Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

• Status: Active, not recruiting
• Trial end date: 2023-10-05
• Target enrollment: 0
• Participant gender: Male

Summary

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PTC Therapeutics

Criteria

Inclusion Criteria:

• Male sex

• Age ≥5 years

• Phenotypic evidence of Duchenne Muscular Dystrophy

• Nonsense point mutation in the dystrophin gene

• Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment

• 6MWD ≥150 meters

• Ability to perform timed function tests within 30 seconds

• Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

• Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.

• Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.

• Prior or ongoing therapy with ataluren.

• Known hypersensitivity to any of the ingredients or excipients of the study drug

• Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.

• History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.

• Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.

• Uncontrolled clinical symptoms and signs of congestive heart failure

• Elevated serum creatinine or cystatin C at screening.