An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT04708314. 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
An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
Study identification
- NCT ID
- NCT04708314
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 4
- Lead sponsor
- Rare Disease Research, LLC
- Other
- Enrollment
- 2 participants
Conditions and interventions
Conditions
Interventions
- Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] Drug
Drug
Eligibility (public fields only)
- Age range
- 7 Years and older
- 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
- Oct 30, 2020
- Primary completion
- May 12, 2021
- Completion
- May 12, 2021
- Last update posted
- Apr 2, 2026
2020 – 2021
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
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 NCT04708314, 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 2, 2026 · Synced May 19, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04708314 live on ClinicalTrials.gov.