Bone Marrow Failure Disorders: all 91 studies
Page 1 of 1. Open to participants first (29), then studies with results posted, then the rest; newest registration first within each.
- A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard TreatmentNot yet recruitingNCT07786922Phase 2/3
- The MedMemos Study: Promoting Adherence to Medications in Pediatric Bone Marrow Transplant PatientsRecruitingNCT07718204Not applicable
- A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure SyndromesRecruitingNCT07712562Phase 1/2
- Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant DiseasesNot yet recruitingNCT07660783Phase 2
- Stem Cell Mobilization and Apheresis for Life-threatening Blood DisordersRecruitingNCT07585136Phase 1
- CK0801 for Treatment of Aplastic AnemiaNot yet recruitingNCT07499102Phase 2
- Prenatal Transplantation for Fetuses With Fanconi AnemiaNot yet recruitingNCT07408583Phase 1/2
- The HOPE Biobank Resource (BMT CTN 2402 HOPE)RecruitingNCT07227155
- Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research StudyEnrolling by invitationNCT07102849
- Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCTRecruitingNCT06839456Phase 1/2
- Nucleoside Therapy in Patients With Telomere Biology DisordersRecruitingNCT06817590Phase 1
- CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System DiseasesRecruitingNCT06787560Early Phase 1
- The Role of Brain-Bone Marrow-Gut Interaction Following Major TraumaRecruitingNCT06606119
- A Study of Emapalumab for Pediatric Aplastic AnemiaRecruitingNCT06430788Phase 2
- European Rare Blood Disorders Platform (ENROL)RecruitingNCT06250595
- Shwachman Diamond Syndrome Registry and StudyRecruitingNCT06056908
- Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure DisordersRecruitingNCT05794425Phase 1/2
- Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)RecruitingNCT05196789
- French National Registry of Bone Marrow FailuresRecruitingNCT04781790
- Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant DisordersRecruitingNCT04644016Phase 2
- Haploidentical HCT for Severe Aplastic AnemiaRecruitingNCT04558736Phase 2
- TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in ChildrenRecruitingNCT04356469Phase 2
- Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow FailuresRecruitingNCT04232085Phase 2
- Alpha/Beta TCD HCT in Patients With Inherited BMF DisordersRecruitingNCT03579875Phase 2
- Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSCAvailableNCT03145545
- Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant DisordersRecruitingNCT03128996Phase 1/2
- Pre-myeloid Cancer and Bone Marrow Failure Clinic StudyRecruitingNCT02958462Not applicable
- Investigation of the Genetics of Hematologic DiseasesRecruitingNCT02720679
- Expanded Access Protocol Using CD3+/CD19+ Depleted PBSCRecruitingNCT02356653Early Phase 1
- Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 DepletionActive, not recruitingNCT05236764Not applicableResults posted
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/2Results posted
- Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haplo-HCTCompletedNCT02988466Phase 2Results posted
- NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic AnemiaCompletedNCT02833805Phase 2Results posted
- UCB Transplant for Hematological Diseases Using a Non Myeloablative PrepCompletedNCT02722668Phase 2Results posted
- Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable DiseasesCompletedNCT02277639Phase 2Results posted
- Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic AnemiaCompletedNCT02224872Phase 2Results posted
- Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic DiseasesTerminatedNCT02145039Not applicableResults posted
- CD34+ Stem Cell Infusion to Augment Graft FunctionTerminatedNCT01856582Phase 2Results posted
- Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell TransplantationTerminatedNCT01596699Phase 2Results posted
- Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan AnemiaTerminatedNCT01464164Phase 1/2Results posted
- T-cell Depleted Alternative Donor TransplantationTerminatedNCT00968864Phase 2Results posted
- Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersCompletedNCT00919503Phase 2Results posted
- Purine Analog-Based Conditioning in Patients With Severe Aplastic AnemiaCompletedNCT00427336Not applicableResults posted
- Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan AnemiaCompletedNCT00229619Phase 2Results posted
- Stem Cell Transplant for Bone Marrow Failure SyndromesCompletedNCT00176878Phase 2/3Results posted
