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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)

NCT04336826 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-04-01

Study results available
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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

  • Nonsene Mutation Duchenne Muscular Dystrophy

Interventions

DRUG

Ataluren

Ataluren will be administered as per the dose and schedule specified in the arm.

Sponsors & Collaborators

Principal Investigators

  • Vinay Penematsa · PTC Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
2 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-29
Primary Completion
2023-08-07
Completion
2023-08-07
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
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 NCT04336826 on ClinicalTrials.gov