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 :
Terminated
(Lack of efficacy)
First Posted : April 8, 2019
Last Update Posted : January 18, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: WVE-210201 (suvodirsen) Drug: Placebo | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 6 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 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51) |
Actual Study Start Date : | September 4, 2019 |
Actual Primary Completion Date : | December 16, 2019 |
Actual Study Completion Date : | January 9, 2020 |

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 |
- 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)
- 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

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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 |
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 10 seconds or less at the time of Screening visit (performed as part of the NSAA)
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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:
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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

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

Study Director: | Michael A Panzara, MD, MPH | Wave Life Sciences |
Responsible Party: | Wave Life Sciences Ltd. |
ClinicalTrials.gov Identifier: | NCT03907072 |
Other Study ID Numbers: |
WVE-DMDX51-003 |
First Posted: | April 8, 2019 Key Record Dates |
Last Update Posted: | January 18, 2020 |
Last Verified: | January 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |