An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Stopped early · Phase 3

Conditions studied: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn

In brief

The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study. This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.

Key facts

Study ID

NCT02090959

Run by

PTC Therapeutics

People needed

219

Starts

2014-03-20

Expected to finish

2018-06-12

Last updated by the study team

2020-08-11

Who can join

Age: 7 and older, up to 15. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Completion of study treatment in the previous Phase 3, double-blind study (PTC124-GD-020-DMD).
• Evidence of signed and dated informed consent/assent document(s) indicating that the participant (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. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
• In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the period of study drug administration and 6-week follow-up period.
• Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.

You may not qualify if…

• Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], and magnesium stearate).
• Ongoing participation in any other therapeutic clinical trial.
• Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

Where it is running

• UC Davis Medical Center — Sacramento, California, United States
• Stanford University Medical Center — Stanford, California, United States
• Children's Hospital Colorado - Center for Cancer and Blood Disorders — Aurora, Colorado, United States
• Child Neurology Center of Northwest Florida — Gulf Breeze, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research — St Louis, Missouri, United States
• Columbia University College of Physicians & Surgeons — New York, New York, United States
• Children's Hospital Cincinnati — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Childrens Medical Center Dallas, Texas — Dallas, Texas, United States
• Texas Children's Hospital — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Seattle Children's Hospital - Childhood Cancer and Blood Disorders — Seattle, Washington, United States
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia
• The Royal Children's Hospital — Parkville, Victoria, Australia
• UZ Leuven — Leuven, Belgium
• Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira — Rio de Janeiro, Brazil
• University of California, Los Angeles — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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