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

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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).

Key facts

Study ID

NCT03039686

Run by

Hoffmann-La Roche

People needed

166

Starts

2017-07-06

Expected to finish

2020-04-28

Last updated by the study team

2020-12-21

Who can join

Age: 6 and older, up to 11. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Neuromuscular Research Center — Phoenix, Arizona, United States
• Stanford University — Palo Alto, California, United States
• University of California Davis Medical Center — Sacramento, California, United States
• Yale University School of Medicine ; Pulmonary & Critical Care — New Haven, Connecticut, United States
• University of Florida — Gainesville, Florida, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• Rush University Medical Center - PPDS — Chicago, Illinois, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• University of Massachusetts Memorial Childrens Medical Center; Department of Neurology — Worcester, Massachusetts, United States
• Saint Louis Children's Hospital — St Louis, Missouri, United States
• Las Vegas Clinic — Las Vegas, Nevada, United States
• Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy — Cincinnati, Ohio, United States
• Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital — Columbus, Ohio, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Instituto centenario — Buenos Aires, Argentina
• Children's Hospital Westmead; Paediatrics & Child Health — Westmead, New South Wales, Australia
• Lady Cilento Children's Hospital; Neurosciences Department — South Brisbane, Queensland, Australia
• Royal Children's Hospital — Parkville, Victoria, Australia
• UZ Gent — Ghent, Belgium
• London Health Sciences Centre; Children's Hospital; Pediatrics — London, Ontario, Canada
• Children'S Hospital of Eastern Ontario — Ottawa, Ontario, Canada
• Hospices Civils de Lyon — Lyon, France

Full record on ClinicalTrials.gov

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