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Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT04906460
Recruitment Status : Not yet recruiting
First Posted : May 28, 2021
Last Update Posted : June 2, 2021
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Brief Summary:
This is a Phase 1b/2a 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

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: WVE-N531 Phase 1 Phase 2

Detailed Description:
The study will include a total of 15 patients. An initial cohort will receive ascending doses of WVE-N531. Up to 4 dose levels (administered ≥4 weeks apart) will be evaluated in order to select a dose level for further multiple dose evaluation. The initial patients will receive up to 3 additional doses every other week at that dose level. Additional patients will then be enrolled and dosed every other week at that level. All patients will receive a maximum of 7 total doses followed by a minimum 8 week safety monitoring period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Estimated Study Start Date : June 2021
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2022


Arm Intervention/treatment
Experimental: WVE-N531 Drug: WVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)




Primary Outcome Measures :
  1. Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 (initial dose) to a minimum of 8 weeks after the last dose ]

Secondary Outcome Measures :
  1. Pharmacokinetics: Concentration of WVE-N531 in muscle tissue [ Time Frame: Day 1 (initial dose) through 2 weeks after the last dose ]
  2. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Ambulatory or non-ambulatory male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
  2. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
  3. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL).
  4. 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 <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.
  5. Adequate deltoid muscle at Screening to perform open muscle biopsies.
  6. 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 Criteria:

  1. Cardiac insufficiency:

    1. 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.
    2. Any other evidence of clinically significant structural or functional heart abnormality.
    3. A cardiac troponin I value >0.2 ng/mL on initial and repeat testing if initial test is elevated at Screening.
  2. 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.
  3. Received prior treatment with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) or drisapersen.
  4. Received prior treatment with gene therapy for DMD.
  5. Received treatment with ataluren, viltolarsen, eteplirsen, or golodirsen within the 14 weeks prior to Screening.
  6. Received any investigational drug within 3 months or 5 half-lives, whichever is longer prior to Screening.

Information from the National Library of Medicine

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


Contacts
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Contact: Clinical Operations 855-215-4687 clinicaltrials@wavelifesci.com

Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Medical Director, MD Wave Life Sciences
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Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT04906460    
Other Study ID Numbers: WVE-N531-001
First Posted: May 28, 2021    Key Record Dates
Last Update Posted: June 2, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked