{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Congenital+Bone+Marrow+Failure+Syndromes&page=4", "query": { "condition": "Congenital Bone Marrow Failure Syndromes", "page": 4 }, "page_size": 10 }, "pagination": { "page": 4, "page_size": 10, "total_count": 156, "total_pages": 16, "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Congenital+Bone+Marrow+Failure+Syndromes&page=5&page_size=10", "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Congenital+Bone+Marrow+Failure+Syndromes&page=3&page_size=10" }, "source": "remote", "last_synced_at": "2026-10-09T17:02:36.286Z", "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": "NCT04248439", "title": "Gene Therapy for Fanconi Anemia, Complementation Group A", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Fanconi Anemia Complementation Group A" ], "interventions": [ { "name": "RP-L102", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Rocket Pharmaceuticals Inc.", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "1 Year", "maximum_age": null, "sex": "ALL", "summary": "1 Year and older" }, "enrollment_count": 5, "start_date": "2020-07-15", "completion_date": "2026-05-05", "has_results": false, "last_update_posted_date": "2025-12-22", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 2, "location_summary": "Stanford, California • Minneapolis, Minnesota", "locations": [ { "city": "Stanford", "state": "California" }, { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT04248439" }, { "nct_id": "NCT06056908", "title": "Shwachman Diamond Syndrome Registry and Study", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Shwachman-Diamond Syndrome", "Shwachman-Diamond Syndrome-Like" ], "interventions": [], "intervention_types": [], "sponsor": "Boston Children's Hospital", "sponsor_class": "OTHER", "healthy_volunteers": null, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 5000, "start_date": "2016-01-19", "completion_date": "2090-01-01", "has_results": false, "last_update_posted_date": "2026-04-06", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 4, "location_summary": "Aurora, Colorado • Boston, Massachusetts • Cincinnati, Ohio", "locations": [ { "city": "Aurora", "state": "Colorado" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "Boston", "state": "Massachusetts" }, { "city": "Cincinnati", "state": "Ohio" } ], "official_url": "https://clinicaltrials.gov/study/NCT06056908" }, { "nct_id": "NCT04269889", "title": "Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Anemia, Diamond-Blackfan" ], "interventions": [ { "name": "Eltrombopag", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "National Heart, Lung, and Blood Institute (NHLBI)", "sponsor_class": "NIH", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Years", "maximum_age": null, "sex": "ALL", "summary": "2 Years and older" }, "enrollment_count": 15, "start_date": "2020-12-01", "completion_date": "2024-02-16", "has_results": true, "last_update_posted_date": "2025-04-27", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "Bethesda, Maryland", "locations": [ { "city": "Bethesda", "state": "Maryland" } ], "official_url": "https://clinicaltrials.gov/study/NCT04269889" }, { "nct_id": "NCT01071239", "title": "Hematopoietic Stem Cell Transplant for Fanconi Anemia", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Fanconi Anemia" ], "interventions": [ { "name": "CliniMACs device", "type": "DEVICE" }, { "name": "Busulfan", "type": "DRUG" }, { "name": "Fludarabine", "type": "DRUG" }, { "name": "Cyclophosphamide", "type": "DRUG" }, { "name": "ATG", "type": "DRUG" } ], "intervention_types": [ "DEVICE", "DRUG" ], "sponsor": "Medical College of Wisconsin", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 1, "start_date": "2009-04", "completion_date": "2016-08-30", "has_results": false, "last_update_posted_date": "2019-09-11", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "Milwaukee, Wisconsin", "locations": [ { "city": "Milwaukee", "state": "Wisconsin" } ], "official_url": "https://clinicaltrials.gov/study/NCT01071239" }, { "nct_id": "NCT06999954", "title": "Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Shwachman-Diamond Syndrome", "SDS", "IBMF", "Congenital Neutropenia", "Heme Malignancy", "Shwachman Syndrome", "Inherited Bone Marrow Failure", "Exocrine Pancreatic Insufficiency", "WHIM", "ELANE", "SBDS Gene Mutation", "EFL1 Gene Mutation", "DNAJC21 Gene Mutation", "SRP54 Gene Mutation", "Inherited Cancer Syndrome", "Inherited Cancer-Predisposing Syndrome", "Neutropenia, Severe Chronic", "Neutropenia Other", "Neutropenia Chronic Benign", "Ribosome Alteration", "Ribosomopathy", "Immune Deficiency", "Inherited BMF Syndrome", "Inherited Immunodeficiency Diseases", "Cognitive Delay, Mild", "Myelodysplastic Syndromes", "Pancytopenia" ], "interventions": [], "intervention_types": [], "sponsor": "Shwachman-Diamond Syndrome Alliance Inc", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "ALL", "summary": "Not