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

NCT ID: NCT03907072

Last Updated: 2021-05-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-04
• Study Completion Date: 2020-01-09

Brief 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)

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

WVE-210201 (3 mg/kg)

Weekly IV administrations of WVE-210210 at 3 mg/kg

Group Type: EXPERIMENTAL

WVE-210201 (suvodirsen)

Intervention Type: DRUG

WVE-210201 is a stereopure antisense oligonucleotide (ASO)

WVE-210201 (4.5 mg/kg)

Weekly IV administrations of WVE-210210 at 4.5 mg/kg

Group Type: EXPERIMENTAL

WVE-210201 (suvodirsen)

Intervention Type: DRUG

WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Placebo

Weekly IV administrations of phosphate buffered saline solution visually identical in appearance to WVE-21021

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Buffered saline solution

Interventions

WVE-210201 (suvodirsen)

WVE-210201 is a stereopure antisense oligonucleotide (ASO)

Intervention Type: DRUG

Placebo

Buffered saline solution

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Wave Life Sciences Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael A Panzara, MD, MPH

Role: STUDY_DIRECTOR

Wave Life Sciences

Locations

Yale University

New Haven, Connecticut, United States

Rare Disease Research, LLC.

Atlanta, Georgia, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

University of Massachusetts

Worcester, Massachusetts, United States

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Institut de Myologie

Liège, Liege, Belgium

UZ Gent

Ghent, , Belgium

Universitaire Ziekenhuizen Leuven

Leuven, , Belgium

Alberta Children's Hospital

Calgary, Alberta, Canada

London Health Sciences Centre - Hospital

London, Ontario, Canada

Fakultni Nemocnice v Motole

Prague, , Czechia

Hôpitaux Universitaires de Strasbourg

Strasbourg, Bas-Rhin, France

Hôpital Des Enfants

Toulouse, Haute-Garonne, France

Hopital Armand Trosseau

Paris, , France

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, Italy

U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud

Messina, , Italy

Ospedale San Reffaele Via Olgettina, 60

Milan, , Italy

Fondazione Policlinico Universitario A Gemelli

Roma, , Italy

Drottning Silvias Barn Och Ungdomssjukhus

Gothenburg, , Sweden

Leeds Teaching Hospitals NHS Trust

Leeds, , United Kingdom

Great Ormond Street Hospital (GOSH)

London, , United Kingdom

Countries

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

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

WVE-DMDX51-003

Identifier Type: -

Identifier Source: org_study_id