Duchenne Muscular DystrophyPhase 2/3DYSTANCE 51NCT03907072
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
A Phase 2/3 study testing WVE-210201 (suvodirsen) in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 22 sites in 8 countries.
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) Read more about this study
- Who is it for?
- Ages 5 to 12 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- WVE-210201 (suvodirsen)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 22 sitesin 8 countries
- Study dates
- 4 Sep 2019to 9 Jan 2020
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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)
- 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
Who cannot join
- 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)
Show all 9Show fewer
- 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
Trial locations and contacts
22 sites in 8 countries.
22 sites in 8 countries
United States6 sites
Rare Disease Research, LLC.
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Yale University
New Haven, Connecticut, 06510
University of Massachusetts
Worcester, Massachusetts, 01605
Italy4 sites
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
Messina, 98125
Ospedale San Reffaele Via Olgettina, 60
Milan, 20132
Fondazione Policlinico Universitario A Gemelli
Roma, 8, 00168
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165
Belgium3 sites
UZ Gent
Ghent, 9000
Universitaire Ziekenhuizen Leuven
Leuven
Institut de Myologie
Liège, Liege, 4000
France3 sites
Hopital Armand Trosseau
Paris, 75012
Hôpitaux Universitaires de Strasbourg
Strasbourg, Bas-Rhin, 67098
Hôpital Des Enfants
Toulouse, Haute-Garonne, 31059
Canada2 sites
Alberta Children's Hospital
Calgary, Alberta, T3B6A8
London Health Sciences Centre - Hospital
London, Ontario, N6A 5W9
United Kingdom2 sites
Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX
Great Ormond Street Hospital (GOSH)
London, WC1N EH
About this study
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)
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
- Being tested
- WVE-210201 (suvodirsen), Placebo
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WVE-210201 (suvodirsen)Drug
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
PlaceboDrug
Buffered saline solution
| Study group | Role | What they receive |
|---|---|---|
| WVE-210201 (3 mg/kg) | Experimental | Weekly IV administrations of WVE-210210 at 3 mg/kg Receives: Drug: WVE-210201 (suvodirsen) |
| WVE-210201 (4.5 mg/kg) | Experimental | Weekly IV administrations of WVE-210210 at 4.5 mg/kg Receives: Drug: WVE-210201 (suvodirsen) |
| Placebo | Placebo comparator | Weekly IV administrations of phosphate buffered saline solution visually identical in appearance to WVE-21021 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Dystrophin Level (% Normal Dystrophin)
US/other regions (as applicable)
Measured: Day 1 to Week 12, Week 22, or Week 46
Change From Baseline in North Star Ambulatory Assessment (NSAA)
European Union (EU)/other regions (as applicable)
Measured: Day 1 through Week 48
Secondary outcomes (8)
Change From Baseline in North Star Ambulatory Assessment (NSAA)
US/other regions (as applicable)
Measured: Day 1 through Week 48
Change From Baseline in Dystrophin Level (% Normal Dystrophin)
European Union (EU)/other regions (as applicable)
Measured: Day 1 to Week 12, Week 22, or Week 46
Change From Baseline in Upper Limb Proximal Strength
Measured: Day 1 through Week 48
Change From Baseline in 4-stair Climb
Measured: Day 1 through Week 48
Change From Baseline in the 10-meter Walk/Run Test
Measured: Day 1 through Week 48
Change From Baseline in Forced Vital Capacity
Measured: Day 1 through Week 48
Change From Baseline in the 95th Percentile of Stride Velocity
Measured: Day 1 through Week 48
Change From Baseline in NSAA
Long-term evaluation, open label from Week 48 through Week 96
Measured: Day 1 through Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2019
- Study start4 Sep 2019
- Primary completion16 Dec 2019
- Study completion9 Jan 2020
Why it stopped: Lack of efficacy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael A Panzara, MD, MPH, Study director, Wave Life Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03907072
- Sponsor's protocol ID
- WVE-DMDX51-003
- Acronym
- DYSTANCE 51
- Status last verified
- Apr 2021
- First submitted
- 5 Apr 2019
- Last updated
- 20 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03907072
- Acronym / sponsor ID
- DYSTANCE 51 / WVE-DMDX51-003
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of efficacy
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Wave Life Sciences USA, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Sep 2019 (actual)
- Primary completion
- 16 Dec 2019 (actual)
- Study completion
- 9 Jan 2020 (actual)
- First posted
- 8 Apr 2019 (actual)
- Results
- Posted 20 May 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| WVE-210201 (3 mg/kg) | Experimental | Drug: WVE-210201 (suvodirsen)Weekly IV administrations of WVE-210210 at 3 mg/kg |
| WVE-210201 (4.5 mg/kg) | Experimental | Drug: WVE-210201 (suvodirsen)Weekly IV administrations of WVE-210210 at 4.5 mg/kg |
| Placebo | Placebo comparator | Drug: PlaceboWeekly IV administrations of phosphate buffered saline solution visually identical in appearance to WVE-21021 |
Who may qualify
- Ages
- 5 to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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)
- Stable pulmonary and cardiac function, as measured by:
- 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
Key exclusion criteria (9 listed)
- Cardiac insufficiency:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WVE-210201 (suvodirsen)WVE-210201 is a stereopure antisense oligonucleotide (ASO) | Drug | WVE-210201 (3 mg/kg), WVE-210201 (4.5 mg/kg) |
| PlaceboBuffered saline solution | Drug | Placebo |
Primary outcome
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)
Change From Baseline in North Star Ambulatory Assessment (NSAA)
Time frame: Day 1 through Week 48
European Union (EU)/other regions (as applicable)
8 secondary outcomes
Change From Baseline in North Star Ambulatory Assessment (NSAA)
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 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 10-meter Walk/Run Test
Time frame: Day 1 through Week 48
Change From Baseline in Forced Vital Capacity
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael A Panzara, MD, MPH study director
Wave Life Sciences
22 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing WVE-210201 (suvodirsen) in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 22 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Italy, Belgium, France, Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Dystrophin Level (% Normal Dystrophin) (time frame: Day 1 to Week 12, Week 22, or Week 46). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2021.