NCT04336826Phase 2Completed

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2023-08-07Actual

Enrollment · participants6Actual

Source updated2024-04-01Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Nonsene Mutation Duchenne Muscular Dystrophy

Treatments or interventions studied

AtalurenDrugAlso known as PTC124

Primary outcomes · main study measures

Read the study measures

measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs); description: An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious adverse event (SAE): an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event. A TEAE was defined as an AE that occurred or worsened while on ataluren (on or after first dose of ataluren) up to 4 weeks after the last dose. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.; timeFrame: Baseline up to Week 28

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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