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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.

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30 recruiting trials

for Inherited Bone Marrow Failure Syndrome

Investigation of the Genetics of Hematologic Diseases NCT02720679n/a1 site📍 Memphis St. Jude Children's Research Hospital TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children NCT04356469Phase 21 site📍 St. Petersburg Johns Hopkins All Children's Hospital · Tests: Haploidentical Hematopoietic Cell Transplantation 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) A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) NCT01351545n/a142 sites📍 Birmingham📍 Gilbert📍 Phoenix Center for International Blood and Marrow Transplant Resear… · Tests: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) 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 Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC NCT02356653EARLY Phase 11 site📍 Philadelphia Children's Hospital of Philadelphia · Tests: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) 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 Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders NCT03128996Phase 1, Phase 24 sites📍 New Haven📍 Wilmington📍 Grand Rapids Washington University School of Medicine · Tests: RIC regimen, GVHD prophylaxis regimen Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders NCT04644016Phase 21 site📍 New York Memorial Sloan Kettering Cancer Center · Tests: Clofarabine, Fludarabine, Busulfan, Cyclosporine-A, Mycophenolate Mofetil 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) High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) NCT07205523n/a1 site📍 Xining Yigeng Cao,MD,PhD 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… Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy NCT06689800NA1 site📍 St Louis Washington University School of Medicine · Tests: Let's Get REAL HPC Offered for PRESERVE Expansion NCT07145411n/a4 sites📍 Birmingham📍 Detroit📍 Columbus Ossium Health, Inc. · Tests: Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors) National Longitudinal Cohort of Hematological Diseases NCT04645199n/a1 site📍 Tianjin Institute of Hematology & Blood Diseases Hospital, China European Rare Blood Disorders Platform (ENROL) NCT06250595n/a1 site📍 Barcelona Hospital Universitari Vall d'Hebron Research Institute · Tests: Collection of clinical and laboratory data from EHR.
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