Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 29 January 2024
• Main ID: NCT03179631
• Date of registration: 01/06/2017
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
• Scientific title: A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
• Date of first enrolment: July 6, 2017
• Target sample size: 360
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03179631
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Brazil, Bulgaria, Canada, Chile, China, Hong Kong, India, Japan, Jordan, Korea, Republic of, Malaysia, Mexico, Poland, Puerto Rico, Russian Federation, Sri Lanka, Taiwan, Thailand, Turkey, United States

Contacts

• Name: Vinay Penematsa, MD
• Affiliation: PTC Therapeutics, Inc.

Key inclusion & exclusion 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.



Age minimum: 5 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophies

Muscular Diseases

Neuromuscular Diseases

Musculoskeletal Disease

Nervous System Diseases

Muscular Disorders, Atrophic

Genetic Diseases, Inborn

Genetic Diseases, X-Linked

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: PLACEBO

Drug: Ataluren

Primary Outcome(s)

Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks [Time Frame: 72 weeks]

Secondary Outcome(s)

Change from Baseline to Week 72 in 6MWD [Time Frame: Baseline, Week 72]

Time to Loss of Stair-Climbing Over 72 Weeks [Time Frame: 72 Weeks]

Change from Baseline to Week 72 in Time to Descend 4 Stairs [Time Frame: Baseline, Week 72]

Time to Loss of Ambulation Over 72 Weeks [Time Frame: 72 weeks]

Time to Loss of Stair-Descending Over 72 Weeks [Time Frame: 72 weeks]

Change from Baseline to Week 72 in Time to Climb 4 Stairs [Time Frame: Baseline, Week 72]

Number of Treatment-Emergent Adverse Events Considered Related to Study Drug [Time Frame: 72 weeks]

Change from Baseline to Week 72 in Time to Run/Walk 10 Meters [Time Frame: Baseline, Week 72]

Risk of Loss of NSAA Items Over 72 weeks [Time Frame: 72 weels]

Change from Baseline to Week 72 in North Start Ambulatory Assessment (NSAA) Total Score [Time Frame: Baseline, Week 72]

Secondary ID(s)

PTC124-GD-041-DMD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors