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    Summary
    EudraCT Number:2022-002267-29
    Sponsor's Protocol Code Number:WVE-004-002
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2023-01-25
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2022-002267-29
    A.3Full title of the trial
    A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients with
    C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label Extension (OLE) Study of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
    A.4.1Sponsor's protocol code numberWVE-004-002
    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 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 UK Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace, Inc.
    B.5.2Functional name of contact pointRegulatory Submissions
    B.5.3 Address:
    B.5.3.1Street Address5375 Medpace Way
    B.5.3.2Town/ cityCincinnati
    B.5.3.3Post codeOH 45227
    B.5.3.4CountryUnited States
    B.5.4Telephone number0015135799911
    B.5.5Fax number0015135790444
    B.5.6E-mailregsubmissions@medpace.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.3.2Product code WVE-004
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet assigned
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeWVE-004
    D.3.9.3Other descriptive nameSynthetic stereopure antisense oligonucleotide targeting human C9orf72 hexanucleotide repeat-containing mRNA transcripts
    D.3.9.4EV Substance CodeSUB219364
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Yes
    D.3.11.13.1Other medicinal product typeantisense oligonucleotide
    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
    Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
    E.1.1.1Medical condition in easily understood language
    motor neuron disease; dementia where the front and sides of the brain
    has been affected
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10068968
    E.1.2Term Frontotemporal dementia
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of long-term treatment with WVE-004 in patients with ALS or FTD with a documented mutation in the C9orf72 gene
    E.2.2Secondary objectives of the trial
    To evaluate the clinical and pharmacodynamic (PD) effects of WVE-004 in patients with ALS or FTD with a documented mutation in the C9orf72 gene
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient has the ability and is willing to provide informed consent prior to any study procedures. In instances where signed written informed consent is unable to be obtained, it is acceptable for the patient to provide consent with legally authorized representative signing on the patient's behalf.
    2. Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.
    3. In the opinion of the Investigator, the patient is able to tolerate all study procedures, is willing to comply with all other protocol requirements, and tolerated study drug in the parent study.
    4. Patient is willing to practice highly effective contraception for the duration of the study and for 5 months after the last dose of study drug if the patient or their partner are of childbearing potential. Non-childbearing potential and highly effective methods of contraception are defined in the protocol (Section 5.2.1). In addition, willingness to forego sperm or ova (egg) donation for the duration of the study and 5 months after completion of the study.
    5. Patient has identified a study partner(s) for the duration of the study.
    E.4Principal exclusion criteria
    1. Patient has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the patient unsuitable for participation in and/or completion of the trial procedures.
    a. Prior or ongoing medical conditions, including acute illness, within 28 days of Screening visit;
    b. Clinically significant abnormality on laboratory testing at Screening, including but not limited to renal insufficiency, which is defined as creatinine clearance <40 mL/min.
    2. Patient has a positive hepatitis B surface antigen or hepatitis C antibody test.
    3. Patients who are pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening visit or plans to become pregnant during the trial.
    4. Patients deemed to be at significant risk for suicidal behavior based on Investigator assessment and/or active suicidal ideation.
    5. Patient has a bone, spine, bleeding (e.g., hemophilia, Von Willebrand disease, or liver disease), or other disorder that exposes the patient to a risk of injury or unsuccessful LP.
    6. Patient received prior treatment with viral or cellular-based gene therapy.
    7. Patient received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Patient received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer.
    8. Patient anticipates using antiplatelet or anticoagulant therapy during the course of the study. Patients who received antiplatelet or anticoagulant therapy must complete one of the following washout periods before the Screening visit:
    a. A 7-day washout period for antiplatelet therapy,
    b. A 1-day washout period for anticoagulants (except warfarin), or
    c. A 5-day washout period for warfarin.
    9. Patient was noncompliant in the opinion of the Investigator or Sponsor when participating in study WVE-004-001.
    10. Patient is directly or indirectly involved in the conduct and administration of this trial as an Investigator, sub-investigator, trial coordinator, or other trial 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 trial.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of patients with adverse events (AEs), severe AEs, serious adverse events (SAEs), withdrawals due to AEs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 1-120
    E.5.2Secondary end point(s)
    Clinical Assessments:
    Change from baseline in:
    o Clinical Dementia Rating plus National Alzheimer’s Coordinating Center Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD)
    o ALS Functional Rating Scale-Revised (ALSFRS-R)
    o Handheld dynamometry (HHD)
    o Pulmonary function testing (forced vital capacity [FVC])
    o Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ) 5

    Pharmacodynamic Effects:
    • Change from baseline in concentration of poly-lycine-proline (poly-GP) levels in the cerebrospinal fluid (CSF)
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 1-120
    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 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) 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
    E.7.2Therapeutic exploratory (Phase II) Yes
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Canada
    New Zealand
    Netherlands
    Belgium
    Ireland
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    Last patient Last Visit ( Including Follow-up Visit)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 31
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 11
    F.2 Gender
    F.2.1Female Yes
    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 Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 42
    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)
    None
    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 Decision2023-03-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-06-30
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