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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03907072
• Date of registration: 05/04/2019
• Prospective Registration: Yes
• Primary sponsor: Wave Life Sciences Ltd.
• Public title: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy DYSTANCE 51
• Scientific 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)
• Date of first enrolment: September 4, 2019
• Target sample size: 6
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03907072
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2/Phase 3

Countries of recruitment

Belgium, Canada, Czechia, France, Italy, Sweden, United Kingdom, United States

Contacts

• Name: Michael A Panzara, MD, MPH
• Affiliation: Wave Life Sciences

Key inclusion & exclusion 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 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



Age minimum: 5 Years
Age maximum: 12 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: WVE-210201 (suvodirsen)

Drug: Placebo

Primary Outcome(s)

Change from baseline in dystrophin level (% normal dystrophin) [Time Frame: Day 1 to Week 12, Week 22, or Week 46]

Change from baseline in North Star Ambulatory Assessment (NSAA) [Time Frame: Day 1 through Week 48]

Secondary Outcome(s)

Change from baseline in forced vital capacity [Time Frame: Day 1 through Week 48]

Change from baseline in dystrophin level (% normal dystrophin) [Time Frame: Day 1 to Week 12, Week 22, or Week 46]

Change from baseline in North Star Ambulatory Assessment (NSAA) [Time Frame: Day 1 through Week 48]

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 95th percentile of stride velocity [Time Frame: Day 1 through Week 48]

Change from baseline in NSAA [Time Frame: Day 1 through Week 96]

Change from baseline in the 10-meter walk/run test [Time Frame: Day 1 through Week 48]

Secondary ID(s)

WVE-DMDX51-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors