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

Phase 1

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2016-001654-18-FR
• Lead Sponsor: Bristol-Myers Squibb International Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-10-30
• Last Update Posted: 27 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 160

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).
Are the trial subjects under 18? yes
Number of subjects for this age range: 160
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.
• Prior treatment with BMS-986089 or any other anti-myostatin agent.
• History of lower limb fracture within 3 months prior to the start of study drug administration.
• History of upper limb fracture within 2 months prior to the start of study drug administration.
• Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting.
• Expectation of major surgical procedure, such as scoliosis surgery, during the double blind phase of this study.
• Requirement of daytime ventilator assistance.
• Initiation of nighttime ventilation less than 1 month prior to the start of study drug administration.
• Expectation that daytime or nighttime ventilation may be initiated during the double blind phase of this study.
• Uncontrolled clinical signs or symptoms of congestive heart failure (American College of Cardiology/American Heart Associated Stage C or Stage D).
• For participants participating in cMRI substudy: implanted ferromagnetic metal (implanted metal that is not ferromagnetic, such as surgical steel or titanium implants may be allowed if the implants will not compromise the quality of the cMRI).
• History of hypersensitivity to components of the study drug (histidine, trehalose, diethylenetriaminepentaacetic acid, polysorbate 80).
• Unwilling or unable to administer study drug at home.
For the rest of Exclusion Criteria, please refer to study protocol Section 6.2 (page 51).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified