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NCT04906460 · ClinicalTrials.gov registry record · Phase 1
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
A Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Wave Life Sciences.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
- 5
- Study locations
NCT04906460: Recruiting Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Wave Life Sciences.
NCT04906460 is a Phase 1 study of Duchenne Muscular Dystrophy that is actively recruiting participants, run by Wave Life Sciences. The registered enrollment target is 26 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (94% lower). The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04906460, a Phase 1 study of Duchenne Muscular Dystrophy, is actively recruiting participants, sponsored by Wave Life Sciences.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
- 5
- Study locations
Study Summary
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.
Conditions Studied
Interventions
- DRUG WVE-N531
Study Locations (5)
Other
- Istiklal Hospital/ Clinical Research Unit - Amman
- The Specialty Hospital (TSH)/ Advanced Clinical Center - Amman
Arkansas
- Arkansas Children's Hospital - Little Rock
Georgia
- Rare Disease Research LLC - Atlanta
Oxford
- Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust - Headington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2021-09-28 |
| Est. Completion | 2027-04-24 |
| Phase | Phase 1 |
What NCT04906460 shows while recruiting
NCT04906460 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (94% lower).
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which WVE-N531 is the first listed.
NCT04906460 lists 5 locations in 4 states (Other, Arkansas, Georgia).
Frequently Asked Questions
What is clinical trial NCT04906460 about?
NCT04906460 is a clinical study titled "Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)". This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the D...
What is the current status of trial NCT04906460?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2021-09-28. Estimated completion is 2027-04-24.
What conditions does trial NCT04906460 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT04906460?
The interventions under investigation include: WVE-N531 (DRUG).
Who is sponsoring clinical trial NCT04906460?
This trial is sponsored by Wave Life Sciences, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04906460 being conducted?
This trial has 5 study locations across Arkansas, Georgia, Oxford. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04906460's enrollment target sits among peer trials
26 33rd of 48 higher than 15 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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