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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 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. The trial reports 1 study location across 1 state.
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04336826
The ClinicalTrials.gov registry entry for NCT04336826 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
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