Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 8, 2026, 5:27 AM EDT

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Showing 1–24 of 52 matching trials from the live ClinicalTrials.gov search.
Conditions
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
Interventions
rivogenlecleucel, rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
3 Months to 21 Years
U.S. locations
2
States / cities
Los Angeles, California • Palo Alto, California
Terminated Phase 2 Interventional Results available

Reduced Intensity AlloTransplant For Osteopetrosis

NCT00638820
Conditions
Osteopetrosis
Interventions
Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 45 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Minneapolis, Minnesota
Terminated Not applicable Interventional Results available

Treatment of Sickle Cell Anemia With Stem Cell Transplant

NCT01350232
Conditions
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
Interventions
Fludarabine, Cytarabine, Cellular Infusions, Total Body Irradiation, Cyclophosphamide, Bortezomib, Rituximab, Plasmapheresis
Drug · Device · Radiation + 1 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years to 45 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Refractory Multiple Myeloma
Interventions
fludarabine phosphate, melphalan, total-body irradiation, mycophenolate mofetil, cyclosporine, nonmyeloablative allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, laboratory biomarker analysis
Drug · Radiation · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Hematologic Neoplasms, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Multiple Myeloma
Interventions
haploidentical allogeneic PBSC transp
Other
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
Leukemia, Erythroblastic, Acute, Myelodysplastic Syndromes
Interventions
5-Azacytidine, Fludarabine, Melphalan, Alemtuzumab, Total Body Irradiation
Drug · Radiation
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
New York, New York
Conditions
Myelofibrosis
Interventions
Fludarabine, Melphalan +/- ATG
Drug
Lead sponsor
John Mascarenhas
Other
Eligibility
18 Years to 65 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
7
States / cities
Chicago, Illinois • Baltimore, Maryland • Buffalo, New York + 3 more
Conditions
Myeloproliferative Disorders, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, methotrexate, tacrolimus, nonmyeloablative allogeneic HSCT
Biological · Drug · Procedure
Lead sponsor
Northside Hospital, Inc.
Other
Eligibility
Up to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Myelodysplastic and Myeloproliferative Disorders, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Multiple Myeloma, Plasma Cell Dyscrasia, Lymphoproliferative Disorders, Hematologic Diseases
Interventions
Campath, Busulfan, Fludarabine, Hematopoietic stem cell infusion, FK-506
Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
Houston, Texas
Conditions
AML, MDS, Acute Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndromes
Interventions
Treosulfan, Fludarabine, Total Body Irradiation, Tacrolimus, Mycophenolate Mofetil, Cyclophosphamid, Stem Cell Infusion
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
2 Years to 75 Years
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Acute Myeloid Leukemia, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia, Acute Lymphoblastic Leukemia
Interventions
OrcaGraft (Orca-Q)
Biological
Lead sponsor
Orca Biosystems, Inc.
Industry
Eligibility
12 Years to 78 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
12
States / cities
Duarte, California • Sacramento, California • Stanford, California + 9 more
Conditions
Myeloid Malignancies, Lymphoid Malignancies
Interventions
NK Cell enriched-DLI only, NK-DLI + DUK-CPG-001
Biological
Lead sponsor
Cristina Gasparetto
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Epidermolysis Bullosa
Interventions
Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone marrow infusion, Tacrolimus, Mycophenolate Mofetil, Donor mesenchymal stem cell infusions, Busulfan
Drug · Radiation · Procedure + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 25 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Sickle Cell Disease, Beta Thalassemia-Major
Interventions
alemtuzumab (Campath IH), Fludarabine, Melphalan, Cyclosporine, Mycophenolate mofetil, Tacrolimus, Hematopoietic Stem Cell Transplantation
Drug · Biological
Lead sponsor
Northwell Health
Other
Eligibility
1 Year to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
New Hyde Park, New York
Conditions
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelodysplastic Syndromes, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin's Lymphoma, Multiple Myeloma, Myeloproliferative Syndromes, Hematological Diseases
Interventions
Allopurinol, Fludarabine, Cyclophosphamide, ATG, TBI, Tacrolimus, MMF, Peripheral Blood Stem Cells, Related or Unrelated Bone Marrow Cells
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 75 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
MDS
Interventions
Allogeneic Hematopoietic Cell Transplant, Hypomethylating Therapy / Best Supportive Care
Procedure
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
50 Years to 75 Years
Enrollment
384 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
37
States / cities
Duarte, California • Stanford, California • Gainesville, Florida + 28 more
Conditions
Allogeneic Hematopoietic Cell Transplantation (HCT), Advanced Hematologic Malignancies, Acute Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndromes, Myeloproliferative Disorders
Interventions
Purified regulatory T-cells (Treg) plus CD34+ HSPC, Fludarabine, Melphalan, CliniMACS CD34 Reagent System, Tacrolimus, Cyclophosphamide, Plerixafor, Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent, Thiotepa, Mycophenolate Mofetil (MMF), Ruxolitinib, Sirolimus
Drug · Device
Lead sponsor
Stanford University
Other
Eligibility
18 Years to 75 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2028
U.S. locations
1
States / cities
Stanford, California
Conditions
Hematologic Malignancies, Graft vs Host Disease
Interventions
reduced intensity, related donor stem cell transplant, tacrolimus (standard GVHD prophylaxis), mycophenolate (standard GVHD prophylaxis), vorinostat
Procedure · Drug
Lead sponsor
Pavan Reddy, MD
Other
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Ann Arbor, Michigan • St Louis, Missouri
Completed Phase 3 Interventional Results available

Stem Cell Transplantation for Sickle Cell Anemia

NCT01877837
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Fludarabine, Melphalan, Stem Cells
Drug · Procedure
Lead sponsor
Hackensack Meridian Health
Other
Eligibility
2 Years to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2021
U.S. locations
1
States / cities
Hackensack, New Jersey
Conditions
Neuroblastoma
Interventions
Related donor transplant, Cord blood transplant, Busulfan, Thymoglobulin, Fludarabine
Procedure · Drug
Lead sponsor
Columbia University
Other
Eligibility
Up to 30 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
New York, New York
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Conditions
Immunodeficiencies, Immune Dysregulation Syndromes
Interventions
Apha/beta T and CD19+ cell depletion using CliniMACS device
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Bone Marrow Failure Syndromes, Immunodeficiencies, Immune Dysregulation Syndromes
Interventions
CliniMACs device
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 22 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania