Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT02819557 · ClinicalTrials.gov registry record · Phase 2

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

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

Completed
Registry status
Phase 2
Development phase
14
Enrollment target
6
Study locations

NCT02819557 is a Phase 2 study of Duchenne Muscular Dystrophy that has completed, run by PTC Therapeutics. The registered enrollment target is 14 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower). The trial reports 6 study locations across 6 states.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT02819557, a Phase 2 study of Duchenne Muscular Dystrophy, has completed, sponsored by PTC Therapeutics.

COMPLETED
Registry status
Phase 2
Development phase
14 participants
Enrollment target
6
Study locations

Study Summary

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

Interventions

  • DRUG Ataluren

Study Locations (6)

Florida

  • Child Neuro NWF - Gulf Breeze

Illinois

  • Rush University Medical Center - Chicago

Massachusetts

  • Children's Hospital Boston - Boston

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati

Texas

  • Children's Medical Center Dallas - Dallas

Utah

  • University of Utah - Salt Lake City

Trial Details

FieldValue
Enrollment Target 14 participants
Start Date 2016-06-09
Est. Completion 2018-02-09
Phase Phase 2

Sponsor

PTC Therapeutics

52 total trials

What the Registry Record Tells You About NCT02819557

The ClinicalTrials.gov registry entry for NCT02819557 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 14 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower). The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.

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.

NCT02819557 reports 6 study locations spanning 6 distinct geographic areas - top geographies include Florida, Illinois, Massachusetts.

Frequently Asked Questions

What is clinical trial NCT02819557 about?

NCT02819557 is a clinical study titled "Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy". This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

What is the current status of trial NCT02819557?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 14 participants. The study started on 2016-06-09. Estimated completion is 2018-02-09.

What conditions does trial NCT02819557 study?

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

What interventions are being tested in trial NCT02819557?

The interventions under investigation include: Ataluren (DRUG).

Who is sponsoring clinical trial NCT02819557?

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

Where is trial NCT02819557 being conducted?

This trial has 6 study locations across Florida, Illinois, Massachusetts, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.