Status:
RECRUITING
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Lead Sponsor:
Wave Life Sciences Ltd.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4-18 years
Phase:
PHASE1
PHASE2
Brief 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 musc...
Detailed Description
Following completion of Part A, eligible patients rolled over into Part B to continue to receive treatment. In addition, new patients were enrolled up to a total of 11 patients in Part B. All patients...
Eligibility Criteria
Inclusion
- 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):
- 1\. 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.
- 8\. 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:
- 1\. 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.
- 7\. Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.
- 8\. 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 .
Exclusion
- 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.
Key Trial Info
Start Date :
September 28 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 24 2027
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT04906460
Start Date
September 28 2021
End Date
April 24 2027
Last Update
December 15 2025
Active Locations (5)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202-3500
2
Rare Disease Research LLC
Atlanta, Georgia, United States, 30329
3
Istiklal Hospital/ Clinical Research Unit
Amman, Jordan
4
The Specialty Hospital (TSH)/ Advanced Clinical Center
Amman, Jordan