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TerminatedNCT03907072DYSTANCE 51Updated May 20, 2021Results posted

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

A Phase 2/3 interventional study of WVE-210201 (suvodirsen) and Placebo in Duchenne Muscular Dystrophy, sponsored by Wave Life Sciences Ltd.. Terminated at 22 sites in 8 countries. Open to male participants aged 5 Years to 12 Years. Per ClinicalTrials.gov, last updated 2021-05-20.

Sponsored by Wave Life Sciences Ltd. · Phase 2/3, Interventional, and Treatment

Why this study was terminated
Lack of efficacy
Phase
Phase 2/3
Study type
Interventional
Enrollment
6
Allocation
Randomized
Ages
5 Years to 12 Years
Sex
Male
01

Study 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)

02

Conditions studied

  • Duchenne Muscular Dystrophy
03

In context

Muscular Dystrophies

548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.

This study's enrollment of 6 is below the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.

Browse Muscular Dystrophies studies →

Lead sponsor

Wave Life Sciences Ltd. is the lead sponsor of 5 studies on the registry; none are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years to 12 Years
Sexes eligible
Male
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
6 participants (actual)

Study arms

  • Experimental
    WVE-210201 (3 mg/kg)

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

    Drug: WVE-210201 (suvodirsen)

  • Experimental
    WVE-210201 (4.5 mg/kg)

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

    Drug: WVE-210201 (suvodirsen)

  • Placebo comparator
    Placebo

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

    Drug: Placebo

Interventions

  • DrugWVE-210201 (suvodirsen)

    WVE-210201 is a stereopure antisense oligonucleotide (ASO)

  • DrugPlacebo

    Buffered saline solution

06

What researchers measure

Primary outcomes

  1. Change From Baseline in Dystrophin Level (% Normal Dystrophin)

    US/other regions (as applicable)

    Time frame: Day 1 to Week 12, Week 22, or Week 46

  2. Change From Baseline in North Star Ambulatory Assessment (NSAA)

    European Union (EU)/other regions (as applicable)

    Time frame: Day 1 through Week 48

Secondary outcomes

  1. Change From Baseline in North Star Ambulatory Assessment (NSAA)

    US/other regions (as applicable)

    Time frame: Day 1 through Week 48

  2. Change From Baseline in Dystrophin Level (% Normal Dystrophin)

    European Union (EU)/other regions (as applicable)

    Time frame: Day 1 to Week 12, Week 22, or Week 46

  3. Change From Baseline in Upper Limb Proximal Strength

    Time frame: Day 1 through Week 48

  4. Change From Baseline in 4-stair Climb

    Time frame: Day 1 through Week 48

  5. Change From Baseline in the 10-meter Walk/Run Test

    Time frame: Day 1 through Week 48

  6. Change From Baseline in Forced Vital Capacity

    Time frame: Day 1 through Week 48

  7. Change From Baseline in the 95th Percentile of Stride Velocity

    Time frame: Day 1 through Week 48

  8. Change From Baseline in NSAA

    Long-term evaluation, open label from Week 48 through Week 96

    Time frame: Day 1 through Week 96

07

Results

Posted May 20, 2021

Participant flow

Participant flow — Overall Study
MilestoneWVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)Placebo
Started222
Completed000
Not completed222
Withdrew: Study terminated by sponsor222

Outcome measures

PrimaryChange From Baseline in Dystrophin Level (% Normal Dystrophin)

US/other regions (as applicable)

Time frame:
Day 1 to Week 12, Week 22, or Week 46

No measurements were reported for this outcome.

PrimaryChange From Baseline in North Star Ambulatory Assessment (NSAA)

European Union (EU)/other regions (as applicable)

Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in North Star Ambulatory Assessment (NSAA)

US/other regions (as applicable)

Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in Dystrophin Level (% Normal Dystrophin)

European Union (EU)/other regions (as applicable)

Time frame:
Day 1 to Week 12, Week 22, or Week 46

No measurements were reported for this outcome.

SecondaryChange From Baseline in Upper Limb Proximal Strength
Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in 4-stair Climb
Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in the 10-meter Walk/Run Test
Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in Forced Vital Capacity
Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in the 95th Percentile of Stride Velocity
Time frame:
Day 1 through Week 48

No measurements were reported for this outcome.

SecondaryChange From Baseline in NSAA

Long-term evaluation, open label from Week 48 through Week 96

Time frame:
Day 1 through Week 96

No measurements were reported for this outcome.

Adverse events

Collected over Study enrollment through early study termination by Sponsor (maximum of 11 weeks).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
WVE-210201 (3 mg/kg)0/2 (0%)0/2 (0%)2/2 (100%)
WVE-210201 (4.5 mg/kg)0/2 (0%)1/2 (50%)2/2 (100%)
Placebo0/2 (0%)0/2 (0%)2/2 (100%)
Most frequent serious events
Most frequent serious events
EventWVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)Placebo
Cardiac DisorderCardiac disorders0/21/20/2
Most frequent other events
Most frequent other events
EventWVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)Placebo
PyrexiaGeneral disorders1/22/20/2
Bone contusionInjury, poisoning and procedural complications0/20/21/2
C-reactive protein increasedInvestigations0/21/20/2
HeadacheNervous system disorders0/21/20/2
DiarrhoeaGastrointestinal disorders1/21/20/2
VomitingGastrointestinal disorders0/21/20/2
GastroenteritisInfections and infestations0/20/21/2
NasopharyngitisInfections and infestations1/21/20/2
Oral herpesInfections and infestations0/21/20/2

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)WVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)PlaceboTotal
<=18 years2226
Between 18 and 65 years0000
>=65 years0000
Age, Continuous
Age, Continuous(Years)WVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)PlaceboTotal
Mean6.5 (5 to 8)9 (8 to 10)7 (5 to 9)7.5 (5 to 10)
Sex: Female, Male
Sex: Female, Male(Participants)WVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)PlaceboTotal
Female0000
Male2226
Race (NIH/OMB)
Race (NIH/OMB)(Participants)WVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)PlaceboTotal
American Indian or Alaska Native0000
Asian0000
Native Hawaiian or Other Pacific Islander0000
Black or African American0000
White1225
More than one race0000
Unknown or Not Reported1001
Region of Enrollment
Region of Enrollment(participants)WVE-210201 (3 mg/kg)WVE-210201 (4.5 mg/kg)PlaceboTotal
Sweden0101
Belgium1001
Italy0022
France1102
08

Study locations

22 sites
  • Yale University
    New Haven, Connecticut 06510, United States
  • Rare Disease Research, LLC.
    Atlanta, Georgia 30318, United States
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
  • Kennedy Krieger Institute
    Baltimore, Maryland 21205, United States
  • University of Massachusetts
    Worcester, Massachusetts 01605, United States
  • Children's Hospital of Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • Institut de Myologie
    Liège, Liege 4000, Belgium
  • UZ Gent
    Gent, 9000, Belgium
  • Universitaire Ziekenhuizen Leuven
    Leuven, Belgium
  • Alberta Children's Hospital
    Calgary, Alberta T3B6A8, Canada
  • London Health Sciences Centre - Hospital
    London, Ontario N6A 5W9, Canada
  • Fakultni Nemocnice v Motole
    Praha 5, 15006, Czechia
  • Hôpitaux Universitaires de Strasbourg
    Strasbourg, Bas-Rhin 67098, France
  • Hôpital Des Enfants
    Toulouse, Haute-Garonne 31059, France
  • Hopital Armand Trosseau
    Paris, 75012, France
  • Ospedale Pediatrico Bambino Gesù
    Roma, Lazio 165, Italy
  • U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
    Messina, 98125, Italy
  • Ospedale San Reffaele Via Olgettina, 60
    Milano, 20132, Italy
  • Fondazione Policlinico Universitario A Gemelli
    Roma, 8, 00168, Italy
  • Drottning Silvias Barn Och Ungdomssjukhus
    Göteborg, 41650, Sweden
  • Leeds Teaching Hospitals NHS Trust
    Leeds, LS1 3EX, United Kingdom
  • Great Ormond Street Hospital (GOSH)
    London, WC1N EH, United Kingdom
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Jul 9, 2019

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 20, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03907072
Lead sponsor
Wave Life Sciences Ltd.
Responsible party
Sponsor
First posted
Apr 8, 2019
Start date
Sep 4, 2019
Primary completion
Dec 16, 2019
Completion
Jan 9, 2020
Results posted
May 20, 2021
Last update
May 20, 2021

Study contacts

Michael A Panzara, MD, MPH
study director · Wave Life Sciences

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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