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NCT03796637 · ClinicalTrials.gov registry record · Phase 2

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

A Phase 2 study of Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.

Completed
Registry status
Phase 2
Development phase
6
Enrollment target
1
Study location

NCT03796637: Completed Phase 2 study of Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.

NCT03796637 is a Phase 2 study of Duchenne Muscular Dystrophy that has completed, run by PTC Therapeutics. The registered enrollment target is 6 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT03796637, a Phase 2 study of 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 generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for ≥9 months. The study will have a single visit (Visit 1).

Primary Outcome

The mean dystrophin protein levels were measured by ECL. Dystrophin levels are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). Results below the limit of quantitation were imputed as half of lower limit of quantitation (LLOQ). LLOQ = 0.5 micrograms (μg)/milliliter (mL)

Conditions Studied

Duchenne Muscular Dystrophy

Interventions

  • DRUG Ataluren

Study Locations (1)

California

  • University of California, Los Angeles (UCLA) - Los Angeles

Trial Details

FieldValue
Enrollment Target 6 participants
Start Date 2019-04-11
Est. Completion 2019-06-03
Phase Phase 2
PTC Therapeutics

52 total trials

What the finished NCT03796637 record still lists

NCT03796637 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 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (99% lower).

The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which Ataluren is the first listed.

NCT03796637 reports a single indexed study location in California.

Frequently Asked Questions

What is clinical trial NCT03796637 about?

NCT03796637 is a clinical study titled "A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren". This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for ≥9 months. The study will hav...

What is the current status of trial NCT03796637?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 6 participants. The study started on 2019-04-11. Estimated completion is 2019-06-03.

What conditions does trial NCT03796637 study?

This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.

What interventions are being tested in trial NCT03796637?

The interventions under investigation include: Ataluren (DRUG).

Who is sponsoring clinical trial NCT03796637?

This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03796637 being conducted?

This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Duchenne Muscular Dystrophy

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT03796637's enrollment target sits among peer trials

6 47th of 48 higher than 2 of 48 other Duchenne Muscular Dystrophy trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy 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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Related registry views

Source: ClinicalTrials.gov NCT03796637, the US trial registry maintained by the National Library of Medicine. NCT03796637 (small enrollment · single site footprint · completed) retrieved and formatted by PlainTrial, see methodology.