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Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03508947
Recruitment Status : Completed
First Posted : April 26, 2018
Last Update Posted : April 8, 2019
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Tracking Information
First Submitted Date  ICMJE April 16, 2018
First Posted Date  ICMJE April 26, 2018
Last Update Posted Date April 8, 2019
Actual Study Start Date  ICMJE January 24, 2018
Actual Primary Completion Date March 6, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 25, 2018)
  • Safety: Number of patients with adverse events (AEs) [ Time Frame: Day 1 to Day 85 (end of study) ]
  • Safety: Severity of AEs [ Time Frame: Day 1 to Day 85 (end of study) ]
  • Safety: Number of patients with serious AEs (SAEs) [ Time Frame: Day 1 to Day 85 (end of study) ]
  • Safety and Tolerability: Number of patients who withdraw due to AEs [ Time Frame: Day 1 to Day 85 (end of study) ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 24, 2018)
Safety and tolerability of WVE-210201 [ Time Frame: Day 1 to Day 85 (end of study) ]
Number of patients with adverse events (AEs), serious AEs (SAEs), or who withdraw due to AEs, and severity of AEs.
Change History Complete list of historical versions of study NCT03508947 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2018)
  • Pharmacokinetics (PK): Maximum observed concentration (Cmax) [ Time Frame: Day 1, Day 2, and Day 8 ]
  • PK: Time of occurrence of Cmax (tmax) [ Time Frame: Day 1, Day 2, and Day 8 ]
  • PK: Area under the plasma concentration-time curve (AUC 0-t) [ Time Frame: Day 1, Day 2, and Day 8 ]
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 Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
Brief Summary This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: WVE-210201
    WVE-210201 is a stereopure antisense oligonucleotide (ASO)
  • Drug: Placebo
    Sodium Chloride
Study Arms  ICMJE
  • Experimental: WVE-210201 (Dose A) or placebo
    Interventions:
    • Drug: WVE-210201
    • Drug: Placebo
  • Experimental: WVE-210201 (Dose B) or placebo
    Interventions:
    • Drug: WVE-210201
    • Drug: Placebo
  • Experimental: WVE-210201 (Dose C) or placebo
    Interventions:
    • Drug: WVE-210201
    • Drug: Placebo
  • Experimental: WVE-210201 (Dose D) or placebo
    Interventions:
    • Drug: WVE-210201
    • Drug: Placebo
  • Experimental: WVE-210201 (Dose E) or placebo
    Interventions:
    • Drug: WVE-210201
    • Drug: Placebo
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 Completed
Actual Enrollment  ICMJE
 (submitted: April 5, 2019)
36
Original Estimated Enrollment  ICMJE
 (submitted: April 24, 2018)
40
Actual Study Completion Date  ICMJE March 6, 2019
Actual Primary Completion Date March 6, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase
  • Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping
  • Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 years
  • Stable pulmonary and cardiac function as measured by:

    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 within one year prior to enrollment into the study.

Exclusion Criteria:

  • Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
  • Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
  • Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study.
  • Currently on anticoagulants or antithrombotics.
  • Received treatment with eteplirsen or ataluren within the past 14 weeks.
  • Received prior treatment with drisapersen.
  • Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 5 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   France,   Italy,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03508947
Other Study ID Numbers  ICMJE WVE-DMDX51-001
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
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Wave Life Sciences Ltd.
Study Sponsor  ICMJE Wave Life Sciences Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Michael A Panzara, MD, MPH Wave Life Sciences Ltd.
PRS Account Wave Life Sciences Ltd.
Verification Date April 2019

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