MedTrace Logo

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

NCT02819557 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2020-08-28

Study results available
· View outcomes & findings →

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.

Conditions

Interventions

DRUG

Ataluren

White to off-white powder for oral suspension.

Sponsors & Collaborators

Principal Investigators

  • Francesco Bibbiani, MD · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-06-09
Primary Completion
2018-02-09
Completion
2018-02-09

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02819557 on ClinicalTrials.gov