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

Recruiting now · Phase 1/Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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 facts

Study ID

NCT04906460

Run by

Wave Life Sciences USA, Inc.

People needed

26

Starts

2021-09-28

Expected to finish

2027-04-24

Last updated by the study team

2025-12-15

Who can join

Age: 4 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Part A and Part B:
• Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B
• Diagnosis of DMD based on clinical phenotype.
• Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
• Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ).
• Ambulatory or non-ambulatory male
• Stable pulmonary and cardiac function, as measured by the following: (Part B):
• Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients ≥10 years of age, as measured (and documented) by echocardiogram (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.
• Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.
• Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit (Part B ).
• Part C
• New patients to be screened for Part C.
• Diagnosis of DMD based on clinical phenotype.
• Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
• Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) .
• Ambulatory male
• Stable pulmonary and cardiac function, as measured by the following:
• Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) >55% in patients as measured (and documented) by echocardiogram (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.
• Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.
• Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit .

You may not qualify if…

• Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
• Part B and Part C: Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study.
• Part B: Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit
• Part C: Any recreational substance use (including prescribed cannabinoids), with the exception of nicotine, irrespective of legality, within 2 months prior to Screening and/or unwilling to refrain from such use for the duration of the study.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• Rare Disease Research LLC — Atlanta, Georgia, United States (enrolling)
• Istiklal Hospital/ Clinical Research Unit — Amman, Jordan (enrolling)
• The Specialty Hospital (TSH)/ Advanced Clinical Center — Amman, Jordan (enrolling)
• Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust — Headington, Oxford, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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