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Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

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ClinicalTrials.gov Identifier: NCT03907072
Recruitment Status : Recruiting
First Posted : April 8, 2019
Last Update Posted : October 7, 2019
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Tracking Information
First Submitted Date  ICMJE April 5, 2019
First Posted Date  ICMJE April 8, 2019
Last Update Posted Date October 7, 2019
Actual Study Start Date  ICMJE September 4, 2019
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 2, 2019)
  • Change from baseline in dystrophin level (% normal dystrophin) [ Time Frame: Day 1 to Week 12, Week 22, or Week 46 ]
    US/other regions (as applicable)
  • Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 through Week 48 ]
    European Union (EU)/other regions (as applicable)
Original Primary Outcome Measures  ICMJE
 (submitted: April 5, 2019)
  • Change from baseline in dystrophin level (% normal dystrophin) [ Time Frame: Day 1 to Week 12, Week 22, or Week 46 ]
    US/other regions (as applicable)
  • Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 to Week 48 ]
    European Union (EU)/other regions (as applicable)
Change History Complete list of historical versions of study NCT03907072 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 2, 2019)
  • Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 through Week 48 ]
    US/other regions (as applicable)
  • Change from baseline in dystrophin level (% normal dystrophin) [ Time Frame: Day 1 to Week 12, Week 22, or Week 46 ]
    European Union (EU)/other regions (as applicable)
  • Change from baseline in upper limb proximal strength [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in 4-stair climb [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in the 10-meter walk/run test [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in forced vital capacity [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in the 95th percentile of stride velocity [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in NSAA [ Time Frame: Day 1 through Week 96 ]
    Long-term evaluation, open label from Week 48 through Week 96
Original Secondary Outcome Measures  ICMJE
 (submitted: April 5, 2019)
  • Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 through Week 48 ]
    US/other regions (as applicable)
  • Change from baseline in dystrophin level (% normal dystrophin) [ Time Frame: Day 1 to Week 12, Week 22, or Week 46 ]
    EU/other regions (as applicable)
  • Change from baseline in upper limb proximal strength [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in 4-stair climb [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in the 10-meter walk/run test [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in forced vital capacity [ Time Frame: Day 1 through Week 48 ]
  • Change from baseline in the 95th percentile of stride velocity [ Time Frame: Day 1 through Week 48 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)
Brief Summary This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping intervention (DYSTANCE 51)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: WVE-210201 (suvodirsen)
    WVE-210201 is a stereopure antisense oligonucleotide (ASO)
  • Drug: Placebo
    Buffered saline solution
Study Arms  ICMJE
  • Experimental: WVE-210201 (Dose A)
    Intervention: Drug: WVE-210201 (suvodirsen)
  • Experimental: WVE-210201 (Dose B)
    Intervention: Drug: WVE-210201 (suvodirsen)
  • Placebo Comparator: Placebo
    Intervention: 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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 5, 2019)
150
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date December 2021   (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 Dystrophin gene associated with DMD that is amenable to exon 51 skipping
  3. Ambulatory male, able to walk independently for at least 10 meters in 10 seconds or less at the time of Screening visit (performed as part of the NSAA)
  4. 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
  5. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy occurred ≥6 months prior to Screening, and no changes in dosing ≤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 patient 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
  2. Need for daytime mechanical or non-invasive ventilation OR anticipated need for daytime mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator. Nighttime non-invasive ventilation is permitted
  3. Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
  4. Received prior treatment with gene therapy for DMD
  5. Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection
  6. Received any investigational drug within 3 months or 5 half-lives, whichever is longer, prior to the planned Baseline biopsy collection
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 5 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Operations Wave Life Sciences Ltd. 855-215-4687 clinicaltrials@wavelifesci.com
Listed Location Countries  ICMJE Belgium,   Canada,   France,   Italy,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03907072
Other Study ID Numbers  ICMJE WVE-DMDX51-003
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
PRS Account Wave Life Sciences Ltd.
Verification Date October 2019

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