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An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
(PTC124-GD-049-DMD )

An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy
(PTC124-GD-049-DMD )

Penematsa Vinay

PTC Therapeutics, Inc.

500 Warren Corporate Center Drive Warren, NJ 07059, USA

medinfo@ptcbio.com

Cardona Ritsuko

Medpace Japan K.K.

1-5-8, Jingumae, Shibuya-ku, Tokyo

+81-3-4563-7000

RSJapan1@medpace.com

Not Recruiting

Jan. 06, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent.
Independent Ethics Committee (IEC) rules regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the subject should be followed.
2. Subjects diagnosed with nmDMD who completed study PTC124-GD-041-DMD and expressed interest in continuing to receive ataluren.
3. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 30-day follow-up period.
4. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.

1. Exposure to any investigational drug other than ataluren within 1 month prior to start of study treatment.
2. Eligibility for another ataluren clinical trial that is actively enrolling study participants.
3. Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate).
4. Ongoing IV aminoglycoside or IV vancomycin therapy.
5. Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator opinion, could adversely affect the safety of the subject or make it unlikely that follow up would be completed.

Male

Nonsense Mutation Duchenne Muscular Dystrophy

Subjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of
10 mg/kg, 10 mg/kg, and 20 mg/kg.

Criteria for evaluation:

Efficacy:
1. Determine the long-term effects of ataluren on upper limb muscle function strength as assessed by the Performance of Upper Limb (PUL) and by the DMD Upper Limb Patient-Reported Outcome Measure (PROM)
2. Determine the long-term effects of ataluren on pulmonary function as assessed by forced vital capacity (FVC)
3. Loss of ambulation (LoA), which will be defined as full time wheelchair dependency due to disease progression
4. Determine the long-term effect of ataluren on ambulation and endurance as assessed by the 6-minute walk test (6MWT)

Safety:
Subjects will be monitored for adverse events (AEs), laboratory abnormalities, vital signs, weight, electrocardiograms, echocardiograms, and physical examinations, during the course of the study. The latest version of the Common Terminology Criteria for Adverse Events will be used for grading the severity of AEs and laboratory abnormalities.

+81-52-851-5511

Dec. 07, 2020

No

China