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    Summary
    EudraCT Number:2018-004009-22
    Sponsor's Protocol Code Number:WVE-DMDX51-003
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-03-28
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-004009-22
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy
    A.4.1Sponsor's protocol code numberWVE-DMDX51-003
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorWave Life Sciences UK Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportWave Life Sciences Ltd.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPPD
    B.5.2Functional name of contact pointAdrian Haines
    B.5.3 Address:
    B.5.3.1Street Address1-2 Crown Walk, Jewry Street
    B.5.3.2Town/ cityWinchester, Hampshire
    B.5.3.3Post codeSO23 8BB
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441223 771251
    B.5.6E-mailAdrian.Haines@ppdi.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2051 - EMA/OD/032/18
    D.3 Description of the IMP
    D.3.1Product nameWVE-210201
    D.3.2Product code WVE-210201
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNWVE-210201
    D.3.9.1CAS number 2142024-01-7
    D.3.9.2Current sponsor codeWVE-210201
    D.3.9.3Other descriptive namesuvodirsen
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne muscular dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Double-blind Treatment Period
    1. To evaluate the efficacy of WVE-210201 by assessing changes in dystrophin levels (United States/Other regions, as applicable)
    2. To evaluate the efficacy of WVE-210201 by assessing changes in motor function by North Star Ambulatory Assessment (NSAA) (European Union [EU]/Japan)

    Open-label Treatment Period
    To evaluate the long-term efficacy, safety and PK of WVE-210201
    E.2.2Secondary objectives of the trial
    Double-blind Treatment Period
    1. To evaluate the efficacy of WVE-210201 by assessing changes in upper limb proximal strength
    2. To evaluate the efficacy of WVE-210201 by assessing changes in lower limb motor function
    3. To evaluate the efficacy of WVE-210201 by assessing changes in respiratory function
    4. To evaluate the efficacy of WVE-210201 by assessing changes in stride velocity
    5. To evaluate the safety of WVE-210201
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Magnetic Resonance Imaging (MRI) Sub-study
    A subset of patients at selected sites may be asked to participate in an optional imaging sub-study that will assess cardiac and muscle parameters by MRI. The details will be provided via a separate protocol and patients participating in that study will be consented specifically for the imaging study.
    E.3Principal inclusion criteria
    1. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent prior to any study-related procedures.
    2. Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
    3. Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping.
    4. 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)
    5. Age of ≥5 and ≤12 years at time of randomization
    6. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including undergoing the biopsy procedures.
    7. Stable pulmonary and cardiac function, as measured by:
    a. Reproducible percent predicted forced vital capacity (FVC) ≥50%
    b. 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.
    8. Currently on a stable corticosteroid therapy regimen, defined as: initiation of systemic corticosteroid therapy occurred ≥6 months prior to Screening, and no changes in dose ≤3 months prior to Screening visit.
    9. Adequate deltoid muscle at Screening to perform open muscle biopsies
    10. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.
    11. Patient and caregivers must agree not to post any study-related information on social media.
    E.4Principal exclusion criteria
    1. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
    2. Other prior or ongoing medical conditions including:
    a. Acute illness within 4 weeks of the initial Screening visit;
    b. Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD
    3. Laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results. These include, but are not limited to:
    a. Renal insufficiency;
    b. Impaired hepatic function glutamate dehydrogenase (GLDH) ≥ 2.5x upper limit of normal (ULN) and bilirubin ≥ 2x ULN (or International Normalized Ratio [INR] ≥ 1.5x ULN);
    c. Activated partial thromboplastin time (aPTT) values above ULN;
    d. Platelet count < lower limit of normal (LLN).
    4. Documented positive hepatitis B surface antigen or hepatitis C antibody test.
    5. Known to be positive for human immunodeficiency virus (HIV).
    6. Severe mental retardation and/or behavioral problems that, in the opinion of the Investigator, could prohibit participation in this study.
    7. Cardiac insufficiency:
    a. Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study; however, cardiomyopathy that is managed by ACE inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criterion
    b. Any other evidence of clinically significant structural or functional heart abnormality
    c. A cardiac troponin I value > 0.2 ng/mL on initial and repeat testing if initial test is elevated at screening.
    8. 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.
    9. Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify (dose or regimen) during the study.
    10. Currently on anticoagulants or drugs that are known to significantly increase the risk of bleeding, such as NSAIDs and heparin.
    11. Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO).
    12. Received prior treatment with WVE-210201.
    13. Received prior treatment with gene therapy for DMD
    14. Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection.
    15. Received any investigational drug within 3 months or 5 half-lives, whichever is longer prior to the planned baseline biopsy collection.
    16. Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction such as changes in pulse, blood pressure, breathing function, etc.
    17. Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, sub-investigator, study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study.
    E.5 End points
    E.5.1Primary end point(s)
    Double-blind Treatment Period
    1. Change from baseline in dystrophin level (% normal dystrophin) assessed by Western blot of muscle tissue (United States/Other regions, as applicable)
    2. Change from baseline in NSAA at 48 weeks (EU/Japan)

    Open-label Treatment Period
    1. Long-term efficacy, safety, and PK of WVE-210201
    E.5.1.1Timepoint(s) of evaluation of this end point
    Double-blind Treatment Period
    1. 1 of 3 timepoints, week 12, week 22 or week 46
    2. 48 weeks

    Open-label Treatment Period
    1. 48 weeks
    E.5.2Secondary end point(s)
    Double-blind Treatment Period
    1. Key secondary endpoint - Change from baseline in NSAA at 48 weeks (United States/Other regions as applicable)
    2. Key secondary endpoint - Change from baseline dystrophin level (% normal dystrophin) assessed by Western blot of muscle tissue (EU/Japan)
    3. Change from baseline in upper limb proximal strength assessed by hand held myometry at 48 weeks
    4. Change from baseline in 4-stair climb (4SC) at 48 weeks
    5. Change from baseline in the 10-meter walk/run test at 48 weeks
    6. Change from baseline in FVC (% predicted) at 48 weeks
    7. Change from baseline in the 95th percentile of stride velocity at 48 weeks
    E.5.2.1Timepoint(s) of evaluation of this end point
    Double-blind Treatment Period
    1. 48 weeks
    2. 1 of 3 timepoints, week 12, week 22 or week 46
    3. 48 weeks
    4. 48 weeks
    5. 48 weeks
    6. 48 weeks
    7. 48 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    A Randomized, Double-blind, Placebo-controlled Study with Open-label Extension
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Canada
    Czech Republic
    France
    Germany
    Ireland
    Italy
    Japan
    Netherlands
    Poland
    Spain
    Sweden
    Turkey
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 150
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 125
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children aged ≥5 and ≤12 years at time of randomization.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 85
    F.4.2.2In the whole clinical trial 150
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After participation in the trial, if applicable, patients will be treated with the current standard therapy
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-05-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-01-09
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