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

Penematsa Vinay0 个研究点目标入组 25 人开始时间: 2020年12月25日最近更新: 2023年10月17日

研究概览

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•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.

发起方

Penematsa Vinay