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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.

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30 recruiting trials

for Inherited Bone Marrow Failure Syndrome

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency NCT06090669Phase 11 site📍 Bethesda National Cancer Institute (NCI) · Tests: TruSight Oncology, imatinib 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) 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 Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer NCT01137643n/a1 site📍 Chapel Hill UNC Lineberger Comprehensive Cancer Center · Tests: biologic sample preservation procedure, cytology specimen collection procedure Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415Phase 21 site📍 Pittsburgh Paul Szabolcs · Tests: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa 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. Nucleoside Therapy in Patients With Telomere Biology Disorders NCT06817590Phase 11 site📍 Boston Suneet Agarwal · Tests: deoxycytidine, deoxythymidine High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) NCT07205523n/a1 site📍 Xining Yigeng Cao,MD,PhD Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform NCT06999954n/a1 site📍 Woburn Shwachman-Diamond Syndrome Alliance Inc Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia NCT05687149n/a1 site📍 Bethesda National Cancer Institute (NCI) Natural History of Acquired and Inherited Bone Marrow Failure Syndromes NCT05012111n/a1 site📍 Bethesda National Heart, Lung, and Blood Institute (NHLBI) Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia NCT07901959Phase 12 sites📍 Minneapolis📍 New York Rockefeller University · Tests: Metformin, Naproxen Sodium 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 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 Familial Investigations of Childhood Cancer Predisposition NCT03050268n/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 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 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,… 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 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 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 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 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) 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 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) The HOPE Biobank Resource (BMT CTN 2402 HOPE) NCT07227155n/a14 sites📍 Duarte📍 Gainesville📍 Atlanta Medical College of Wisconsin Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes NCT05436587n/a1 site📍 Sohag Sohag University · Tests: The whole-exome sequencing 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 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… 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
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