Ignite Creation Date:
2025-12-24 @ 9:12 PM
Ignite Modification Date:
2025-12-24 @ 9:12 PM
Study NCT ID:
NCT05244304
Status:
COMPLETED
Last Update Posted:
2025-10-31
First Post:
2022-01-20
Is Possible Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000080362', 'term': 'Stargardt Disease'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D008268', 'term': 'Macular Degeneration'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Eligible subjects will be randomly assigned to begin treatment in 2:1 ratio to receive the study drug (either Tinlarebant 5 mg or matching placebo)'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 104}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-03-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2025-09-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-30', 'studyFirstSubmitDate': '2022-01-20', 'studyFirstSubmitQcDate': '2022-02-16', 'lastUpdatePostDateStruct': {'date': '2025-10-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-02-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure change in quantitative autofluorescence (qAF) level from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure change in retinal sensitivity by microperimetry from baseline', 'timeFrame': 'Baseline thru month 24'}], 'primaryOutcomes': [{'measure': 'To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline', 'timeFrame': 'Baseline thru month 24'}], 'secondaryOutcomes': [{'measure': 'To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To measure change in plasma concentration of RBP4 levels (μM) from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline', 'timeFrame': 'Baseline thru month 24'}, {'measure': 'To assess the systemic and ocular safety and tolerability of tinlarebant', 'timeFrame': 'Baseline thru month 24', 'description': 'Frequency, duration, and severity of AEs'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Stargardt Disease 1']}, 'descriptionModule': {'briefSummary': 'The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1', 'detailedDescription': 'Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female subjects 12 to 20 years old, inclusive.\n* Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.\n* Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score\n* 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.\n* Subject agrees to comply with all protocol requirements.\n\nExclusion Criteria:\n\n* Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect.\n* History of ocular surgery in the study eye in the last 3 months.\n* Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.\n* Any prior gene therapy.\n* Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).'}, 'identificationModule': {'nctId': 'NCT05244304', 'acronym': 'DRAGON', 'briefTitle': 'Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Belite Bio, Inc'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'LBS-008-CT03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Tinlarebant', 'description': '5 mg tablet taken orally once a day', 'interventionNames': ['Drug: Tinlarebant']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Tinlarebant', 'type': 'DRUG', 'description': 'Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.', 'armGroupLabels': ['Tinlarebant']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Not active drug', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94303', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '55435', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Belite Study Site', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'city': 'East Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Belite Study Site', 'geoPoint': {'lat': -37.81667, 'lon': 144.9879}}, {'city': 'South Brisbane', 'country': 'Australia', 'facility': 'Belite Study Site', 'geoPoint': {'lat': -27.48034, 'lon': 153.02049}}, {'city': 'Ghent', 'country': 'Belgium', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'city': 'Beijing', 'country': 'China', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'city': 'Shanghai', 'country': 'China', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}, {'city': 'Paris', 'country': 'France', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Bonn', 'country': 'Germany', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 50.73438, 'lon': 7.09549}}, {'city': 'Tübingen', 'country': 'Germany', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 48.52266, 'lon': 9.05222}}, {'city': 'Kowloon', 'country': 'Hong Kong', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 22.31667, 'lon': 114.18333}}, {'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'city': 'Basel', 'country': 'Switzerland', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 47.55839, 'lon': 7.57327}}, {'city': 'Taipei', 'country': 'Taiwan', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'city': 'Taoyuan', 'country': 'Taiwan', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 24.99368, 'lon': 121.29696}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Southampton', 'country': 'United Kingdom', 'facility': 'Belite Study Site', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Belite Bio, Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}