Recruiting studies: Congenital Bone Marrow Failure Syndromes

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Phase N/A · 6 location(s) · Medical College of Wisconsin · NCT07227155 AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Phase 2 · 1 location(s) · Mitchell Cairo · NCT04099966 Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Phase N/A · 1 location(s) · National Heart, Lung, and Blood Institute (NHLBI) · NCT05012111 Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia
Phase N/A · 1 location(s) · National Cancer Institute (NCI) · NCT06744283 Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
Phase 1 · 1 location(s) · Children's Hospital Medical Center, Cincinnati · NCT07628972 Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes
Phase N/A · 1 location(s) · Sohag University · NCT05436587 Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
Phase N/A · 1 location(s) · Northwell Health · NCT07186179 Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Phase 1/Phase 2 · 4 location(s) · Washington University School of Medicine · NCT03128996 HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Phase 2 · 3 location(s) · Children's Hospital Medical Center, Cincinnati · NCT02143830 Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
EARLY_Phase 1 · 1 location(s) · Children's Hospital of Philadelphia · NCT02356653 Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
NA · 3 location(s) · Mayo Clinic · NCT02958462 High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)
Phase N/A · 1 location(s) · Yigeng Cao,MD,PhD · NCT07205523 Nucleoside Therapy in Patients With Telomere Biology Disorders
Phase 1 · 1 location(s) · Suneet Agarwal · NCT06817590 A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combine
Phase 2 · 2 location(s) · Franziska Wachter · NCT05796570 Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Phase 2 · 1 location(s) · Masonic Cancer Center, University of Minnesota · NCT03579875 Familial Investigations of Childhood Cancer Predisposition
Phase N/A · 1 location(s) · St. Jude Children's Research Hospital · NCT03050268 HPC Offered for PRESERVE Expansion
Phase N/A · 4 location(s) · Ossium Health, Inc. · NCT07145411 National Longitudinal Cohort of Hematological Diseases
Phase N/A · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT04645199 French National Registry of Bone Marrow Failures
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT04781790 Role of Acetaldehyde in the Development of Oral Cancer
NA · 1 location(s) · Masonic Cancer Center, University of Minnesota · NCT05973656 Cancer in Inherited Bone Marrow Failure Syndromes
Phase N/A · 2 location(s) · National Cancer Institute (NCI) · NCT00027274 TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Phase 2 · 1 location(s) · Johns Hopkins All Children's Hospital · NCT04356469 Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Phase N/A · 1 location(s) · Paul Szabolcs · NCT04528355 Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Phase 1/Phase 2 · 1 location(s) · Children's Hospital of Philadelphia · NCT06839456 FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03351868 T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Phase 1/Phase 2 · 1 location(s) · Paul Szabolcs · NCT03653338 MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients
Phase N/A · 1 location(s) · University of Minnesota · NCT07649031 Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
Phase N/A · 1 location(s) · UNC Lineberger Comprehensive Cancer Center · NCT01137643 European Rare Blood Disorders Platform (ENROL)
Phase N/A · 1 location(s) · Hospital Universitari Vall d'Hebron Research Institute · NCT06250595 Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient D
Phase 1 · 2 location(s) · Apriligen, Inc. · NCT07476183

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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.