Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD | DuchenneXchange

welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
active, not recruiting

Study of an Investigational Drug, RO7239361 (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 RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

intervention: RO7239361, Placebo

mechanism of action: Anti-myostatin adnectin to promote muscle growth

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

last updated: January 08, 2019

study details

start date: December 2, 2015

estimated completion: June 29, 2022

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 ]
  • 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, Hoffmann-La Roche

locations: United States, Canada