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    Summary
    EudraCT Number:2021-003253-36
    Sponsor's Protocol Code Number:LBS-008-CT03
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-12-20
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-003253-36
    A.3Full title of the trial
    Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3 Study of Tinlarebant in the Treatment in Adolescent Subjects
    A.4.1Sponsor's protocol code numberLBS-008-CT03
    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 SponsorBelite Bio, Inc
    B.1.3.4CountryCayman Islands
    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 supportBelite Bio, Inc
    B.4.2CountryCayman Islands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBelite Bio, Inc
    B.5.2Functional name of contact pointNathan Mata
    B.5.3 Address:
    B.5.3.1Street AddressPO Box 309, Ugland House
    B.5.3.2Town/ cityGrand Cayman
    B.5.3.3Post codeKY1 1104
    B.5.3.4CountryCayman Islands
    B.5.6E-mailnmata@belitebio.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/3/18/2014
    D.3 Description of the IMP
    D.3.1Product nameTinlarebant
    D.3.2Product code LBS-008, BPN-14967
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTinlarebant
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeLBS-008, BPN-14967
    D.3.9.4EV Substance CodeSUB204010
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Stargardt Disease
    E.1.1.1Medical condition in easily understood language
    Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration by the first or second decade of life. It is a rare trait, causing severe and irreversible blindness.
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
    E.2.2Secondary objectives of the trial
    To assesses the efficacy of tinlarebant in adolescent subjects with STGD1 for secondary endpoints (Secondary efficacy endpoints will be assessed at Month 24.)
    To evaluate the pharmacodynamics (PD) of tinlarebant in adolescent subjects with STGD1
    To assess systemic and ocular safety and tolerability of tinlarebant
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects 12 to 18 years old, inclusive.
    2. Subject must have clinically diagnosed STGD1 with at least 1 mutation identified in the ABCA4 gene.
    3. Ability to adequately examine the fundus of the study eye at enrollment.
    a. The study eye will be the eye that meets all inclusion and exclusion criteria.
    b. If both eyes meet all inclusion and exclusion criteria, the eye with the smaller lesion will be selected to be the study eye.
    c. If both eyes meet all inclusion and exclusion criteria and have lesions of equal size, then the eye with the better BCVA will be selected to be the study eye.
    d. If both eyes meet all inclusion and exclusion criteria, have lesions of equal size, and have equal BCVA, the default will be the right eye.
    4. Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2) as imaged by FAF (DDAF) in the study eye and confirmed by the
    central reading center (Fleckenstein et al, 2011; Sunness et al, 2007). The lesion, or at least 1 focal lesion if multiple lesions exist (multifocal atrophic lesions), must be in the macular region and greater than 0.02 disc areas (0.05 mm2).
    5. Subjects must present with BCVA of 20/200 or better for the study eye based on ETDRS letter score. No minimum VA is required in the fellow eye.
    6. Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
    7. Subject agrees to comply with all protocol requirements.
    E.4Principal exclusion criteria
    1. Any ocular disease other than STGD1 at screening that, in the opinion of the investigator, would complicate assessment of a treatment effect.
    2. History of ocular surgery in the study eye in the last 3 months.
    3. Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
    4. Any prior gene therapy.
    5. Vitamin A deficiency as defined based upon plasma values less than 20 µg/dL (=0.7 μmol/L).
    6. Use of medications such as isotretinoin (13-cis-retinoic acid) or other retinol modulators or derivatives that may impact the effect of the study drug in the last
    2 weeks or within the washout period of the medication, whichever is longer, before beginning study treatment administration.
    7. Unwilling to discontinue vitamin A or beta-carotene supplement use.
    8. Use of any known drugs or supplements that are moderate or strong inhibitors/inducers of cytochrome P450 (CYP) enzymes (eg, rifampin, barbiturates, phenothiazines, cimetidine, carbamazepine, St. John’s wort) within 30 days of study drug administration or consumption of foods that are moderate or strong inhibitors/inducers of CYP enzymes (eg, grapefruit, pomegranate, star fruit, bitter orange [Seville orange]) within 48 hours of study drug administration, and that, in the investigator’s judgement, may impact subject safety or the validity of the study results.
    9. Presence of any life-threatening disease(s), including current treatment for malignancies.
    10. Alanine transaminase/aspartate aminotransferase >2.5 × the upper limit of normal at screening.
    11. Renal insufficiency, as defined by an estimated glomerular filtration rate (Bedside Schwartz) <30 mL/min/1.73 m2at screening.
    12. Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of contraception (or abstinence). Females of childbearing age must have a negative pregnancy test prior to randomization (Appendix 13.5).
    13. Male subjects who do not agree that female spouses/partners will use adequate contraception (eg, condoms) or be of nonchildbearing potential (ie, surgically sterile) (Appendix 13.5).
    14. Unwilling or unable to provide signed informed consent/assent.
    15. In the opinion of the investigator, the subject is not suitable for entry into the study.
    E.5 End points
    E.5.1Primary end point(s)
    Rate of change from baseline in aggregate area of atrophy (definitely decreased autofluorescence [DDAF]) assessed by FAF photography.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    E.5.2Secondary end point(s)
    • Change in retinal thickness and morphology by SDOCT from baseline
    • Change in retinal sensitivity by microperimetry from baseline
    • Change in BCVA measured by the ETDRS method from baseline
    • Change in RBP4 levels from baseline
    • Correlation between the change in RBP4 level and the rate of lesion size growth from baseline
    • Physical examination, vital signs measurement, ECG, ophthalmic examination, clinical laboratory tests (including serum chemistry and hematology panels, urinalysis, and pregnancy tests on all female subjects), retinol chemistries (plasma retinol, plasma RBP4), visual function questionnaire, measurement of intraocular pressure (IOP), dark adaptation test, dilated funduscopy, contrast sensitivity, assessment of adverse events (AEs), and monitoring of concomitant medications.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Evaluation on the following months: 1, 4, 7, 10, 13, 16, 19 and 22
    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 No
    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) 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 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 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 EEA5
    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
    Hong Kong
    China
    Germany
    Switzerland
    Taiwan
    United Kingdom
    United States
    France
    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 years
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months36
    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: 60
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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 state5
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 60
    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)
    Standard of Care
    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 Decision2022-11-09
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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