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A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT03648827 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2022-04-05

Study results available
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Summary

This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and 2 validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.

Conditions

Interventions

DRUG

Ataluren

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

Sponsors & Collaborators

Principal Investigators

  • Francesco Bibbiani, MD · PTC Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2018-12-21
Primary Completion
2020-10-23
Completion
2020-10-23
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT03648827 on ClinicalTrials.gov