Latest Clinical Trials
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity National Cancer Institute (NCI) NA
RECRUITING
(Sep 22, 2026)
PHASE2 Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy National Institute of Allergy and Infectious Diseases (NIAID) NA
ENROLLING BY INVITATION
(Sep 21, 2026)
PHASE1, PHASE2 Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) National Institute of Allergy and Infectious Diseases (NIAID) NA
RECRUITING
(Sep 18, 2026)
PHASE1, PHASE2 Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies National Cancer Institute (NCI) NA
RECRUITING
(Sep 16, 2026)
PHASE2 Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM National Institute of Allergy and Infectious Diseases (NIAID) NA
ENROLLING BY INVITATION
(Sep 15, 2026)
PHASE1, PHASE2 Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies National Cancer Institute (NCI) NA
RECRUITING
(Sep 10, 2026)
PHASE1, PHASE2 Allogeneic Hematopoietic Cell Transplantation for Peripheral T Cell Lymphoma National Cancer Institute (NCI) NA
ACTIVE NOT RECRUITING
(Sep 9, 2026)
PHASE2 pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD) National Institute of Allergy and Infectious Diseases (NIAID) NA
ENROLLING BY INVITATION
(Sep 9, 2026)
PHASE1, PHASE2 Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations National Cancer Institute (NCI) NA
RECRUITING
(Sep 4, 2026)
PHASE2 Therapy for Children With Advanced Stage Neuroblastoma St. Jude Children's Research Hospital NA
COMPLETED
(Sep 1, 2026)
PHASE2 Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia St. Anna Kinderkrebsforschung NA
RECRUITING
(Sep 1, 2026)
PHASE2, PHASE3 Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) St. Jude Children's Research Hospital NA
ACTIVE NOT RECRUITING
(Sep 1, 2026)
PHASE1, PHASE2 A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies Ossium Health, Inc. NA
ACTIVE NOT RECRUITING
(Sep 1, 2026)
PHASE1, PHASE2 High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly M.D. Anderson Cancer Center NA
RECRUITING
(Sep 1, 2026)
PHASE2 ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia Children's Hospital of Philadelphia NA
RECRUITING
(Sep 1, 2026)
PHASE1, PHASE2 BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS BlueSphere Bio, Inc NA
TERMINATED
(Sep 1, 2026)
PHASE1, PHASE2 Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) National Cancer Institute (NCI) NA
NOT YET RECRUITING
(Sep 1, 2026)
PHASE2 Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers National Cancer Institute (NCI) NA
NOT YET RECRUITING
(Aug 21, 2026)
PHASE2 Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency National Cancer Institute (NCI) NA
ACTIVE NOT RECRUITING
(Aug 20, 2026)
PHASE2 Studies of Temozolomide in Combination With Topotecan in Refractory and Relapsed Paediatric Solid Tumours Gustave Roussy, Cancer Campus, Grand Paris NA
COMPLETED
(Aug 1, 2026)
PHASE2 Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders Children's Hospital of Philadelphia NA
COMPLETED
(Aug 1, 2026)
PHASE2 Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma City of Hope Medical Center NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE1 The Ruxo-BEAT Trial in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia RWTH Aachen University NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE2 Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant M.D. Anderson Cancer Center NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE2 A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease Vertex Pharmaceuticals Incorporated NA
COMPLETED
(Aug 1, 2026)
PHASE2, PHASE3 Allogeneic Hematopoietic Stem Cell Transplantation With Briquilimab-Based Conditioning in Participants With GATA2 Deficiency National Cancer Institute (NCI) NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE2 HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis Center for International Blood and Marrow Transplant Research NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE2 A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006) Merck Sharp & Dohme LLC NA
