A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy

Bristol-Myers Squibb International Corporation0 sites160 target enrollmentJuly 4, 2017

ConditionsDuchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Duchenne Muscular Dystrophy
Sponsor: Bristol-Myers Squibb International Corporation
Enrollment: 160
Status: Active, not recruiting
Last Updated: 6 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

July 4, 2017

End Date

TBD

Last Updated

6 years ago

Study Type

Interventional clinical trial of medicinal product

Investigators

Sponsor

Bristol-Myers Squibb International Corporation

Eligibility Criteria

Inclusion Criteria

•Key Inclusion Criteria:
•Males, 6 \>\= to \< 12 years of age at time of randomization.
•Diagnosis of DMD, confirmed by medical history (eg., onset of clinical signs or symptoms before 5 years of age together with an elevated serum creatine kinase level observed before or after initial diagnosis) and by genotyping.
•Participants \>\= 15 kg.
•Ambulatory without assistance.
•Participants must be receiving corticosteroids (prednisone, prednisolone, or deflazacort) for at least 6 months prior to the start of study drug, with no significant change in dosage (\> 0\.2 mg/kg prednisone or \> 0\.24 mg/kg deflazacort) or dosing regimen for at least 3 months prior to the start of study drug, with the expectation that dosage and dosing regimen will not change significantly for the duration of the study.
•4SC\<\= 8 seconds at screening.
•Participants must agree to avoid major changes in their physical or respiratory therapy regimen during the double blind phase, to the extent possible.
•Males who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with study treatment(s) plus 5 half\-lives of the study treatment \[BMS\-986089; 50 days] plus 90 days (duration of sperm turnover) for a total of 140 days (5 months) post\-treatment completion.
•For the rest of Inclusion Criteria, please refer to study protocol Section 6\.1 (page 50\).

Exclusion Criteria

•Key Exclusion Criteria:
•Participants with cognitive impairment or behavioral issues that, in the judgement of the investigator, will compromise their ability to comply with study procedures.
•Participants on intermittent corticosteroid regimens with off periods of 20 days or longer (eg.: 10 days on, 20 days off).
•Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re\-initiation) in prophylaxis /treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment.
•Any change (initiation, change in drug class, dose modification unrelated to change in body weight, interruption or re\-initiation) in prophylaxis/treatment for bone density within 3 months prior to start of study treatment.
•Treatment with exon skipping therapies within 6 months prior to the start of study drug administration.
•Treatment with ataluren, or any other investigational drug (excluding deflazacort and exon skipping therapies) currently or within 3 months prior to the start of study drug administration.
•Participants with a FVC of \< 50% (in participants able to produce a valid FVC, as judged by the clinical evaluator or respiratory therapist).
•Cutaneous AEs sustained during participation in a prior clinical trial that resolved less than 3 months prior to the start of study drug administration.
•Current or prior treatment within 3 months prior to the start of study drug administration with androgens or human growth hormone.