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

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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)

key information

study id #: NCT03907072

condition: Duchenne Muscular Dystrophy

status: not yet recruiting

purpose:

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)

intervention: WVE-210201 (Suvodirsen), Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03907072

last updated: April 18, 2019

study details

start date: July 2019

estimated completion: August 2021

phase of development: Phase 2/Phase 3

size / enrollment: 150

primary outcomes:

  • 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)

secondary outcomes:

  • 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 ]

inclusion criteria:
• Diagnosis of 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 male, able to walk independently for at least 10 meters in 20 seconds or less at the time of Screening visit
• Stable pulmonary and cardiac function, as measured by:
- Reproducible percent predicted forced vital capacity (FVC) >=50%
- 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
• 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:
• Cardiac insufficiency:
- 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
- Any other evidence of clinically significant structural or functional heart abnormality
- A cardiac troponin I value > 0.2 ng/mL
• 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
• Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
• Received prior treatment with gene therapy for DMD
• Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection
• Received any investigational drug within 3 months or 5 half-lives, whichever is longer, prior to the planned Baseline biopsy collection

study contacts

sponsor: Wave Life Sciences Ltd.

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

investigators: Michael A Panzara, MD, MPH