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NCT04336826 · ClinicalTrials.gov registry record · Phase 2
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)
A Phase 2 study of Nonsene Mutation Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 6
- Enrollment target
- 1
- Study location
NCT04336826: Completed Phase 2 study of Nonsene Mutation Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.
NCT04336826 is a Phase 2 study of Nonsene Mutation Duchenne Muscular Dystrophy that has completed, run by PTC Therapeutics. The registered enrollment target is 6 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04336826, a Phase 2 study of Nonsene Mutation Duchenne Muscular Dystrophy, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 6 participants
- Enrollment target
- 1
- Study location
Study Summary
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
Primary Outcome
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 def
Conditions Studied
Interventions
- DRUG Ataluren
Study Locations (1)
Georgia
- Rare Disease Research, LLC - Atlanta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2021-12-29 |
| Est. Completion | 2023-08-07 |
| Phase | Phase 2 |
What the finished NCT04336826 record still lists
NCT04336826 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Nonsene Mutation Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which Ataluren is the first listed.
NCT04336826 reports a single indexed study location in Georgia.
Frequently Asked Questions
What is clinical trial NCT04336826 about?
NCT04336826 is a clinical study titled "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)". This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
What is the current status of trial NCT04336826?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 6 participants. The study started on 2021-12-29. Estimated completion is 2023-08-07.
What conditions does trial NCT04336826 study?
This clinical trial studies the following conditions: Nonsene Mutation Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT04336826?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT04336826?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04336826 being conducted?
This trial has 1 study location across Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04336826's enrollment target sits among peer trials
6 1996th of 2000 higher than 1 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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