ACTIVE NOT RECRUITING
(Aug 1, 2026)
PHASE3 Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML Washington University School of Medicine NA
RECRUITING
(Aug 1, 2026)
PHASE1, PHASE2 Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes Fred Hutchinson Cancer Center NA
RECRUITING
(Aug 1, 2026)
PHASE2 Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies Sawa Ito, MD NA
RECRUITING
(Aug 1, 2026)
PHASE1 Allogeneic ORCA-Q Stem Cell Transplant for the Treatment of Relapsed and Refractory High Risk Multiple Myeloma University of California, Davis NA
NOT YET RECRUITING
(Aug 1, 2026)
PHASE1 26DT052; Scaling Busulfan Dose to Body Surface Area in Children Children's Hospital of Philadelphia NA
NOT YET RECRUITING
(Aug 1, 2026)
PHASE1 Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease National Institute of Allergy and Infectious Diseases (NIAID) NA
RECRUITING
(Jul 29, 2026)
PHASE1, PHASE2 Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study National Institute of Allergy and Infectious Diseases (NIAID) NA
RECRUITING
(Jul 29, 2026)
PHASE1, PHASE2 A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome National Cancer Institute (NCI) NA
RECRUITING
(Jul 8, 2026)
PHASE2 Stem Cell Gene Therapy for Cystinosis University of California, San Diego NA
COMPLETED
(Jul 1, 2026)
PHASE1, PHASE2 Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS M.D. Anderson Cancer Center NA
RECRUITING
(Jul 1, 2026)
PHASE2, PHASE3 A Phase 1/3 Study of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation TScan Therapeutics, Inc. NA
RECRUITING
(Jul 1, 2026)
PHASE1 Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease Daniel Bauer NA
NOT YET RECRUITING
(Jul 1, 2026)
PHASE1 Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia Prince of Wales Hospital, Shatin, Hong Kong NA
NOT YET RECRUITING
(Jul 1, 2026)
PHASE2 Sorafenib, Busulfan and Fludarabine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia Undergoing Donor Stem Cell Transplant M.D. Anderson Cancer Center NA
ACTIVE NOT RECRUITING
(Jun 1, 2026)
PHASE1, PHASE2 Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders Masonic Cancer Center, University of Minnesota NA
RECRUITING
(Jun 1, 2026)
PHASE2 A Study Evaluating Temferon in Patients With Glioblastoma & Unmethylated MGMT Genenta Science NA
TERMINATED
(Jun 1, 2026)
PHASE1, PHASE2 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) Vertex Pharmaceuticals Incorporated NA
ACTIVE NOT RECRUITING
(Jun 1, 2026)
PHASE3 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Vertex Pharmaceuticals Incorporated NA
ACTIVE NOT RECRUITING
(Jun 1, 2026)
PHASE3 Human Leukocyte Antigen (HLA) Mismatched Unrelated Allogeneic Hematopoietic Stem Cell Transplantation He Huang NA
RECRUITING
(Jun 1, 2026)
PHASE1 Allo HSCT for High Risk Hemoglobinopathies Masonic Cancer Center, University of Minnesota NA
RECRUITING
(Jun 1, 2026)
PHASE2 Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA Chinese PLA General Hospital NA
ENROLLING BY INVITATION
(Jun 1, 2026)
PHASE1, PHASE2 Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT Peking University People's Hospital NA
RECRUITING
(Jun 1, 2026)
PHASE2 Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists National Institute of Allergy and Infectious Diseases (NIAID) NA
RECRUITING
(May 12, 2026)
PHASE1, PHASE2 High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease National Institute of Allergy and Infectious Diseases (NIAID) NA
ACTIVE NOT RECRUITING
(May 8, 2026)
PHASE1, PHASE2 Busulfan, Etoposide, and Total-Body Irradiation in Treating Patients Undergoing Donor Stem Cell or Bone Marrow Transplant for Advanced Hematologic Cancer City of Hope Medical Center NA
COMPLETED
(May 1, 2026)
PHASE2 Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders City of Hope Medical Center NA
ACTIVE NOT RECRUITING
(May 1, 2026)
PHASE2 Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins NA
TERMINATED
(May 1, 2026)
