Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 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-NL
• Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-07-12
• Study Completion: 2020-04-28
• Last Update Posted: 9 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 166

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 (CS, 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) or dosing regimen for at least 12 weeks 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.
• North Star Ambulatory Assessment (NSAA) score >=15 points at
screening
• 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.
• Subjects 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 [RO7239361; 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: 159
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 CS 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 12 weeks 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 12 weeks 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 currently or within 12 weeks prior to the start
of study drug administration
• Treatment with any other investigational drug (excluding deflazacort
in CS dose-finding trials) currently or within 12 weeks prior to the start
of study drug administration.
•Concurrent or previous participation at any time in a gene therapy study.
• Participants with a FVC of < 50% of predicted value (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 12 weeks prior to the start of study drug administration.
• Current or prior treatment within 12 weeks prior to the start of study drug administration with androgens or human growth hormone.
• Prior treatment with RO7239361 or any other anti-myostatin agent.
• History of lower limb fracture within 12 weeks prior to the start of study drug administration.
• History of upper limb fracture within 8 weeks prior to the start of study drug administration.
• Any injury that 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 4 weeks 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.
• Clinical signs or symptoms of uncontrolled congestive heart failure (CHF)(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).
• Unwilling or unable to administer study drug at home.

Study & Design

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