Study of an Investigational Drug, BMS-986089, in Ambulatory Boys With DMD | DuchenneXchange

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active, not recruiting

Study of an Investigational Drug, BMS-986089, in Ambulatory Boys With DMD

key information

study id #: NCT02515669

condition: Muscular Dystrophy (DMD)

status: active, not recruiting

purpose:

The purpose of this study is to determine the safety and tolerability of BMS-986089 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

intervention: BMS-986089, Placebo

mechanism of action: Myostatin blocker to promote muscular health

results: https://clinicaltrials.gov/ct2/show/results/NCT02515669

last updated: November 22, 2018

study details

start date: December 2, 2015

estimated completion: February 8, 2018

phase of development: Phase 1/Phase 2

size / enrollment: 43

primary outcomes:

  • Safety and Tolerability based on number of incidences of AEs, serious AEs, AEs leading to discontinuation and death, as well as marked treatment emergent abnormalities in clinical laboratory tests [Time Frame: 24 weeks]
    Adverse event (AEs)
  • Safety and Tolerability based on number of incidences of vital sign measurements, ECGs, echocardiograms, and physical examinations [ Time Frame: 24 weeks ]

secondary outcomes:

  • Trough concentrations of RO7239361 [Time Frame: At pre-defined intervals from day 8 through week 24]
  • Maximum observed concentration (Cmax) of RO7239361 [Time Frame: Day 1 and 29]
  • Frequency of subjects with positive anti-RO7239361 antibodies (ADA) assessment [Time Frame: Day 8 through Week 24]
  • Frequency of subjects who develop positive ADA following a negative baseline [Time Frame: Day 8 through Week 24]
  • Anti-RO7239361 antibodies on selected days [Time Frame: Day 8 through Week 24]
  • Serum concentration of free myostatin [Time Frame: Baseline through week 24]
  • Change from baseline in volume of thigh muscle contractile and non-contractile tissue [Time Frame: Day 8 through Week 24]
  • Change from baseline in thigh muscle maximal cross sectional area [Time Frame: Day 8 through Week 24]
  • Serum concentration of drug-myostatin complex [Time Frame: Baseline through week 24]
  • Serum concentration of percent inhibition of free myostatin at trough [Time Frame: At pre-defined intervals from day 8 through week 24]

inclusion criteria:
• Diagnosed with DMD
• Able to walk without assistance
• Able to walk up 4 stairs in 8 seconds or less
• Weigh at least 15 kg
• Taking corticosteroids for DMD

exclusion criteria:
• Ejection fraction < 55% on echocardiogram, based on central read
• 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

study contacts

sponsor: Hoffmann-La Roche

investigators: Clinical Trials

locations: United States, Canada