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Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
study id #: NCT02515669
condition: Muscular Dystrophy (DMD)
status: active, not recruitingpurpose:
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
last updated: January 08, 2019
start date: December 2, 2015
estimated completion: June 29, 2022
phase of development: Phase 1/Phase 2
size / enrollment: 43
- 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 ]
- 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 ]
• 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
• 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
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