- Methadone as an Alternative Treatment for Children Underdoing HSCTSuspendedNCT06940570Phase 3
- MNCs for Bone Marrow Failure After Malignant Diseases Children ChemotherapyStatus unknownNCT05628272Not applicable
- The Use of Eltrombopag Post HSCT in BMFDCompletedNCT05466201Phase 2/3
- Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)CompletedNCT04965597Phase 2
- Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)CompletedNCT04959188
- The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.Status unknownNCT04819607
- Response to SARS-CoV-2 Vaccine in Stem Cell Transplant and Cellular Therapy PatientsCompletedNCT04723706
- Revlimid® Capsules General Drug Use-results Surveillance (Relapsed or Refractory FL and MZL)CompletedNCT04618081
- A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisTerminatedNCT04525352Phase 1
- TPO-Mimetic Use in Children for Hematopoietic FailureCompletedNCT04478227Early Phase 1
- Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene TherapyActive, not recruitingNCT04437771
- Gene Therapy for Fanconi Anemia, Complementation Group AActive, not recruitingNCT04248439Phase 2
- Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow FailureActive, not recruitingNCT04211714Phase 1
- Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype AActive, not recruitingNCT04069533Phase 2
- Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell TransplantActive, not recruitingNCT03622788Phase 1/2
- Identification of Autoantibodies and Autoantigens in the Cerebrospinal Fluid of Patients With Spinal Cord TraumaWithdrawnNCT03249129
- Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot StudyWithdrawnNCT03086252Not applicable
- Fludarabine Based RIC for Bone Marrow Failure SyndromesActive, not recruitingNCT02928991Early Phase 1
- The Impact of Red Cell Age on Product Utilization in the Chronically Transfused Outpatient PopulationStatus unknownNCT02393508Phase 3
- Genetic Variation and Variability in Posaconazole Pharmacokinetics in ChildrenCompletedNCT02358499Phase 1
- Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant DiseasesCompletedNCT02349906Phase 2
- Memory T-cell Infusion to Improve Immunity After TCR-alpha/Beta Depleted Hematopoietic Stem Cell TransplantationCompletedNCT02337595Phase 1/2
- CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell TransplantActive, not recruitingNCT02061800Phase 1/2
- Nandrolone Decanoate in the Treatment of TelomeropathiesCompletedNCT02055456Phase 1/2
- CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationActive, not recruitingNCT01966367Phase 1/2
- Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)Status unknownNCT01760096Phase 2
- Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood TransplantationStatus unknownNCT01757145Phase 2
- Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaActive, not recruitingNCT01659606Phase 2
- Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal HemoglobinuriaStatus unknownNCT01642979Phase 2
- Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesWithdrawnNCT01419704Phase 1/2
- Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell TransplantNo longer availableNCT01200017
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure SyndromesCompletedNCT01192425
- Peripheral Blood Stem Cell Transplantation Using the CliniMACS DeviceCompletedNCT01071226Not applicable
- Unrelated Donor Transplant for Malignant and Non-Malignant DisordersTerminatedNCT01050439Phase 2
- CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor TransplantCompletedNCT01049854Phase 2
- Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesCompletedNCT01019876Phase 2/3
- Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic DisordersCompletedNCT00897260Not applicable
- Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCTCompletedNCT00774527Phase 3
- Identifying Characteristics of Bone Marrow Failure SyndromesStatus unknownNCT00315419
- Improving Immunosuppressive Treatment for Patients With Severe Aplastic AnemiaCompletedNCT00061360Phase 2
- Collection of Biological Samples From Patients With Hepatitis-Associated Aplastic AnemiaCompletedNCT00050115
- Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic AnemiaCompletedNCT00005935Phase 2
- Combination Therapy of Severe Aplastic AnemiaCompletedNCT00001964Phase 2
- Gene Therapy for the Treatment of Fanconi's Anemia Type CCompletedNCT00001399Phase 1
- Stem Cell Factor Medication for Aplastic AnemiaCompletedNCT00001398Phase 1
- Collection of Blood From Patients With PancytopeniaCompletedNCT00001214