Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

April 29, 2021 updated by: Wave Life Sciences Ltd.

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)

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)

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Gent, Belgium, 9000
        • UZ Gent
      • Leuven, Belgium
        • Universitaire Ziekenhuizen Leuven
    • Liege
      • Liège, Liege, Belgium, 4000
        • Institut de Myologie
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B6A8
        • Alberta Children's Hospital
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • London Health Sciences Centre - Hospital
  • Czechia
      • Praha 5, Czechia, 15006
        • Fakultni nemocnice v Motole
  • France
      • Paris, France, 75012
        • Hopital Armand Trosseau
    • Bas-Rhin
      • Strasbourg, Bas-Rhin, France, 67098
        • Hopitaux Universitaires de Strasbourg
    • Haute-Garonne
      • Toulouse, Haute-Garonne, France, 31059
        • Hopital des Enfants
  • Italy
      • Messina, Italy, 98125
        • U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
      • Milano, Italy, 20132
        • Ospedale San Reffaele Via Olgettina, 60
      • Roma, Italy, 8, 00168
        • Fondazione Policlinico Universitario A Gemelli
    • Lazio
      • Roma, Lazio, Italy, 165
        • Ospedale Pediatrico Bambino Gesu
  • Sweden
      • Göteborg, Sweden, 41650
        • Drottning Silvias Barn Och Ungdomssjukhus
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds Teaching Hospitals NHS Trust
      • London, United Kingdom, WC1N EH
        • Great Ormond Street Hospital (GOSH)
  • United States
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Rare Disease Research, LLC.
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Worcester, Massachusetts, United States, 01605
        • University of Massachusetts
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hospital of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 8 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: WVE-210201 (3 mg/kg)
Weekly IV administrations of WVE-210210 at 3 mg/kg
Drug: WVE-210201 (suvodirsen)
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
Experimental: WVE-210201 (4.5 mg/kg)
Weekly IV administrations of WVE-210210 at 4.5 mg/kg
Drug: WVE-210201 (suvodirsen)
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
Placebo Comparator: Placebo
Weekly IV administrations of phosphate buffered saline solution visually identical in appearance to WVE-21021
Drug: Placebo
Buffered saline solution

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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)
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
European Union (EU)/other regions (as applicable)
Day 1 through Week 48

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in North Star Ambulatory Assessment (NSAA)
Time Frame: Day 1 through Week 48
US/other regions (as applicable)
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
European Union (EU)/other regions (as applicable)
Day 1 to Week 12, Week 22, or Week 46
Change From Baseline in Upper Limb Proximal Strength
Time Frame: Day 1 through Week 48
Day 1 through Week 48
Change From Baseline in 4-stair Climb
Time Frame: Day 1 through Week 48
Day 1 through Week 48
Change From Baseline in the 10-meter Walk/Run Test
Time Frame: Day 1 through Week 48
Day 1 through Week 48
Change From Baseline in Forced Vital Capacity
Time Frame: Day 1 through Week 48
Day 1 through Week 48
Change From Baseline in the 95th Percentile of Stride Velocity
Time Frame: Day 1 through Week 48
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
Day 1 through Week 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Wave Life Sciences Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Michael A Panzara, MD, MPH, Wave Life Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 4, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 16, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 9, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 5, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 5, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 8, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 20, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 29, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • WVE-DMDX51-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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