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A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 (Suvodirsen) in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

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ClinicalTrials.gov Identifier: NCT03907072
Recruitment Status : Not yet recruiting
First Posted : April 8, 2019
Last Update Posted : April 8, 2019
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Brief Summary:
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study 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)

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: WVE-210201 (suvodirsen) Drug: Placebo Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients With Duchenne Muscular Dystrophy
Estimated Study Start Date : July 2019
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : August 2021


Arm Intervention/treatment
Experimental: WVE-210201 (Dose A) Drug: WVE-210201 (suvodirsen)
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Experimental: WVE-210201 (Dose B) Drug: WVE-210201 (suvodirsen)
WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Placebo Comparator: Placebo Drug: Placebo
Buffered saline solution




Primary Outcome Measures :
  1. 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)

  2. Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 to Week 48 ]
    European Union (EU)/other regions (as applicable)


Secondary Outcome Measures :
  1. Change from baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Day 1 through Week 48 ]
    US/other regions (as applicable)

  2. 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)

  3. Change from baseline in upper limb proximal strength [ Time Frame: Day 1 through Week 48 ]
  4. Change from baseline in 4-stair climb [ Time Frame: Day 1 through Week 48 ]
  5. Change from baseline in the 10-meter walk/run test [ Time Frame: Day 1 through Week 48 ]
  6. Change from baseline in forced vital capacity [ Time Frame: Day 1 through Week 48 ]
  7. Change from baseline in the 95th percentile of stride velocity [ Time Frame: Day 1 through Week 48 ]


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Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
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 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 20 seconds or less at the time of Screening visit
  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

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): NCT03907072


Contacts
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Contact: Clinical Operations Wave Life Sciences Ltd. 855-215-4687 clinicaltrials@wavelifesci.com

Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Michael A Panzara, MD, MPH Wave Life Sciences

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Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT03907072     History of Changes
Other Study ID Numbers: WVE-DMDX51-003
First Posted: April 8, 2019    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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