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    Summary
    EudraCT Number:2019-003781-41
    Sponsor's Protocol Code Number:CLTW888A11301
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2026-11-02
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2019-003781-41
    A.3Full title of the trial
    An open-label, single-arm study to provide efficacy and safety data of voretigene neparvovec administered assubretinal injection in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of efficacy and safety of voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy
    A.4.1Sponsor's protocol code numberCLTW888A11301
    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 SponsorNovartis Pharma K.K
    B.1.3.4CountryJapan
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.6E-mailclinicaltrial.enquiries@novartis.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 Yes
    D.2.1.1.1Trade name Luxturna
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Pharma K.K.
    D.2.1.2Country which granted the Marketing AuthorisationJapan
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/040/15
    D.3 Description of the IMP
    D.3.1Product nameVoretigene neparvovec
    D.3.2Product code LTW888
    D.3.4Pharmaceutical form Solution for injection in vial
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubretinal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVoretigene neparvovec
    D.3.9.1CAS number 1646819-03-5
    D.3.9.2Current sponsor codeLTW888
    D.3.9.4EV Substance CodeSUB182301
    D.3.10 Strength
    D.3.10.1Concentration unit Vector genome
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150000000000
    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 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
    Biallelic RPE65 mutation-associated retinal dystrophy
    E.1.1.1Medical condition in easily understood language
    retinal dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10038857
    E.1.2Term Retinal dystrophy
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the efficacy of voretigene neparvovec in Japanese participants.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of voretigene neparvovec in Japanese participants in terms of visual fields (VF) and visual acuity (VA) during the study period
    To evaluate the long term efficacy of voretigene neparvovec in Japanese participants
    To evaluate the safety of voretigene neparvovec during the study period
    To evaluate the vector shedding of voretigene neparvovec during the study period
    To evaluate the immunogenicity of voretigene neparvovec during the study period
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis-designated laboratory in Japan.

    Age four years or older.

    Visual acuity worse than 20/60 (both eyes) and/or visual field less than 20 degrees in any meridian as measured by a III4e isopter or equivalent (both eyes).

    Sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/ or ophthalmoscopy.

    Must have either:
    An area of retina within the posterior pole of > 100 μm thickness shown on OCT, or ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole, or remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent
    E.4Principal exclusion criteria
    Any prior participation in a study in which a gene therapy vector was administered.

    Participation in a clinical study with an investigational drug in the past 6 months from screening visit.

    Known hypersensitivity to any of the study treatments including excipients or to medications planned for use in the peri-operative period.

    Unable to reliably perform the FST assessment.

    Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme in the past 6 months from screening visit.

    Prior intraocular surgery within 6 months from screening visit.

    Prior use of any medicines that, in the opinion of the investigator, may have caused retinal damage (e.g., sildenafil or related compounds, hydroxychloroquine, chloroquine, thioridazine, any other retino-toxic compounds).

    Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery or interfere with the interpretation of study.

    Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline in full-field light sensitivity threshold (FST; white light, averaged over both eyes) at Day 30B, 90B, 180B, 270B and Year 1B.
    *"B" refers to the time points after the second eye injection. e.g. Year 1B means one year after second-eye injection.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 30B, 90B, 180B, 270B and Year 1B.
    *"B" refers to the time points after the second eye injection. e.g. Year 1B means one year after second-eye injection.
    E.5.2Secondary end point(s)
    Change from Baseline in the sum total degrees for VF, averaged over both eyes, as measured using Goldmann kinetic perimetry testing with a III4e target over time up to Year 5B.
    Change from Baseline in macular threshold, averaged over both eyes, as measured using Humphrey static VF testing over time up to Year 5B.
    Change from Baseline in VA (averaged over both eyes) over time up to Year 5B.
    Change from Baseline in FST (white light, averaged over both eyes) at Year 2B, 3B, 4B and 5B.
    Frequency of treatment-emergent adverse events and treatment-emergent serious adverse events over time up to Year 5B.
    Presence of vector in peripheral blood or collected tear over time up to Year 1B.
    Presence of systemic cell-mediated or humoral responses to capsid or transgene product over time up to Year 1B.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Over time up to Year 5B.
    Over time up to Year 1B.
    *"B" refers to the time points after the second eye injection. e.g. Year 1B means one year after second-eye injection.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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) No
    E.7.1.1First administration to humans No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Japan
    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
    Study completion is defined as when the last participant finishes their EOS visit and any repeat assessments associated with this visit have been documented and followed up appropriately by the Investigator or, in the event of an early study termination decision, the date of that decision.
    All patients should have a safety follow-up call conducted 30 days after the end of study visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years7
    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: 2
    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: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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 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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 4
    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)
    All patients should have a safety follow-up call conducted 30 days after the end of study visit.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Japan
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