Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Public ClinicalTrials.gov record NCT04004065. 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
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose \[MAD\]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
Study identification
- NCT ID
- NCT04004065
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 62 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 7 Years to 21 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
- Jun 25, 2019
- Primary completion
- Oct 29, 2023
- Completion
- Feb 6, 2025
- Last update posted
- Mar 9, 2025
2019 – 2025
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California Davis Health | Sacramento | California | 95817 | — |
| Connecticut Children's | Farmington | Connecticut | 06032 | — |
| Northwest Florida Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center Research Inst. | Kansas City | Kansas | 66103 | — |
| University of Massachusetts | Worcester | Massachusetts | 01655 | — |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Austin Neuromuscular Center | Austin | Texas | 78756 | — |
| Children's Health Ambulatory Pavilion | Dallas | Texas | 75207 | — |
| Seattle Children's | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 14 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04004065, 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 Mar 9, 2025 · Synced Sep 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04004065 live on ClinicalTrials.gov.