Ignite Creation Date:
2025-12-25 @ 2:16 AM
Ignite Modification Date:
2025-12-27 @ 11:02 PM
Study NCT ID:
NCT05878860
Status:
RECRUITING
Last Update Posted:
2025-02-19
First Post:
2023-05-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D041441', 'term': 'Retinoschisis'}], 'ancestors': [{'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D005128', 'term': 'Eye Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'Cohort 3 will be partially masked.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 21}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-08-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2029-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-02-18', 'studyFirstSubmitDate': '2023-05-12', 'studyFirstSubmitQcDate': '2023-05-24', 'lastUpdatePostDateStruct': {'date': '2025-02-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-05-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events', 'timeFrame': 'From baseline to week 52', 'description': 'Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).'}], 'secondaryOutcomes': [{'measure': 'Visual acuity as assessed by best-corrected visual acuity', 'timeFrame': 'From baseline to week 52', 'description': 'Change in best-corrected visual acuity (BCVA).'}, {'measure': 'Visual acuity as assessed by low-luminance visual acuity', 'timeFrame': 'From baseline to week 52', 'description': 'Change in low-luminance visual acuity (LLVA).'}, {'measure': 'Visual function as assessed by contrast sensitivity', 'timeFrame': 'From baseline to week 52', 'description': 'Change in contrast sensitivity.'}, {'measure': 'Visual function as assessed by full-field electroretinogram parameters', 'timeFrame': 'From baseline to week 52', 'description': 'Change in full-field electroretinogram (ffERG) parameters.'}, {'measure': 'Visual function as assessed by microperimetry', 'timeFrame': 'From baseline to week 52', 'description': 'Change in microperimetry.'}, {'measure': 'Visual function as assessed by static perimetry', 'timeFrame': 'From baseline to week 52', 'description': 'Change in static perimetry.'}, {'measure': 'Macular structure as assessed by spectral domain optical coherence tomography', 'timeFrame': 'From baseline to week 52', 'description': 'Change in spectral domain optical coherence tomography (SD-OCT).'}, {'measure': 'Macular structure as assessed by fundus autofluorescence', 'timeFrame': 'From baseline to week 52', 'description': 'Change in fundus autofluorescence (FAF).'}, {'measure': 'Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects', 'timeFrame': 'From baseline to week 52', 'description': "Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects with scores from 0 to 100 where a higher score indicates a better outcome."}, {'measure': 'Subject-reported visual function as assessed by the CVAQC in pediatric subjects', 'timeFrame': 'From baseline to week 52', 'description': 'Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects with scores from -3.00 to +2.80 where a higher score indicates a worse outcome.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['XLRS, RS1'], 'conditions': ['X-linked Retinoschisis']}, 'descriptionModule': {'briefSummary': 'This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).', 'detailedDescription': 'Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \\< 18 years for Cohort 4.\n2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1.\n3. Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40).\n\nExclusion Criteria:\n\n1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.\n2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study.\n3. Treatment in a prior ocular gene or cell therapy study.'}, 'identificationModule': {'nctId': 'NCT05878860', 'acronym': 'LIGHTHOUSE', 'briefTitle': 'ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Atsena Therapeutics Inc.'}, 'officialTitle': 'A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects With RS1-Associated X-linked Retinoschisis', 'orgStudyIdInfo': {'id': 'ATSN-201-1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1, Low Dose', 'interventionNames': ['Biological: ATSN-201']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2, High Dose', 'interventionNames': ['Biological: ATSN-201']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3, Mid Dose', 'interventionNames': ['Biological: ATSN-201']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4, Low Volume', 'interventionNames': ['Biological: ATSN-201']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4, High Volume', 'interventionNames': ['Biological: ATSN-201']}, {'type': 'NO_INTERVENTION', 'label': 'Cohort 4, Control'}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5, Pediatric', 'interventionNames': ['Biological: ATSN-201']}], 'interventions': [{'name': 'ATSN-201', 'type': 'BIOLOGICAL', 'description': 'AAV.SPR-hGRK1-hRS1syn', 'armGroupLabels': ['Cohort 1, Low Dose', 'Cohort 2, High Dose', 'Cohort 3, Mid Dose', 'Cohort 4, High Volume', 'Cohort 4, Low Volume', 'Cohort 5, Pediatric']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Aaron Nagiel, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Children's Hospital of Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Byron Lam, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Bascom Palmer Eye Institute', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Lesley Everett, MD, PhD, MPhil', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Oregon Health Sciences University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Tomas Aleman, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}], 'centralContacts': [{'name': 'Atsena Therapeutics Clinical Trials', 'role': 'CONTACT', 'email': 'clinicaltrials@atsenatx.com', 'phone': '984-261-2001'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Atsena Therapeutics Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}