Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
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Official title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks).
Study identification
- NCT ID
- NCT05524883
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 86 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 16 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
- Aug 12, 2022 (Actual)
- Primary completion
- Sep 2031 (Estimated)
- Completion
- Sep 2031 (Estimated)
- Last update posted
- Aug 11, 2026
2022 – 2031
United States locations
- U.S. sites
- 11
- U.S. states
- 9
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California San Diego | La Jolla | California | 92037 | — |
| UCLA University California of Los Angeles | Los Angeles | California | 90095 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| UMass Memorial Medical Center | Worcester | Massachusetts | 01655 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Shriners Hospitals for Children Portland | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224-1334 | — |
| University of Utah - PPDS | Salt Lake City | Utah | 08412 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23219 | — |
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 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05524883, 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 Aug 11, 2026 · Synced Nov 14, 2026
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