Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT04906460|
Recruitment Status : Recruiting
First Posted : May 28, 2021
Last Update Posted : July 21, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: WVE-N531||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||September 28, 2021|
|Estimated Primary Completion Date :||December 2022|
|Estimated Study Completion Date :||December 2022|
WVE-N531 is an antisense oligonucleotide (ASO)
- Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 (initial dose) to a minimum of 8 weeks after the last dose ]
- Pharmacokinetics: Concentration of WVE-N531 in muscle tissue [ Time Frame: Day 1 (initial dose) through 2 weeks after the last dose ]
- Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531 [ Time Frame: Day 1 (initial dose) through 2 weeks after the last dose ]
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|Ages Eligible for Study:||5 Years to 18 Years (Child, Adult)|
|Sexes Eligible for Study:||Male|
|Gender Based Eligibility:||Yes|
|Gender Eligibility Description:||Ambulatory or non-ambulatory male|
|Accepts Healthy Volunteers:||No|
- Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
- 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).
Stable pulmonary and cardiac function, as measured by the following:
- Reproducible percent predicted forced vital capacity (FVC) ≥50%;
- 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) or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.
- Adequate deltoid muscle at Screening to perform open muscle biopsies.
- 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.
- Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study; however, cardiomyopathy that is managed by angiotensin-converting-enzyme (ACE) inhibitors or beta blockers is acceptable provided the participant meets the LVEF inclusion criterion.
- Any other evidence of clinically significant structural or functional heart abnormality.
- Need for daytime mechanical or noninvasive ventilation OR anticipated need for daytime mechanical or noninvasive ventilation within the next year in the opinion of the Investigator. Nighttime noninvasive ventilation is permitted.
- Received prior treatment with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) or drisapersen.
- Received prior treatment with gene therapy for DMD.
- Received treatment with ataluren, viltolarsen, eteplirsen, or golodirsen within the 14 weeks prior to Screening.
- Received any investigational drug within 3 months or 5 half-lives, whichever is longer prior to Screening.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04906460
|Contact: Clinical Operationsemail@example.com|
|London Health Sciences Centre - Hospital||Recruiting|
|London, Ontario, Canada, N6A 5W9|
|Contact: Craig Campbell, MD MSC FRCPC 519-685-8500 ext 56216 Craig.Campbell@lhsc.on.ca|
|Contact: Cara Grobbecker 519-685-8500 ext 56216 Cara.Grobbecker@lhsc.on.ca|
|Principal Investigator: Craig Campbell, MD MSC FRCPC|
|Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust||Recruiting|
|Headington, Oxford, United Kingdom, OX3 9DU|
|Contact: Laurent Servais, MD, PhD 01865 227503 Laurent.Servais@ouh.nhs.uk|
|Principal Investigator: Laurent Servais, MD, PhD|
|Study Director:||Medical Director, MD||Wave Life Sciences|
|Responsible Party:||Wave Life Sciences Ltd.|
|Other Study ID Numbers:||
|First Posted:||May 28, 2021 Key Record Dates|
|Last Update Posted:||July 21, 2022|
|Last Verified:||July 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||Yes|
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked