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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

🎯 Match trials to me

30 recruiting trials

for Inherited BMF Syndrome

Investigation of the Genetics of Hematologic Diseases NCT02720679n/a1 site📍 Memphis St. Jude Children's Research Hospital Role of Acetaldehyde in the Development of Oral Cancer NCT05973656NA1 site📍 Minneapolis Masonic Cancer Center, University of Minnesota · Tests: Alcohol, Biospecimen Collection, Breath Test Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency NCT06090669Phase 11 site📍 Bethesda National Cancer Institute (NCI) · Tests: TruSight Oncology, imatinib Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC NCT04528355n/a1 site📍 Pittsburgh Paul Szabolcs · Tests: data collection Natural History of Acquired and Inherited Bone Marrow Failure Syndromes NCT05012111n/a1 site📍 Bethesda National Heart, Lung, and Blood Institute (NHLBI) T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias NCT03653338Phase 1, Phase 21 site📍 Pittsburgh Paul Szabolcs · Tests: CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study) NCT05196789n/a1 site📍 Melbourne Peter MacCallum Cancer Centre, Australia · Tests: whole genome and transcriptome sequencing Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform NCT06999954n/a1 site📍 Woburn Shwachman-Diamond Syndrome Alliance Inc Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) NCT07186179n/a1 site📍 New Hyde Park Northwell Health · Tests: Mobilization Regimen Familial Investigations of Childhood Cancer Predisposition NCT03050268n/a1 site📍 Memphis St. Jude Children's Research Hospital Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) NCT06648096Phase 1, Phase 22 sites📍 Hanover📍 Barcelona Fundació Institut de Recerca de l'Hospital de la Santa Creu… · Tests: Afatinib HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy NCT02143830Phase 23 sites📍 New York📍 Cincinnati📍 Seattle Children's Hospital Medical Center, Cincinnati · Tests: Busulfan, Cyclophosphamide, Fludarabine, rabbit ATG, G-CSF A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections NCT06056297Phase 3114 sites📍 Phoenix📍 Irvine📍 La Jolla X4 Pharmaceuticals · Tests: Mavorixafor, Placebo MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients NCT07649031n/a1 site📍 Minneapolis University of Minnesota · Tests: MRIs Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data NCT07877727n/a4 sites📍 Avellino📍 Genova📍 Naples Istituto Giannina Gaslini Pre-myeloid Cancer and Bone Marrow Failure Clinic Study NCT02958462NA3 sites📍 Scottsdale📍 Jacksonville📍 Rochester Mayo Clinic · Tests: Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy, Buccal Swab, Clinical Evaluation Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures NCT04232085Phase 21 site📍 Baltimore Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Tests: Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide French National Registry of Bone Marrow Failures NCT04781790n/a1 site📍 Paris Assistance Publique - Hôpitaux de Paris · Tests: Bone Marrow Failure Cancer in Inherited Bone Marrow Failure Syndromes NCT00027274n/a2 sites📍 Bethesda📍 Rockville National Cancer Institute (NCI) Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders NCT03579875Phase 21 site📍 Minneapolis Masonic Cancer Center, University of Minnesota · Tests: Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Dono… Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT NCT06839456Phase 1, Phase 21 site📍 Philadelphia Children's Hospital of Philadelphia · Tests: Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1,… Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD) NCT07628972Phase 11 site📍 Cincinnati Children's Hospital Medical Center, Cincinnati · Tests: Quercetin Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia NCT07476183Phase 12 sites📍 Stanford📍 Boston Apriligen, Inc. · Tests: APR-2020 Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab NCT04784052Phase 1, Phase 21 site📍 Stanford Porteus, Matthew, MD · Tests: JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes NCT05436587n/a1 site📍 Sohag Sohag University · Tests: The whole-exome sequencing Shwachman Diamond Syndrome Registry and Study NCT06056908n/a4 sites📍 Aurora📍 Boston📍 Boston Boston Children's Hospital Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia NCT05687149n/a1 site📍 Bethesda National Cancer Institute (NCI) Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415Phase 21 site📍 Pittsburgh Paul Szabolcs · Tests: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion NCT04099966Phase 21 site📍 Valhalla Mitchell Cairo · Tests: alpha beta depletion Nucleoside Therapy in Patients With Telomere Biology Disorders NCT06817590Phase 11 site📍 Boston Suneet Agarwal · Tests: deoxycytidine, deoxythymidine
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