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    Summary
    EudraCT Number:2018-000975-34
    Sponsor's Protocol Code Number:WVE-DMDX51-002
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-08-16
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2018-000975-34
    A.3Full title of the trial
    A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
    A.4.1Sponsor's protocol code numberWVE-DMDX51-002
    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 Ltd.
    B.1.3.4CountryUnited States
    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 pointBruno Rocton
    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+441932 563 375
    B.5.6E-mailbruno.rocton@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameWVE-210201
    D.3.2Product code WVE-210201
    D.3.4Pharmaceutical form 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 21420224017
    D.3.9.2Current sponsor codeWVE-210201
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of WVE-210201
    E.2.2Secondary objectives of the trial
    To evaluate the effect of WVE-210201 treatment on dystrophin production
    To evaluate the concentration of WVE-210201 in plasma following treatment with WVE-210201
    To evaluate the concentration of WVE-210201 in urine following treatment with WVE-210201
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent/minor assent prior to any study-related procedures.
    2. Patient successfully completed the Phase 1 study with WVE-210201, WVE-DMDX51-001.
    3. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures.
    4. 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.
    5. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.
    6. 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 unable to complete the study procedures.
    2. Other prior or ongoing medical conditions including:
    a. Acute illness within Screening period;
    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 as measured by glutamate dehydrogenase (GLDH) ≥ 2.5x upper limit of normal (ULN) and Bilirubin ≥ 2x ULN (or INR ≥ 1.5x ULN;
    c. Activated partial thromboplastin time [aPTT] values above the ULN;
    d. Platelet count less than lower limit of normal (LLN).
    e. Any evidence of clinically significant structural or functional heart abnormality would prohibit participation in this study.
    f. Troponin I value above 2x ULN
    4. 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)
    Safety and tolerability of WVE-210201 as assessed by the number of patients with adverse events (AEs), severity of AEs, number of patients with serious adverse events (SAEs), and the number of patients who withdraw due to AEs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During study treatment - week 1 to week 13
    E.5.2Secondary end point(s)
    1. Dystrophin level assessed by Western blot of muscle biopsy
    2. Concentration of WVE-210201 in plasma at predefined time points
    3. Concentration of WVE-210201 in urine at predefined time points
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Dystrophin level assessed by Western blot of muscle biopsy - wk1, wk14
    2. Concentration of WVE-210201 in plasma at predefined time points - wk1, wk5
    3. Concentration of WVE-210201 in urine at predefined time points - wk1, wk2, wk5, wk6-13
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVEDMDX51-001
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Belgium
    Canada
    France
    Italy
    Netherlands
    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 years0
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months7
    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: 40
    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: 24
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 16
    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 ≤18 years at randomization in the WVE-DMDX51-001 study
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 40
    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 Decision2018-08-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-01-20
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