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    Summary
    EudraCT Number:2020-000963-22
    Sponsor's Protocol Code Number:VTX-801_CLN_001
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-03-10
    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 ConcernedGermany - PEI
    A.2EudraCT number2020-000963-22
    A.3Full title of the trial
    A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson’s Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial: treatment of adult Wilson's Disease patients with gene therapy
    A.3.2Name or abbreviated title of the trial where available
    GATEWAY clinical trial
    A.4.1Sponsor's protocol code numberVTX-801_CLN_001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04537377
    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 SponsorVivet Therapeutics SAS
    B.1.3.4CountryFrance
    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 supportVivet Therapeutics SAS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAixial Group UK Ltd
    B.5.2Functional name of contact pointProject Lead
    B.5.3 Address:
    B.5.3.1Street AddressAshurst, Broadlands Business Campus, Langhurstwood Road
    B.5.3.2Town/ cityHorsham
    B.5.3.3Post codeRH12 4QP
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailCROinfo@aixial.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/3/20/2321 - EMA/ODD/0000034624
    D.3 Description of the IMP
    D.3.1Product nameN/A
    D.3.2Product code VTX-801
    D.3.4Pharmaceutical form 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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeVTX-801
    D.3.9.3Other descriptive nameAdeno-associated viral vector serotype 3B encoding shortened human ATP7B
    D.3.9.4EV Substance CodeSUB218423
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1E13
    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 Yes
    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
    Wilson's Disease
    E.1.1.1Medical condition in easily understood language
    Wilson’s Disease is a rare genetic disorder. It causes excess copper to build up in the body, particularly in the liver and brain.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10047988
    E.1.2Term Wilson'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
    Assessing, for up to 5 years, the safety and tolerability of single ascending doses of VTX-801 administered intravenously (IV) to adult patients with Wilson’s Disease prior to and following background WD therapy withdrawal.
    E.2.2Secondary objectives of the trial
    •Exploring VTX-801 pharmacodynamics and efficacy
    •Assessing humoral and cellular immune responses to VTX-801
    •Providing data to support VTX-801 dose level selection
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female aged 18-65 years old (inclusive)
    2. Confirmed diagnosis of WD
    3. Treated for WD according to international recommendations with no current evidence for inadequate treatment (EASL, 2012; Roberts et al, 2008)
    4. Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder, and (ii) stable laboratory parameters used to assess copper metabolism including 24-hour urinary copper, free serum copper such as NCC or CuEXC (when available), as well as liver enzymes, hemoglobin, and white blood cell count
    E.4Principal exclusion criteria
    1. ALT level ≥ 2 x ULN that is not readily explained by extrinsic factors (e.g., strenuous exercise, medication use)
    2. Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's
    syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
    3. Platelet count < 120.000 / µL
    4. Any signs of liver cirrhosis decompensation, including gastrointestinal
    bleed within 6 months (24 weeks) prior to screening/enrolment visit
    5. Patient has moderate or severe renal impairment or patient has
    nephritis or nephrotic syndrome
    6. Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
    7. Any history or current evidence of hepatitis B infection
    8. Any history of hepatitis C infection, unless previous viral RNA assays
    in two samples, collected at least 6 months apart, are negative
    9. Positive QuantiFERON®-TB Gold tuberculosis test result
    10. Any concomitant disorder/condition - including hepatic disorders - or
    treatment possibly interfering with the conduct or evaluation of the
    study, according to the Investigator
    11. Pregnancy or breastfeeding
    12. Body Mass Index ≥ 35 kg/m2
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability profile, including treatment-emergent adverse events (TEAE).
    AEs will be summarized based on the date of onset for the event.
    Number of treatment-emergent AEs will be provided by SOC and PT, by dose cohort and overall.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The safety of the study intervention will be assessed through the
    recording, reporting and analyzing of baseline medical conditions, AEs,
    general physical examination, laboratory tests, and vital signs data.

    End points will be assessed over the course of the study from baseline to Month 60.
    All SAEs and VTX-801-related AEs linked to copper deficiency and WD deterioration.
    E.5.2Secondary end point(s)
    1. Free serum Cu
    2. Total serum Cu
    3. 24-hour urinary Cu
    4. Serum ceruloplasmin activity (enzymatic assay)
    5. VTX-801 Responder status
    6. Immune response to VTX-801
    E.5.2.1Timepoint(s) of evaluation of this end point
    Serum / urinary copper and ceruloplasmin activity will be assessed over the course of the study from baseline to Month 60.

    The Responder status will be assessd at Week 12 and Week 36 following an IV administration.
    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 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 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 EEA3
    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
    United Kingdom
    United States
    Denmark
    Germany
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years12
    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 years12
    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: 16
    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 8
    F.4.2.2In the whole clinical trial 16
    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)
    It is a single dose infusion trial. Gene therapy remains active after the trial.
    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 Decision2021-10-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-05-05
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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