listed" }, "enrollment_count": 8000, "start_date": "2024-02-07", "completion_date": "2088-12", "has_results": false, "last_update_posted_date": "2025-05-31", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "Woburn, Massachusetts", "locations": [ { "city": "Woburn", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT06999954" }, { "nct_id": "NCT00305708", "title": "Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Congenital Amegakaryocytic Thrombocytopenia", "Diamond-blackfan Anemia", "Fanconi Anemia", "Leukemia", "Severe Congenital Neutropenia", "Thrombocytopenia" ], "interventions": [ { "name": "anti-thymocyte globulin", "type": "BIOLOGICAL" }, { "name": "busulfan", "type": "DRUG" }, { "name": "fludarabine phosphate", "type": "DRUG" }, { "name": "allogeneic bone marrow transplantation", "type": "PROCEDURE" }, { "name": "peripheral blood stem cell transplantation", "type": "PROCEDURE" }, { "name": "umbilical cord blood transplantation", "type": "PROCEDURE" }, { "name": "radiation therapy", "type": "RADIATION" } ], "intervention_types": [ "BIOLOGICAL", "DRUG", "PROCEDURE", "RADIATION" ], "sponsor": "University of California, San Francisco", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "17 Years", "sex": "ALL", "summary": "Up to 17 Years" }, "enrollment_count": 40, "start_date": "2000-08", "completion_date": "2004-07", "has_results": false, "last_update_posted_date": "2012-11-12", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "San Francisco, California", "locations": [ { "city": "San Francisco", "state": "California" } ], "official_url": "https://clinicaltrials.gov/study/NCT00305708" }, { "nct_id": "NCT04528355", "title": "Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Primary Immunodeficiency (PID)", "Congenital Bone Marrow Failure Syndromes", "Inherited Metabolic Disorders (IMD)", "Hereditary Anemias", "Inflammatory Conditions" ], "interventions": [ { "name": "data collection", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Paul Szabolcs", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Months", "maximum_age": "60 Years", "sex": "ALL", "summary": "2 Months to 60 Years" }, "enrollment_count": 50, "start_date": "2020-08-20", "completion_date": "2028-06-30", "has_results": false, "last_update_posted_date": "2026-01-13", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT04528355" }, { "nct_id": "NCT03966053", "title": "The Use of Trifluoperazine in Transfusion Dependent DBA", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Diamond Blackfan Anemia", "Pure Red Cell Aplasia" ], "interventions": [ { "name": "Trifluoperazine", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Adrianna Vlachos, MD", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": "65 Years", "sex": "ALL", "summary": "18 Years to 65 Years" }, "enrollment_count": 2, "start_date": "2019-09-13", "completion_date": "2021-10-13", "has_results": true, "last_update_posted_date": "2022-12-14", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 2, "location_summary": "Boston, Massachusetts • Manhasset, New York", "locations": [ { "city": "Boston", "state": "Massachusetts" }, { "city": "Manhasset", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT03966053" }, { "nct_id": "NCT07660783", "title": "Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases", "overall_status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Bone Marrow Failure", "Hemoglobinopathies", "Primary Immune Deficiency (PID)", "Autoimmune Cytopenia", "Immune Dysregulation", "Hemophagocytic Lymphohistiocytosis (HLH)", "Non Malignant Disorders" ], "interventions": [ { "name": "ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Fred Hutchinson Cancer Center", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "6 Months", "maximum_age": "50 Years", "sex": "ALL", "summary": "6 Months to 50 Years" }, "enrollment_count": 40, "start_date": "2026-09", "completion_date": "2035-09", "has_results": false, "last_update_posted_date": "2026-06-22", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 2, "location_summary": "Seattle, Washington", "locations": [ { "city": "Seattle", "state": "Washington" }, { "city": "Seattle", "state": "Washington" } ], "official_url": "https://clinicaltrials.gov/study/NCT07660783" }, { "nct_id": "NCT00856388", "title": "Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "NA" ], "conditions": [ "Accelerated Phase Chronic Myelogenous Leukemia", "Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome", "Adult Acute Lymphoblastic Leukemia in Remission", "Adult Acute Myeloid Leukemia in Remission", "Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities", "Adult Acute Myeloid Leukemia With Del(5q)", "Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)", "Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)", "Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)", "Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)", "Adult