A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Purpose

We are doing this research study to find out if RO7239361 (previously known as BMS-986089) can help people with Duchenne. We also want to find out if RO7239361 is safe to take and if it is tolerated well. RO7239361 is not approved by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), or similar government agencies abroad. This means that RO7239361 is an investigational (experimental) drug and can be used only in research studies.

Duchenne is caused by a mutation (a change) in the gene (the part of cells that contain the instructions which tell our bodies how to grow and work) that makes dystrophin (a protein). Dystrophin is important for protecting muscles from stress and damage during activity. If a person has Duchenne, his body is not able to make enough working dystrophin to protect his muscles and the muscle mass progressively decreases.

RO7239361 is an anti-myostatin agent able to block myostatin, a protein that is involved in the regulation of muscle mass. Myostatin has a negative effect on muscle growth, and muscle mass may increase by blocking myostatin.

Procedures

There are four parts to this study.

Part 1: The first part of the study will be the screening period, when the study team will evaluate your child and determine if he is eligible to participate. This part will last up to 42 days.

Part 2: The second part of the study will be the 48-week double blind period, which means that neither you nor your child nor your study doctor will know if your child is receiving the investigational drug or a placebo treatment (the placebo looks like the study drug but does not contain any drug).

Part 3: The third part of this study will be the open label period, during which all children will receive the RO7239361 investigational drug. The Open Label Phase will last up to 192 weeks (approximately 4 years), or until the study drug is commercially available in your country (as per local regulations), or until the study is terminated, whichever comes first. During the Open Label Phase, your child will receive one of the two doses being tested in this study, and you will be informed which dose but will not be informed what treatment (RO7239361 or the placebo) your child was originally assigned in the Double Blind Phase.

Part 4: The fourth part of the study will be a follow-up period of 24 weeks, which will start when the treatment is stopped (at the end of the Open Label Phase or earlier in the case of early discontinuation).

Eligibility

  • Male
  • 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

Additional inclusion and exclusion criteria apply. Please Gee Kim for more information:

Age

18 and under

Gender

Male

Monetary compensation

Yes

Can be done from home

No

Keywords

Muscular dystrophy, Muscular dystrophy - resources, Neurology

Principal Investigator

Barry J. Byrne, MD, PhD

Department

Neurology

Contact Information

geekim@ufl.edu

(352) 294-8283


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