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    Summary
    EudraCT Number:2016-005142-39
    Sponsor's Protocol Code Number:WVE-HDSNP2-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-05-19
    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 number2016-005142-39
    A.3Full title of the trial
    A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120102 Administered Intrathecally in Patients with Huntington’s Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120102 Administered Intrathecally in Patients with Huntington’s Disease
    A.4.1Sponsor's protocol code numberWVE-HDSNP2-001
    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 pointFabienne Bouquet
    B.5.3 Address:
    B.5.3.1Street Address27-35 rue Victor Hugo
    B.5.3.2Town/ cityIvry-sur-Seine Cedex
    B.5.3.3Post code94853
    B.5.3.4CountryFrance
    B.5.4Telephone number+3361916 77 22
    B.5.6E-mailfabienne.bouquet@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-120102
    D.3.2Product code WVE-120102
    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 INNWVE-120102
    D.3.9.2Current sponsor codeWVE-120102
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    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 injection
    D.8.4Route of administration of the placeboIntrathecal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Huntington’s Disease
    E.1.1.1Medical condition in easily understood language
    Huntington’s Disease
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    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
    Primary objective:
    • Evaluate the safety and tolerability of WVE-120102 in patients with early manifest HD.
    E.2.2Secondary objectives of the trial
    -Characterize the pharmacokinetics (PK) of WVE-120102 in plasma.
    -Characterize the exposure of WVE-120102 in cerebrospinal fluid (CSF).
    -Assess the pharmacodynamic (PD) effect of WVE-120102 on levels of mutant huntingtin protein (mHTT) in CSF.
    -Assess the effect of WVE-120102 on signs and symptoms of HD, as measured by the Total Functional Capacity (TFC), administered as part of the Unified Huntington’s Disease Rating Scale (UHDRS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented ability to understand the written study ICF(s), and has provided signed written informed consent prior to any study procedures.
    2. Ambulatory male or female.
    3. Age ≥25 to ≤65 years old.
    4. Body mass index (BMI) ≤32 kg/m2.
    5. Documented CAG triplet repeats ≥36 in the Huntingtin gene.
    6. Documented heterozygosity for SNP2.
    7. Documented presence of the T variant of SNP2 on the same allele as the pathogenic CAG expansion
    8. Has clinical diagnostic motor features of HD, defined as UHDRS Diagnostic Confidence Score = 4.
    9. Stage I or Stage II HD, defined as UHDRS Total Functional Capacity scores ≥7 and ≤13.
    10. In the opinion of the Investigator, the patient is able to tolerate all study procedures, and is willing to comply with all other protocol requirements.
    11. Willingness to practice highly effective contraception for the duration of the study if patients or their partners are of childbearing potential. Non-childbearing potential and highly effective methods of contraception will be defined in the protocol.
    E.4Principal exclusion criteria
    1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
    2. Positive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
    3. Known to be positive for human immunodeficiency virus (HIV).
    4. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
    5. Received an investigational drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
    6. Implantable central nervous system (CNS) device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan.
    7. Diagnostic and Statistical Manual of Mental Disorders 5th Edition (DSM-5) diagnosis at the Screening Visit of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening Visit.
    8. Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit.
    9. Started or changed dose for concomitant medication for the treatment of HD symptoms or psychiatric disorders within 30 days prior to the Screening Visit (concomitant medications that have been administered on a stable regimen for ≥30 days are permitted).
    10. Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study.
    11. Clinically significant laboratory abnormality at Screening, including, but not limited to:
    a Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values at Screening or Baseline >3 times the upper limit of normal (ULN).
    b Renal insufficiency, defined as either as serum creatinine >1.8 mg/dL or creatinine clearance <40 mL/min.
    12. Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) ≥450 msec for males or ≥470 msec for females.
    13. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary,
    gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in
    the opinion of the Investigator, precludes the patient’s safe participation in the study or
    would interfere with the study assessments.
    14. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or
    unsuccessful lumbar puncture.
    15. Inability to undergo brain MRI (with or without sedation).
    16. Deemed to be at significant risk for suicidal behavior based on:
    a The opinion of the Investigator; or
    b Answers “yes” to Actual Suicide Attempts or Suicidal Behaviors in the Suicidal
    Behaviors section of the Columbia Suicide Severity Rating Scale (C-SSRS) with
    reference to a 2-year period prior to the Screening Visit; or
    c Answers “yes” on any items in the Suicidal Ideation section of the C-SSRS with
    reference to a 6-month period prior to the Screening Visit; or
    d Answers “yes” on any items in the Suicidal Ideation section of the C-SSRS at the
    Baseline Visit since the last visit (Screening Visit).
    17. Involved directly or indirectly 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)
    The primary endpoint is the safety and tolerability of WVE-120102, as compared with placebo, as assessed by the number of patients with adverse events (AEs), severity of AEs, number of patients with serious AEs (SAEs), and the number of patients who withdraw due to AEs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary safety endpoints will be assessed as incidence of events from baseline through end of study
    E.5.2Secondary end point(s)
    Secondary endpoints include:

    Pharmacokinetics (PK):
    -Pharmacokinetic parameters of WVE-120102 in plasma at predefined time points.
    -Exposure of WVE-120102 in CSF at predefined time points.

    Pharmacodynamics (PD):
    -Concentration of mHTT protein in CSF predose (baseline value) and at last measured time point

    Clinical Effects:
    -Change from baseline value to last measured time point) and difference from placebo in the TFC, administered as part of the UHDRS.

    Exploratory Endpoints
    • Change from baseline baseline value to last measured time point and difference
    from placebo in the motor, cognitive, behavioral, independence, and functional
    assessments administered as part of the UHDRS.
    • Change from baseline value to last measured time point and difference
    from placebo in the PBA-s.
    • Changes from baseline MRI of the brain (Screening to last measured time point)
    E.5.2.1Timepoint(s) of evaluation of this end point
    PK: At study time points from baseline value to last measured time point
    PD: At study time points from baseline value to last measured time point
    Clinical effects: At study timepoints from baseline value to last measured time point
    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 Yes
    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 Yes
    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) No
    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 Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    Denmark
    France
    Germany
    Poland
    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
    The end of the study is defined as the date on which the last patient completes the last visit (includes 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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 80
    F.1.3Elderly (>=65 years) No
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 80
    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)
    Following completion of this study, patients may be eligible to participate in an open-label extension study.
    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 Decision2017-07-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-02
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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