NCT03907072

DYSTANCE 51 [TERMINATED]

A Randomised, Double-blind, Placebo-controlled, Efficacy and Safety Study of Suvodirsen (WVE-210201) With Open Label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

Locations:

London - GOSH

Newcastle

Oxford

Alder Hey

Leeds

DYSTANCE 51 is a global, phase 2/3, multicentre, randomised, double-blind, placebo-controlled clinical trial that will evaluate the efficacy and safety of suvodirsen in ambulatory male paediatric patients with DMD amenable to exon 51 skipping intervention.

Primary Outcome Measures

Change from baseline in dystrophin level (% normal dystrophin) - US/other regions (as applicable)
Change from baseline in North Star Ambulatory Assessment (NSAA) - EU/other regions (as applicable)

Secondary Outcome Measures

Change from baseline in North Star Ambulatory Assessment (NSAA)
Change from baseline in dystrophin level (% normal dystrophin)
Change from baseline in upper limb proximal strength
Change from baseline in 4-stair climb
Change from baseline in the 10-meter walk/run test
Change from baseline in forced vital capacity
Change from baseline in the 95th percentile of stride velocity

Can I take part?

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)
5 to 12 years of age (up to 13th birthday) at the time of randomisation
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

Age

5 - 12 years

Mutation Specific

Muscle Biopsy

Required

Ambulation

ambulant