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

Duchenne Muscular Dystrophy Clinical Trial

— DYSTANCE 51  

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03907072
• Other study ID #: WVE-DMDX51-003
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: September 4, 2019
• Est. completion date: January 9, 2020

Study information

• Verified date: April 2021
• Source: Wave Life Sciences Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period 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)

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: January 9, 2020
• Est. primary completion date: December 16, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• WVE-210201 (suvodirsen)
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
• Placebo
Buffered saline solution

Locations

Country	Name	City	State
Belgium	UZ Gent	Gent
Belgium	Universitaire Ziekenhuizen Leuven	Leuven
Belgium	Institut de Myologie	Liège	Liege
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	London Health Sciences Centre - Hospital	London	Ontario
Czechia	Fakultni Nemocnice v Motole	Praha 5
France	Hopital Armand Trosseau	Paris
France	Hôpitaux Universitaires de Strasbourg	Strasbourg	Bas-Rhin
France	Hôpital Des Enfants	Toulouse	Haute-Garonne
Italy	U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud	Messina
Italy	Ospedale San Reffaele Via Olgettina, 60	Milano
Italy	Fondazione Policlinico Universitario A Gemelli	Roma
Italy	Ospedale Pediatrico Bambino Gesù	Roma	Lazio
Sweden	Drottning Silvias Barn Och Ungdomssjukhus	Göteborg
United Kingdom	Leeds Teaching Hospitals NHS Trust	Leeds
United Kingdom	Great Ormond Street Hospital (GOSH)	London
United States	Rare Disease Research, LLC.	Atlanta	Georgia
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Yale University	New Haven	Connecticut
United States	University of Massachusetts	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Dystrophin Level (% Normal Dystrophin)	US/other regions (as applicable)	Day 1 to Week 12, Week 22, or Week 46
Primary	Change From Baseline in North Star Ambulatory Assessment (NSAA)	European Union (EU)/other regions (as applicable)	Day 1 through Week 48
Secondary	Change From Baseline in North Star Ambulatory Assessment (NSAA)	US/other regions (as applicable)	Day 1 through Week 48
Secondary	Change From Baseline in Dystrophin Level (% Normal Dystrophin)	European Union (EU)/other regions (as applicable)	Day 1 to Week 12, Week 22, or Week 46
Secondary	Change From Baseline in Upper Limb Proximal Strength		Day 1 through Week 48
Secondary	Change From Baseline in 4-stair Climb		Day 1 through Week 48
Secondary	Change From Baseline in the 10-meter Walk/Run Test		Day 1 through Week 48
Secondary	Change From Baseline in Forced Vital Capacity		Day 1 through Week 48
Secondary	Change From Baseline in the 95th Percentile of Stride Velocity		Day 1 through Week 48
Secondary	Change From Baseline in NSAA	Long-term evaluation, open label from Week 48 through Week 96	Day 1 through Week 96