{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Rare+Diseases", "query": { "condition": "Rare Diseases" }, "page_size": 10 }, "pagination": { "page": 1, "page_size": 10, "total_count": 404, "total_pages": 41, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Rare+Diseases&page=2&page_size=10", "previous_page_url": null }, "source": "remote", "last_synced_at": "2026-05-21T22:26:48.638Z", "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": "NCT04240886", "title": "Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Invasive Fungal Infections" ], "interventions": [ { "name": "fosmanogepix", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Basilea Pharmaceutica", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 21, "start_date": "2020-01-04", "completion_date": "2022-05-09", "has_results": true, "last_update_posted_date": "2025-09-16", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 6, "location_summary": "Duarte, California • Durham, North Carolina", "locations": [ { "city": "Duarte", "state": "California" }, { "city": "Duarte", "state": "California" }, { "city": "Durham", "state": "North Carolina" }, { "city": "Durham", "state": "North Carolina" }, { "city": "Durham", "state": "North Carolina" } ], "official_url": "https://clinicaltrials.gov/study/NCT04240886" }, { "nct_id": "NCT04221451", "title": "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Tay-Sachs Disease", "Sandhoff Disease" ], "interventions": [ { "name": "venglustat GZ402671", "type": "DRUG" }, { "name": "placebo", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Genzyme, a Sanofi Company", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "2 Years", "maximum_age": null, "sex": "ALL", "summary": "2 Years and older" }, "enrollment_count": 75, "start_date": "2020-06-29", "completion_date": "2024-12-26", "has_results": true, "last_update_posted_date": "2026-01-28", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 5, "location_summary": "Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more", "locations": [ { "city": "Los Angeles", "state": "California" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Bethesda", "state": "Maryland" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT04221451" }, { "nct_id": "NCT06539169", "title": "FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Alpha-Thalassemia", "Beta-Thalassemia", "Amyloidosis", "Amyotrophic Lateral Sclerosis", "Creutzfeld-Jakob Disease", "Cystic Fibrosis", "Duchenne Muscular Dystrophy", "Early-Onset Alzheimer Disease", "Ehlers-Danlos Syndrome", "Huntington Disease", "Gaucher Disease", "GM1 Gangliosidosis", "Myasthenia Gravis", "Pompe Disease", "Sickle Cell Disease", "Transthyretin Amyloid Cardiomyopathy", "Rare Diseases" ], "interventions": [], "intervention_types": [], "sponsor": "xCures", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1000, "start_date": "2024-06-10", "completion_date": "2026-06-10", "has_results": false, "last_update_posted_date": "2024-11-14", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 1, "location_summary": "Los Altos, California", "locations": [ { "city": "Los Altos", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT06539169" }, { "nct_id": "NCT00634049", "title": "Isavuconazole in the Treatment of Renally Impaired Aspergillosis and Rare Fungi", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE3" ], "conditions": [ "Aspergillosis", "Invasive Fungal Infections" ], "interventions": [ { "name": "isavuconazole", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Astellas Pharma Inc", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 149, "start_date": "2008-04-22", "completion_date": "2016-05-05", "has_results": true, "last_update_posted_date": "2024-12-11", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 24, "location_summary": "Birmingham, Alabama • Duarte, California • Sacramento, California + 19 more", "locations": [ { "city": "Birmingham", "state": "Alabama" }, { "city": "Duarte", "state": "California" }, { "city": "Sacramento", "state": "California" }, { "city": "San Francisco", "state": "California" }, { "city": "San Francisco", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT00634049" }, { "nct_id": "NCT00010335", "title": "Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Systemic Sclerosis", "Systemic Lupus Erythematosus", "Dermatomyositis", "Juvenile Rheumatoid Arthritis", "Autoimmune Diseases" ], "interventions": [ { "name": "Stem Cell Transplantation", "type": "PROCEDURE" }, { "name": "CD34 selection", "type": "DEVICE" } ], "intervention_types": [ "PROCEDURE", "DEVICE" ], "sponsor": "Fred Hutchinson Cancer Center", "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": 6, "start_date": "2000-11", "completion_date": "2011-05", "has_results": false, "last_update_posted_date": "2015-03-06", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 