{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Batten+Disease", "query": { "condition": "Batten Disease" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 40, "total_pages": 4, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Batten+Disease&page=2&page_size=10", "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-21T23:39:38.153Z", "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.", "notes": [ "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.", "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.", "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees." ], "trials": [ { "nct_id": "NCT01414985", "title": "AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Late Infantile Neuronal Ceroid Lipofuscinosis", "Batten Disease" ], "interventions": [ { "name": "AAVrh.10CUCLN2", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Weill Medical College of Cornell University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "3 Years", "maximum_age": "18 Years", "sex": "ALL", "summary": "3 Years to 18 Years" }, "enrollment_count": 8, "start_date": "2010-04-15", "completion_date": "2017-02-08", "has_results": false, "last_update_posted_date": "2020-08-31", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT01414985" }, { "nct_id": "NCT02435940", "title": "Inherited Retinal Degenerative Disease Registry", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Eye Diseases Hereditary", "Retinal Disease", "Achromatopsia", "Bardet-Biedl Syndrome", "Bassen-Kornzweig Syndrome", "Batten Disease", "Best Disease", "Choroidal Dystrophy", "Choroideremia", "Cone Dystrophy", "Cone-Rod Dystrophy", "Congenital Stationary Night Blindness", "Enhanced S-Cone Syndrome", "Fundus Albipunctatus", "Goldmann-Favre Syndrome", "Gyrate Atrophy", "Juvenile Macular Degeneration", "Kearns-Sayre Syndrome", "Leber Congenital Amaurosis", "Refsum Syndrome", "Retinitis Pigmentosa", "Retinitis Punctata Albescens", "Retinoschisis", "Rod-Cone Dystrophy", "Rod Dystrophy", "Rod Monochromacy", "Stargardt Disease", "Usher Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Foundation Fighting Blindness", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 20000, "start_date": "2014-06", "completion_date": "2037-06", "has_results": false, "last_update_posted_date": "2026-05-19", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Columbia, Maryland", "locations": [ { "city": "Columbia", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT02435940" }, { "nct_id": "NCT03333200", "title": "Longitudinal Study of Neurodegenerative Disorders", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "MLD", "Krabbe Disease", "ALD", "MPS I", "MPS II", "MPS III", "Vanishing White Matter Disease", "GM3 Gangliosidosis", "PKAN", "Tay-Sachs Disease", "NP Deficiency", "Osteopetrosis", "Alpha-Mannosidosis", "Sandhoff Disease", "Niemann-Pick Diseases", "MPS IV", "Gaucher Disease", "GAN", "GM1 Gangliosidoses", "Morquio Disease", "S-Adenosylhomocysteine Hydrolase Deficiency", "Batten Disease", "Pelizaeus-Merzbacher Disease", "Leukodystrophy", "Lysosomal Storage Diseases", "Purine Nucleoside Phosphorylase Deficiency", "Multiple Sulfatase Deficiency Disease" ], "interventions": [ { "name": "Palliative Care", "type": "OTHER" }, { "name": "Hematopoetic Stem Cell Transplantation", "type": "BIOLOGICAL" } ], "intervention_types": [ "OTHER", "BIOLOGICAL" ], "sponsor": "University of Pittsburgh", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1500, "start_date": "2012-01-11", "completion_date": "2035-01", "has_results": false, "last_update_posted_date": "2026-02-09", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT03333200" }, { "nct_id": "NCT01035424", "title": "Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Batten Disease", "Late Infantile Neuronal Ceroid Lipofuscinosis" ], "interventions": [], "intervention_types": [], "sponsor": "Weill Medical College of Cornell University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": "18 Years", "sex": "ALL", "summary": "2 Years to 18 Years" }, "enrollment_count": 48, "start_date": "2009-06", "completion_date": "2016-01", "has_results": false, "last_update_posted_date": "2020-07-29", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT01035424" }, { "nct_id": "NCT02678689", "title": "A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Jansky-Bielschowsky Disease", "Batten Disease", "Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2", "CLN2 Disease", "CLN2 Disorder" ], "interventions": [ { "name": "BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)", "type": "BIOLOGICAL" }, { "name": "Intracerebroventricular