Roche- BMS-986089

Clinical trial to Evaluate the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy

Hub Summary

This placebo-controlled study is designed to assess the efficacy, safety and tolerability of two different doses of BMS-986089 in ambulatory males with DMD. BMS-986089 is a subcutaneously delivered treatment which inhibits myostatin.

Myostatin is a protein in the body which inhibits muscle growth and it is required to stop muscles from growing too large. Myostatin production increases naturally with age. It is thought that inhibiting myostatin function could lead to increased muscle size and strength in patients with DMD.

Study Number: NCT03039686

Description by Hoffmann-La Roche

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of BMS-986089 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

 
Primary Outcome Measures

  • Change from baseline in the 4 stair climb velocity on BMS-986089 treated participants,
  • Change from baseline in the 4 stair climb velocity in placebo treated participants.

 
Can I take part?

 
Inclusion Criteria

  • Diagnosed with DMD by a blood test
  • Able to walk without assistance
  • 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 behaviour 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

Other protocol defined Inclusion/Exclusion Criteria could apply.

 
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Trial Status
Recruiting
Trial Sponsor
Hoffmann-La Roche
Age
6-11
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
MRI
No
Phase
2/3
Length Of Participation
48 weeks
Recruitment Target
159
Ambulatory
Ambulant
Therapeutic Category
Myostatin inhibition