A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Public ClinicalTrials.gov record NCT03648827. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03648827
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years to 7 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 20, 2018
- Primary completion
- Oct 22, 2020
- Completion
- Oct 22, 2020
- Last update posted
- Apr 4, 2022
2018 – 2020
United States locations
- U.S. sites
- 9
- U.S. states
- 7
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Childrens Hospital | Phoenix | Arizona | 85016 | — |
| University of California, Los Angeles (UCLA) | Los Angeles | California | 90025 | — |
| University of California (UC) Davis Medical Center | Sacramento | California | 95817 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Columbia University College of Physicians & Surgeons | New York | New York | 10032 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| University of Texas Heath Science Center at San Antonio | San Antonio | Texas | 78229-3900 | — |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03648827, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 4, 2022 · Synced Sep 2, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03648827 live on ClinicalTrials.gov.