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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.

Tracking Information
First Submitted Date  ICMJE May 24, 2021
First Posted Date  ICMJE May 28, 2021
Last Update Posted Date June 2, 2021
Estimated Study Start Date  ICMJE June 2021
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 24, 2021)
Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 (initial dose) to a minimum of 8 weeks after the last dose ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 24, 2021)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: WVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
Study Arms  ICMJE Experimental: WVE-N531
Intervention: Drug: WVE-N531
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: May 24, 2021)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2022
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Ambulatory or non-ambulatory male
Ages  ICMJE 5 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Operations 855-215-4687 clinicaltrials@wavelifesci.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04906460
Other Study ID Numbers  ICMJE WVE-N531-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Wave Life Sciences Ltd.
Study Sponsor  ICMJE Wave Life Sciences Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director, MD Wave Life Sciences
PRS Account Wave Life Sciences Ltd.
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP