital | Recruiting |
Parkville, Victoria, Australia, 3052 | |
Belgium | |
UZ Gent | Recruiting |
Gent, Belgium, 9000 | |
UZ Leuven; Neurology | Recruiting |
Leuven, Belgium, 3000 | |
Canada, Alberta | |
Alberta Children's Hospital | Recruiting |
Calgary, Alberta, Canada, T3B 6A8 | |
Canada, Ontario | |
McMaster University Health Sciences Center | Withdrawn |
Hamilton, Ontario, Canada, L8N 3Z5 | |
London Health Sciences Centre; Children's Hospital; Pediatrics | Recruiting |
London, Ontario, Canada, N6A 5W9 | |
Children'S Hospital of Eastern Ontario | Recruiting |
Ottawa, Ontario, Canada, K1H 8L1 | |
France | |
Hospices Civils de Lyon | Recruiting |
Lyon, France, 69004 | |
Hotel Dieu; Service Pharmacie Essais Cliniques | Recruiting |
Nantes, France, 44093 | |
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie | Recruiting |
Paris Cedex 12, France, 75571 | |
Hopitaux Universitaires de Strasbourg | Recruiting |
Strasbourg, France, 67091 | |
Germany | |
Universitatsklinikum Essen; Innere Klinik | Recruiting |
Essen, Germany, 45122 | |
Italy | |
Fondazione Serena Onlus - CENTRO CLINICO NEMO | Recruiting |
Milano, Emilia-Romagna, Italy, 20162 | |
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia | Recruiting |
Rome, Lazio, Italy, 00168 | |
Istituto G Gaslini Ospedale Pediatrico IRCCS | Not yet recruiting |
Genova, Liguria, Italy, 16147 | |
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta | Not yet recruiting |
Milano, Lombardia, Italy, 20133 | |
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest. | Recruiting |
Messina, Sicilia, Italy, 98125 | |
Japan | |
Hyogo College of Medicine Hospital | Recruiting |
Hyogo, Japan, 663-8501 | |
Miyagi Children's Hospital | Recruiting |
Miyagi, Japan, 989-3126 | |
Shinshu University Hospital | Recruiting |
Nagano, Japan, 390-8621 | |
National Hospital Organization Toneyama National Hospital | Recruiting |
Osaka, Japan, 560-8552 | |
National Center of Neurology and Psychiatry | Recruiting |
Tokyo, Japan, 187-8551 | |
Netherlands | |
Leids Universitair Medisch Centrum | Recruiting |
Leiden, Netherlands, 2300 RC | |
Radboud University Nijmegen Medical Centre; Ophthalmology | Not yet recruiting |
Nijmegen, Netherlands, 6525 EX | |
Spain | |
Hospital Sant Joan De Deu | Recruiting |
Esplugues De Llobregas, Barcelona, Spain, 08950 | |
Hospital Universitario Vall d'Hebron - PPDS | Not yet recruiting |
Barcelona, Spain, 08035 | |
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia | Not yet recruiting |
Valencia, Spain, 46026 | |
Sweden | |
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin | Recruiting |
Goeteborg, Sweden, 41685 | |
United Kingdom | |
Alder Hey Children s Hospital; Department of Pediatrics | Recruiting |
Liverpool, United Kingdom, L12 2AP | |
UCL Institute of Child Health & Great Ormond Street Hospital for Children | Recruiting |
London, United Kingdom, WC1N 1EH |
Study Director: | Clinical Trials | Hoffmann-La Roche |
Additional Information:


Responsible Party: | Hoffmann-La Roche |
ClinicalTrials.gov Identifier: | NCT03039686 History of Changes |
Other Study ID Numbers: |
CN001-016 2016-001654-18 ( EudraCT Number ) WN40227 ( Other Identifier: Hoffman-La Roche ) |
First Posted: | February 1, 2017 Key Record Dates |
Last Update Posted: | November 16, 2018 |
Last Verified: | November 2018 |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Hoffmann-La Roche:
muscular dystrophy Duchenne's Muscular Dystrophy DMD |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |