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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 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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04906460
The ClinicalTrials.gov registry entry for NCT04906460 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (94% lower). The listed sponsor is Wave Life Sciences, which has 2 total studies on file at ClinicalTrials.gov.
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 reports 5 study locations spanning 4 distinct geographic areas - top geographies include 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.
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.