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    Summary
    EudraCT Number:2020-000739-28
    Sponsor's Protocol Code Number:CT-AMT-060-04
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2020-11-27
    Trial 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 number2020-000739-28
    A.3Full title of the trial
    A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/IIb extension study assessing the long-term safety and efficacy of a gene therapy (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
    A.3.2Name or abbreviated title of the trial where available
    Phase I/IIb extension study of AAV5-hFIX in severe or moderately severe haemophilia B
    A.4.1Sponsor's protocol code numberCT-AMT-060-04
    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 SponsoruniQure biopharma B.V.
    B.1.3.4CountryNetherlands
    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 supportuniQure biopharma B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationuniQure biopharma B.V.
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressPaasheuvelweg 25A
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105 BP
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31020240 6154 |
    B.5.6E-mailE.Destree@uniqure.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/3/11/938
    D.3 Description of the IMP
    D.3.1Product nameAAV5-hFIX
    D.3.2Product code AMT-060
    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.3.9.2Current sponsor codeAMT-060
    D.3.9.3Other descriptive nameAAV5-hFIX
    D.3.9.4EV Substance CodeSUB167020
    D.3.10 Strength
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number1 x 10e13
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Haemophilia B
    E.1.1.1Medical condition in easily understood language
    Haemophilia B - Bleeding disorder
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B..
    E.2.2Secondary objectives of the trial
    To assess the long-term efficacy of a systemic administration of AAV5-hFIX, an adeno-associated viral (AAV) vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult patients with severe or moderately severe haemophilia B.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients with congenital hemophilia B who completed Study CT-AMT-060-01

    2. Able to provide informed consent following receipt of verbal and written information about the trial.
    E.4Principal exclusion criteria
    Enrolled patients will have already been assessed based on the exclusion criteria for Study CT-AMT-060-01.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of AAV5-hFIX.

    Primary safety endpoints include the following:
    - AEs possibly or probably related to previous AAV5-hFIX administration
    - Neutralizing FIX antibodies (FIX inhibitors)
    - ALT/AST levels
    - Liver pathology (assessed by ultrasound)
    - Alpha-fetoprotein (AFP)
    E.5.1.1Timepoint(s) of evaluation of this end point
    The individual subjects will be followed for an additional five years, 6-10 years after dosing of AAV5-hFIX.
    The occurrence of adverse events (AEs) related to previous AAV5-hFIX administration in patients will be continuously monitored.
    ALT/AST will be measuredevery 6 months
    Abdominal ultrasounds will be conducted at study entry (Visit 36) and study exit (Visit 45).
    Alpha-fetoprotein (AFP) and Anti-AAV5 antibodies will be measured every 12 months
    E.5.2Secondary end point(s)
    The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of AAV5-hFIX on FIX activity, overall FIX utilization, bleeding events requiring FIX replacement therapy, any procedures, and QoL.

    Secondary efficacy endpoints include the following:
    - Endogenous FIX activity
    - Utilization of FIX-replacement therapy
    - Annualized bleeding rate (FIX-requiring); including the following:
    o All bleeds
    o Spontaneous bleeds
    o Traumatic bleeds
    o Joint bleeds
    - Procedures (including major and minor surgeries)
    - Short form (SF-36) and EQ-5D-5L QoL scores
    - Hemophilia Joint Health Score (HJHS)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Endogenous FIX activity, Utilization of FIX-replacement therapy, FIX inhibitors and bleeding rates and procedures will be measured every 6 months

    QoL and HJHS will be measured every 12 months
    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 No
    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) 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
    Extension study to assess long term safety and efficacy of previously administered treatment
    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.8.2.4Number 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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: 9
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 10
    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 Decision2020-08-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-13
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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