access device", "type": "DEVICE" } ], "intervention_types": [ "BIOLOGICAL", "DEVICE" ], "sponsor": "BioMarin Pharmaceutical", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "17 Years", "sex": "ALL", "summary": "Up to 17 Years" }, "enrollment_count": 14, "start_date": "2016-01-22", "completion_date": "2022-04-20", "has_results": true, "last_update_posted_date": "2025-02-17", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Columbus, Ohio", "locations": [ { "city": "Columbus", "state": "Ohio" } ], "official_url": "https://clinicaltrials.gov/study/NCT02678689" }, { "nct_id": "NCT01399047", "title": "Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Juvenile Neuronal Ceroid Lipofuscinosis" ], "interventions": [ { "name": "Mycophenolate mofetil", "type": "DRUG" }, { "name": "Liquid Placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Rochester", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Years", "maximum_age": "25 Years", "sex": "ALL", "summary": "6 Years to 25 Years" }, "enrollment_count": 19, "start_date": "2011-07", "completion_date": "2015-11", "has_results": true, "last_update_posted_date": "2019-05-21", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Rochester, New York", "locations": [ { "city": "Rochester", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT01399047" }, { "nct_id": "NCT04476862", "title": "Cerliponase Alfa Observational Study in the US", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2" ], "interventions": [ { "name": "Cerliponase Alfa", "type": "DRUG" }, { "name": "Administration Kit", "type": "DEVICE" } ], "intervention_types": [ "DRUG", "DEVICE" ], "sponsor": "BioMarin Pharmaceutical", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 35, "start_date": "2020-08-19", "completion_date": "2030-08-31", "has_results": false, "last_update_posted_date": "2026-01-14", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 16, "location_summary": "Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more", "locations": [ { "city": "Little Rock", "state": "Arkansas" }, { "city": "Orange", "state": "California" }, { "city": "Aurora", "state": "Colorado" }, { "city": "Washington D.C.", "state": "District of Columbia" }, { "city": "Orlando", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT04476862" }, { "nct_id": "NCT01161576", "title": "Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Batten Disease", "Late-Infantile Neuronal Ceroid Lipofuscinosis" ], "interventions": [ { "name": "AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies", "type": "BIOLOGICAL" }, { "name": "AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Weill Medical College of Cornell University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": "18 Years", "sex": "ALL", "summary": "2 Years to 18 Years" }, "enrollment_count": 12, "start_date": "2010-08-19", "completion_date": "2020-12-31", "has_results": false, "last_update_posted_date": "2021-02-02", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT01161576" }, { "nct_id": "NCT00337636", "title": "Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Neuronal Ceroid Lipofuscinosis" ], "interventions": [ { "name": "Surgery to implant human CNS stem cells (HuCNS-SC)", "type": "PROCEDURE" }, { "name": "Medication to suppress the immune system", "type": "DRUG" } ], "intervention_types": [ "PROCEDURE", "DRUG" ], "sponsor": "StemCells, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Months", "maximum_age": "12 Years", "sex": "ALL", "summary": "18 Months to 12 Years" }, "enrollment_count": 6, "start_date": "2006-05", "completion_date": "2009-09", "has_results": false, "last_update_posted_date": "2015-01-15", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Portland, Oregon", "locations": [ { "city": "Portland", "state": "Oregon" } ], "official_url": "https://clinicaltrials.gov/study/NCT00337636" }, { "nct_id": "NCT05007990", "title": "Caregiving Networks Across Disease Context and the Life Course", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Inherited Metabolic Disorders", "Undiagnosed Diseases", "Batten's Disease", "Tay Sachs", "Diabetes" ], "interventions": [], "intervention_types": [], "sponsor": "National Human Genome Research Institute (NHGRI)", "sponsor_class": "NIH", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "100 Years", "sex": "ALL", "summary": "18 Years to 100 Years" }, "enrollment_count": 2800, "start_date": "2022-09-08", "completion_date": "2030-12-31", "has_results": false, "last_update_posted_date": "2026-03-30", "last_synced_at": "2026-05-21T23:39:38.153Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT05007990" } ] }