Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Wave Life Sciences USA, Inc.
Study ID
NCT04906460
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
4 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • WVE-N531 — DRUG
    WVE-N531 is an antisense oligonucleotide (ASO)

Study Details

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.

Key Dates

Start date
Sep 28, 2021
Status verified
Sep 2025
Primary completion
Jun 27, 2026
Completion
Apr 24, 2027

Study Design

Enrollment
26 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: WVE-N531

Primary Outcome Measure

Part A: Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 (initial dose) up to 24 weeks after the last dose of Part A ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202-3500
Aravindhan Veerapandiyan, Dr
[email protected]
501-364-1850
Aravindhan Veerapandiyan, Dr (PRINCIPAL_INVESTIGATOR)
Rare Disease Research LLCAtlantaGeorgia30329
Scott Batchelor, Dr
[email protected]
404-782-8239
Scott Batchelor, Dr (PRINCIPAL_INVESTIGATOR)

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital· Little Rock, ARRare Disease Research LLC· Atlanta, GA

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