{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=ALSP", "query": { "condition": "ALSP" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 7, "total_pages": 1, "next_page_url": null, "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-22T00:59:18.682Z", "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": "NCT02171104", "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Mucopolysaccharidosis Disorders", "Hurler Syndrome", "Hunter Syndrome", "Maroteaux Lamy Syndrome", "Sly Syndrome", "Alpha-Mannosidosis", "Fucosidosis", "Aspartylglucosaminuria", "Glycoprotein Metabolic Disorders", "Sphingolipidoses", "Recessive Leukodystrophies", "Globoid Cell Leukodystrophy", "Metachromatic Leukodystrophy", "Niemann-Pick B", "Niemann-Pick C Subtype 2", "Sphingomyelin Deficiency", "Peroxisomal Disorders", "Adrenoleukodystrophy With Cerebral Involvement", "Zellweger Syndrome", "Neonatal Adrenoleukodystrophy", "Infantile Refsum Disease", "Acyl-CoA Oxidase Deficiency", "D-Bifunctional Enzyme Deficiency", "Multifunctional Enzyme Deficiency", "Alpha-methylacyl-CoA Racmase Deficiency", "Mitochondrial Neurogastrointestingal Encephalopathy", "Severe Osteopetrosis", "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)", "Inherited Metabolic Disorders" ], "interventions": [ { "name": "Stem Cell Transplantation", "type": "BIOLOGICAL" }, { "name": "IMD Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Only Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Haploidentical Only Preparative Regimen", "type": "DRUG" }, { "name": "cALD SR-A (Standard-Risk, Regimen A)", "type": "DRUG" }, { "name": "cALD SR-B (Standard-Risk, Regimen B)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen C)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen D)", "type": "DRUG" } ], "intervention_types": [ "BIOLOGICAL", "DRUG" ], "sponsor": "Masonic Cancer Center, University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "55 Years", "sex": "ALL", "summary": "Up to 55 Years" }, "enrollment_count": 149, "start_date": "2014-07-10", "completion_date": "2029-07-14", "has_results": false, "last_update_posted_date": "2026-01-07", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02171104" }, { "nct_id": "NCT03047369", "title": "The Myelin Disorders Biorepository Project", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "Leukoencephalopathies", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD Gene Mutation", "ALD (Adrenoleukodystrophy)", "X-linked Adrenoleukodystrophy", "X-ALD", "Adrenomyeloneuropathy", "Aicardi Goutieres Syndrome", "AGS", "Alexander Disease", "Alexanders Leukodystrophy", "AxD", "ADLD", "Canavan Disease", "CTX", "Cerebrotendinous Xanthomatoses", "Krabbe Disease", "GALC Deficiency", "Globoid Leukodystrophy", "TUBB4A-Related Leukodystrophy", "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum", "HBSL", "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity", "LBSL", "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)", "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation", "ALSP", "CSF1R Gene Mutation", "HCC - Hypomyelination and Congenital Cataract", "MLC1", "Megalencephalic Leukoencephalopathy With Subcortical Cysts", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus Merzbacher Like Disease", "Peroxisomal Biogenesis Disorder", "Zellweger Syndrome", "Refsum Disease", "Salla Disease", "Sialic Storage Disease", "Sjögren", "Sjogren-Larsson Syndrome", "Van Der Knapp Disease", "Vanishing White Matter Disease", "Charcot-Marie-Tooth", "CMT", "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency", "Allan-Herndon-Dudley Syndrome", "Cadasil", "Cockayne Syndrome", "Multiple Sulfatase Deficiency", "Gangliosidoses", "GM2 Gangliosidosis", "BPAN", "Labrune Syndrome", "LCC", "Mucopolysaccharidoses", "TBCK-Related Intellectual Disability Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Children's Hospital of Philadelphia", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 12000, "start_date": "2016-12-08", "completion_date": "2030-12-08", "has_results": false, "last_update_posted_date": "2025-10-23", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 23, "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 18 more", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "Orange", "state": "California" }, { "city": "Palo Alto", "state": "California" }, { "city": "Sacramento", "state": "California" }, { "city": "San Diego", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT03047369" }, { "nct_id": "NCT05020743", "title": "Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia", "overall_status": "TERMINATED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "ALSP" ], "interventions": [ { "name": "No intervention", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Vigil Neuroscience, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 56, "start_date": "2021-09-13", "completion_date": "2025-05-30", "has_results": true, "last_update_posted_date": "2025-08-13", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 6, "location_summary": "San Francisco, California • Englewood, Colorado • Boca Raton, Florida + 3 more", "locations": [ { "city": "San Francisco", "state": "California" }, { "city": "Englewood", "state": "Colorado" }, { "city": "Boca Raton", "state": "Florida" }, { "city": "Jacksonville", "state": "Florida" }, { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT05020743" }, { "nct_id": "NCT04503213", "title": "A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation", "overall_status": "ENROLLING_BY_INVITATION", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "CSF1R-related Leukoencephalopathy", "ALSP", "POLD", "Hematopoietic Stem Cell Transplantation" ], "interventions": [], "intervention_types": [], "sponsor": "Mayo Clinic", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 20, "start_date": "2020-07-21", "completion_date": "2027-12", "has_results": false, "last_update_posted_date": "2026-01-08", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 1, "location_summary": "Jacksonville, Florida", "locations": [ { "city": "Jacksonville", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT04503213" }, { "nct_id": "NCT01962415", "title": "Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Primary Immunodeficiency (PID)", "Congenital Bone Marrow Failure Syndromes", "Inherited Metabolic Disorders (IMD)", "Hereditary Anemias", "Inflammatory Conditions", "Systemic Juvenile Idiopathic Arthritis (sJIA)", "Juvenile Rheumatoid Arthritis (JRA)" ], "interventions": [ { "name": "Hydroxyurea", "type": "DRUG" }, { "name": "Alemtuzumab", "type": "DRUG" }, { "name": "Fludarabine", "type": "DRUG" }, { "name": "Melphalan", "type": "DRUG" }, { "name": "Thiotepa", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Paul Szabolcs", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Months", "maximum_age": "55 Years", "sex": "ALL", "summary": "2 Months to 55 Years" }, "enrollment_count": 100, "start_date": "2014-02-04", "completion_date": "2027-11", "has_results": false, "last_update_posted_date": "2025-12-15", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT01962415" }, { "nct_id": "NCT02699190", "title": "LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Leukodystrophy", "White Matter Disease", "4H Syndrome", "Adrenoleukodystrophy", "AMN", "ALD", "ALD (Adrenoleukodystrophy)", "X-linked Adrenoleukodystrophy", "X-ALD", "Adrenomyeloneuropathy", "Aicardi Goutieres Syndrome", "AGS", "Alexander Disease", "Alexanders Leukodystrophy", "AxD", "ADLD", "Canavan Disease", "CTX", "Cerebrotendinous Xanthomatoses", "Krabbe Disease", "GALC Deficiency", "Globoid Leukodystrophy", "TUBB4A-Related Leukodystrophy", "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum", "HBSL", "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity", "LBSL", "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)", "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation", "ALSP", "CSF1R Gene Mutation", "HCC - Hypomyelination and Congenital Cataract", "MLC1", "Megalencephalic Leukoencephalopathy With Subcortical Cysts 1", "MLD", "Metachromatic Leukodystrophy", "PMD", "Pelizaeus-Merzbacher Disease", "PLP1 Null Syndrome", "PLP1 Gene Duplication | Blood or Tissue | Mutations", "Pelizaeus-Merzbacher-Like Disease, 1", "Peroxisomal Biogenesis Disorder", "Zellweger Syndrome", "Refsum Disease", "Salla Disease", "Sialic Storage Disease", "Sjögren", "Sjogren-Larsson Syndrome", "Van Der Knapp Disease", "Vanishing White Matter Disease", "Charcot-Marie-Tooth", "CMT", "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency", "Allan-Herndon-Dudley Syndrome", "Cadasil", "Cockayne Syndrome", "Multiple Sulfatase Deficiency", "Gangliosidoses", "GM2 Gangliosidosis", "BPAN", "Labrune Syndrome", "LCC", "Mucopolysaccharidoses", "TBCK-Related Intellectual Disability Syndrome" ], "interventions": [], "intervention_types": [], "sponsor": "Children's Hospital of Philadelphia", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "18 Years", "sex": "ALL", "summary": "Up to 18 Years" }, "enrollment_count": 236, "start_date": "2017-01-06", "completion_date": "2024-10-31", "has_results": true, "last_update_posted_date": "2025-11-10", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 1, "location_summary": "Philadelphia, Pennsylvania", "locations": [ { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT02699190" }, { "nct_id": "NCT05677659", "title": "A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "ALSP" ], "interventions": [ { "name": "VGL101", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Vigil Neuroscience, Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 20, "start_date": "2022-12-14", "completion_date": "2025-06-04", "has_results": false, "last_update_posted_date": "2025-06-11", "last_synced_at": "2026-05-22T00:59:18.682Z", "location_count": 5, "location_summary": "San Francisco, California • Englewood, Colorado • Jacksonville, Florida + 2 more", "locations": [ { "city": "San Francisco", "state": "California" }, { "city": "Englewood", "state": "Colorado" }, { "city": "Jacksonville", "state": "Florida" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT05677659" } ] }