PHASE2 Personalized NK Cell Therapy in CBT M.D. Anderson Cancer Center NA
RECRUITING
(May 1, 2026)
PHASE2 Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease City of Hope Medical Center NA
RECRUITING
(May 1, 2026)
PHASE1 Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant Fred Hutchinson Cancer Center NA
ACTIVE NOT RECRUITING
(May 1, 2026)
PHASE2 A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant Memorial Sloan Kettering Cancer Center NA
ACTIVE NOT RECRUITING
(May 1, 2026)
PHASE2 A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) David Williams NA
ACTIVE NOT RECRUITING
(May 1, 2026)
PHASE2 Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant City of Hope Medical Center NA
RECRUITING
(May 1, 2026)
PHASE1 FLUDARABINE-TREOSULFAN REDUCED INTENSITY CONDITIONING REGIMEN PRIOR HAPLOIDENTICAL STEM CELL TRANSPLANTATION WITH POST TRANSPLANTATION CYCLOPHOSPHAMIDE FOR OLDER AND/OR FRAIL PATIENTS WITH AML Institut Paoli-Calmettes NA
NOT YET RECRUITING
(May 1, 2026)
PHASE2 ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene San Rocco Therapeutics NA
ACTIVE NOT RECRUITING
(Apr 1, 2026)
PHASE1 Venetoclax and Sequential Busulfan, Cladribine, and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome M.D. Anderson Cancer Center NA
ACTIVE NOT RECRUITING
(Apr 1, 2026)
PHASE2 High-Dose Post-Transplant Cyclophosphamide and Bortezomib (CyBor) for the Prevention of Graft-versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) NYU Langone Health NA
COMPLETED
(Apr 1, 2026)
PHASE2 Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis Fred Hutchinson Cancer Center NA
ACTIVE NOT RECRUITING
(Apr 1, 2026)
PHASE2 Safety And Efficacy Of TKI Cessation For CML Patients With Stable Molecular Response In A Real World Population Baylor College of Medicine NA
ACTIVE NOT RECRUITING
(Apr 1, 2026)
PHASE2 Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease Mark Walters, MD NA
RECRUITING
(Apr 1, 2026)
PHASE1, PHASE2 Itacitinib for the Prevention of Graft Versus Host Disease M.D. Anderson Cancer Center NA
ACTIVE NOT RECRUITING
(Apr 1, 2026)
PHASE2 Ruxolitinib and Chidamide Intensified Bu/CY Conditioning Regimen Chinese PLA General Hospital NA
RECRUITING
(Apr 1, 2026)
PHASE2 Phase III Study Comparing GVHD Prophylaxis With ATG-thymoglobulin to ATLG-grafalon in Elderly Patients With Acute Myeloid Leukemia or Myelodysplasic Syndrome and Receiving an Allogeneic Hematopoietic Stem Cell Transplantation With a 10/10 HLA Matched Unrelated Donor Assistance Publique - Hôpitaux de Paris NA
RECRUITING
(Apr 1, 2026)
PHASE3 Gene Editing For Sickle Cell Disease St. Jude Children's Research Hospital NA
RECRUITING
(Apr 1, 2026)
PHASE1 Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML/MDS Institute of Hematology and Blood Transfusion, Czech Republic NA
RECRUITING
(Apr 1, 2026)
PHASE1, PHASE2 Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector David Williams NA
COMPLETED
(Mar 1, 2026)
PHASE1, PHASE2 Fludarabine and Busulfan vs. Fludarabine, Busulfan and Total Body Irradiation Hackensack Meridian Health NA
TERMINATED
(Mar 1, 2026)
PHASE3 Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS) Fred Hutchinson Cancer Center NA
RECRUITING
(Mar 1, 2026)
PHASE2 Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders Memorial Sloan Kettering Cancer Center NA
RECRUITING
(Mar 1, 2026)
PHASE2 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease Vertex Pharmaceuticals Incorporated NA
RECRUITING
(Mar 1, 2026)
PHASE3 MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies Masonic Cancer Center, University of Minnesota NA
RECRUITING
(Mar 1, 2026)
PHASE2 Study on the Efficacy and Safety of the TmBU Conditioning Regimen in High-risk or Relapsed/Refractory Acute Leukemia The First Affiliated Hospital of Soochow University NA
RECRUITING
(Mar 1, 2026)
PHASE2 A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation Center for International Blood and Marrow Transplant Research NA
RECRUITING
(Mar 1, 2026)
PHASE2 