1, "location_summary": "Seattle, Washington", "locations": [ { "city": "Seattle", "state": "Washington" } ], "official_url": "https://clinicaltrials.gov/study/NCT00010335" }, { "nct_id": "NCT07074015", "title": "IntelliWell: An AI-Assisted Imaging Platform for Detection and Location of Ultra-Rare Testicular Sperm in Surgical Specimens", "overall_status": "RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Infertility", "Azoospermia" ], "interventions": [ { "name": "Use of IntelliWell to identify and localize sperm for ICSI", "type": "DEVICE" } ], "intervention_types": [ "DEVICE" ], "sponsor": "Brigham and Women's Hospital", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "MALE", "summary": "18 Years and older · Male only" }, "enrollment_count": 20, "start_date": "2026-04-15", "completion_date": "2027-12-01", "has_results": false, "last_update_posted_date": "2026-04-15", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 1, "location_summary": "Boston, Massachusetts", "locations": [ { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT07074015" }, { "nct_id": "NCT04795206", "title": "Natural Disease Progression in Participants With Choroideremia", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Choroideremia" ], "interventions": [ { "name": "No Intervention", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "Biogen", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "10 Years", "maximum_age": null, "sex": "ALL", "summary": "10 Years and older" }, "enrollment_count": 1178, "start_date": "2020-08-26", "completion_date": "2021-08-10", "has_results": false, "last_update_posted_date": "2021-09-08", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 1, "location_summary": "San Francisco, California", "locations": [ { "city": "San Francisco", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT04795206" }, { "nct_id": "NCT04482309", "title": "A Phase 2 Study of T-DXd in Patients With Selected HER2 Expressing Tumors", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Part 1: Bladder, Biliary Tract, Cervical, Endometrial, Ovarian, Pancreatic Cancer, Rare Tumors, Any Tumor Type Excluding Breast, Gastric, Colorectal Cancer", "Part 2: HER2 Expressing/Amplified Solid Tumors Excluding Breast, Gastric, Colorectal Cancer" ], "interventions": [ { "name": "Trastuzumab deruxtecan", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "AstraZeneca", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "120 Years", "sex": "ALL", "summary": "18 Years to 120 Years" }, "enrollment_count": 477, "start_date": "2020-08-18", "completion_date": "2027-03-23", "has_results": false, "last_update_posted_date": "2026-04-09", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 12, "location_summary": "Duarte, California • Santa Rosa, California • Chicago, Illinois + 8 more", "locations": [ { "city": "Duarte", "state": "California" }, { "city": "Santa Rosa", "state": "California" }, { "city": "Chicago", "state": "Illinois" }, { "city": "Muncie", "state": "Indiana" }, { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT04482309" }, { "nct_id": "NCT00004431", "title": "Randomized Study of L-Baclofen in Patients With Refractory Trigeminal Neuralgia", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Trigeminal Neuralgia" ], "interventions": [ { "name": "L-baclofen", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Pittsburgh", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "80 Years", "sex": "ALL", "summary": "18 Years to 80 Years" }, "enrollment_count": 30, "start_date": "1998-06", "completion_date": "1999-09", "has_results": false, "last_update_posted_date": "2015-03-25", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT00004431" }, { "nct_id": "NCT02574728", "title": "Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Cancer" ], "interventions": [ { "name": "Sirolimus", "type": "DRUG" }, { "name": "Celecoxib", "type": "DRUG" }, { "name": "Etoposide", "type": "DRUG" }, { "name": "Cyclophosphamide", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Emory University", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "12 Months", "maximum_age": "30 Years", "sex": "ALL", "summary": "12 Months to 30 Years" }, "enrollment_count": 46, "start_date": "2015-06", "completion_date": "2026-06", "has_results": false, "last_update_posted_date": "2026-03-02", "last_synced_at": "2026-05-21T22:26:48.638Z", "location_count": 6, "location_summary": "Phoenix, Arizona • Wilmington, Delaware • Atlanta, Georgia + 2 more", "locations": [ { "city": "Phoenix", "state": "Arizona" }, { "city": "Wilmington", "state": "Delaware" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Atlanta", "state": "Georgia" }, { "city": "Kansas City", "state": "Missouri" } ], "official_url": "https://clinicaltrials.gov/study/NCT02574728" } ] }