Nasal Type Extranodal NK/T-cell Lymphoma", "Anaplastic Large Cell Lymphoma", "Angioimmunoblastic T-cell Lymphoma", "Aplastic Anemia", "Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative", "Childhood Acute Lymphoblastic Leukemia in Remission", "Childhood Acute Myeloid Leukemia in Remission", "Childhood Chronic Myelogenous Leukemia", "Childhood Diffuse Large Cell Lymphoma", "Childhood Immunoblastic Large Cell Lymphoma", "Childhood Myelodysplastic Syndromes", "Childhood Nasal Type Extranodal NK/T-cell Lymphoma", "Chronic Eosinophilic Leukemia", "Chronic Myelomonocytic Leukemia", "Chronic Neutrophilic Leukemia", "Chronic Phase Chronic Myelogenous Leukemia", "de Novo Myelodysplastic Syndromes", "Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue", "Fanconi Anemia", "Juvenile Myelomonocytic Leukemia", "Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable", "Nodal Marginal Zone B-cell Lymphoma", "Noncontiguous Stage II Adult Burkitt Lymphoma", "Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma", "Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma", "Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma", "Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma", "Noncontiguous Stage II Adult Lymphoblastic Lymphoma", "Noncontiguous Stage II Grade 1 Follicular Lymphoma", "Noncontiguous Stage II Grade 2 Follicular Lymphoma", "Noncontiguous Stage II Grade 3 Follicular Lymphoma", "Noncontiguous Stage II Mantle Cell Lymphoma", "Noncontiguous Stage II Marginal Zone Lymphoma", "Noncontiguous Stage II Small Lymphocytic Lymphoma", "Paroxysmal Nocturnal Hemoglobinuria", "Previously Treated Myelodysplastic Syndromes", "Primary Myelofibrosis", "Recurrent Adult Acute Lymphoblastic Leukemia", "Recurrent Adult Acute Myeloid Leukemia", "Recurrent Adult Burkitt Lymphoma", "Recurrent Adult Diffuse Large Cell Lymphoma", "Recurrent Adult Diffuse Mixed Cell Lymphoma", "Recurrent Adult Diffuse Small Cleaved Cell Lymphoma", "Recurrent Adult Grade III Lymphomatoid Granulomatosis", "Recurrent Adult Hodgkin Lymphoma", "Recurrent Adult Immunoblastic Large Cell Lymphoma", "Recurrent Adult Lymphoblastic Lymphoma", "Recurrent Adult T-cell Leukemia/Lymphoma", "Recurrent Childhood Acute Lymphoblastic Leukemia", "Recurrent Childhood Acute Myeloid Leukemia", "Recurrent Childhood Anaplastic Large Cell Lymphoma", "Recurrent Childhood Grade III Lymphomatoid Granulomatosis", "Recurrent Childhood Large Cell Lymphoma", "Recurrent Childhood Lymphoblastic Lymphoma", "Recurrent Childhood Small Noncleaved Cell Lymphoma", "Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma", "Recurrent Grade 1 Follicular Lymphoma", "Recurrent Grade 2 Follicular Lymphoma", "Recurrent Grade 3 Follicular Lymphoma", "Recurrent Mantle Cell Lymphoma", "Recurrent Marginal Zone Lymphoma", "Recurrent Mycosis Fungoides/Sezary Syndrome", "Recurrent Small Lymphocytic Lymphoma", "Recurrent/Refractory Childhood Hodgkin Lymphoma", "Refractory Chronic Lymphocytic Leukemia", "Refractory Multiple Myeloma", "Relapsing Chronic Myelogenous Leukemia", "Secondary Acute Myeloid Leukemia", "Secondary Myelodysplastic Syndromes", "Splenic Marginal Zone Lymphoma", "Stage III Adult Diffuse Small Cleaved Cell Lymphoma", "Stage III Adult Immunoblastic Large Cell Lymphoma", "Stage III Adult Lymphoblastic Lymphoma", "Stage III Grade 1 Follicular Lymphoma", "Stage III Grade 2 Follicular Lymphoma", "Stage III Grade 3 Follicular Lymphoma", "Stage III Mantle Cell Lymphoma", "Stage III Marginal Zone Lymphoma", "Stage III Small Lymphocytic Lymphoma", "Stage IV Adult Burkitt Lymphoma", "Stage IV Adult Diffuse Small Cleaved Cell Lymphoma", "Stage IV Adult Immunoblastic Large Cell Lymphoma", "Stage IV Adult Lymphoblastic Lymphoma", "Stage IV Grade 1 Follicular Lymphoma", "Stage IV Grade 2 Follicular Lymphoma", "Stage IV Grade 3 Follicular Lymphoma", "Stage IV Mantle Cell Lymphoma", "Stage IV Marginal Zone Lymphoma", "Stage IV Small Lymphocytic Lymphoma", "Waldenström Macroglobulinemia" ], "interventions": [ { "name": "fludarabine phosphate", "type": "DRUG" }, { "name": "melphalan", "type": "DRUG" }, { "name": "total-body irradiation", "type": "RADIATION" }, { "name": "allogeneic hematopoietic stem cell transplantation", "type": "PROCEDURE" }, { "name": "anti-thymocyte globulin", "type": "BIOLOGICAL" } ], "intervention_types": [ "DRUG", "RADIATION", "PROCEDURE", "BIOLOGICAL" ], "sponsor": "Roswell Park Cancer Institute", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "3 Years", "maximum_age": "75 Years", "sex": "ALL", "summary": "3 Years to 75 Years" }, "enrollment_count": 62, "start_date": "2009-01-14", "completion_date": "2019-03-13", "has_results": true, "last_update_posted_date": "2019-11-13", "last_synced_at": "2026-10-09T17:02:36.286Z", "location_count": 1, "location_summary": "Buffalo, New York", "locations": [ { "city": "Buffalo", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT00856388" } ] }