- Conditions
- Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
- Interventions
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
- Biological
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 30 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Interventions
- Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
- Drug · Radiation · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 0 Years to 70 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
- Interventions
- Daclizumab
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
- Interventions
- CliniMACS
- Device
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 2
- States / cities
- Houston, Texas
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
- Interventions
- Abatacept
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Fanconi Anemia, Aplastic Anemia
- Interventions
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, therapeutic allogeneic lymphocytes, cyclophosphamide, fludarabine phosphate, methylprednisolone, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
- Biological · Drug · Procedure
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 4 Years to 75 Years
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2018
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2041
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fanconi's Anemia, Hematologic Disease
- Interventions
- Antithymocyte globulin, Cyclosporine
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Chronic Kidney Disease, Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkin's Lymphoma (NHL), Hodgkin Disease, Multiple Myeloma, Myelodysplastic Syndrome (MDS), Aplastic Anemia, AL Amyloidosis, Diamond Blackfan Anemia, Myelofibrosis, Myeloproliferative Disease, Sickle Cell Anemia, Autoimmune Diseases, Thalassemia
- Interventions
- Haploidentical Bone Marrow/Kidney
- Procedure
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2025
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura, Anemia, Hemolytic, Autoimmune, Autoimmune Neutropenia, Lupus Erythematosus, Systemic, Inflammatory Bowel Disease, Rheumatoid Arthritis
- Interventions
- sirolimus
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Year to 30 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2016
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Fanconi Anemia, Dyskeratosis Congenita
- Interventions
- danazol
- Drug
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 3 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Fanconi Anemia
- Interventions
- Specimen Donation
- Diagnostic Test
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Woburn, Massachusetts
- Conditions
- Neutropenia
- Interventions
- Mavorixafor, Placebo
- Drug
- Lead sponsor
- X4 Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 176 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
- Conditions
- Fanconi Anemia
- Interventions
- JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide, Fludarabine, Rituximab
- Drug · Device · Biological
- Lead sponsor
- Porteus, Matthew, MD
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Fanconi Anemia
- Interventions
- microarray analysis, biopsy
- Genetic · Procedure
- Lead sponsor
- OHSU Knight Cancer Institute
- Other
- Eligibility
- 1 Year to 55 Years
- Enrollment
- 90 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2002 – 2007
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Fanconi Anemia
- Interventions
- Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure, Myelodysplastic Syndromes (MDS), Myeloproliferative Disorders
- Interventions
- CIK cells, Cyclosporine, Mycophenolate Mofetil, Thymoglobulin, Total Lymphoid Irradiation (TLI)
- Drug · Radiation
- Lead sponsor
- Everett Meyer
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2017
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- RPS19 Deficient Diamond-Blackfan Anemia
- Interventions
- APR-2020
- Biological
- Lead sponsor
- Apriligen, Inc.
- Industry
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 2
- States / cities
- Stanford, California • Boston, Massachusetts
- Conditions
- Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
- Interventions
- Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Month to 25 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloid Malignancies, MDS, Inherited Bone Marrow Failure Syndrome, Myeloid Neoplasm, Aml
- Interventions
- Decitabine, Filgrastim
- Drug
- Lead sponsor
- Franziska Wachter
- Other
- Eligibility
- 1 Year to 39 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts
- Conditions
- Dyskeratosis Congenita, Telomere Biology Disorders
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
- Interventions
- A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
- Drug
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Network
- Eligibility
- Not listed
- Enrollment
- 99,999 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2041
- U.S. locations
- 142
- States / cities
- Birmingham, Alabama • Gilbert, Arizona • Phoenix, Arizona + 90 more
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Aplastic Anemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Essential Thrombocythemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Juvenile Myelomonocytic Leukemia, Mastocytosis, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Polycythemia Vera, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Anemia, Refractory Anemia With Ringed Sideroblasts, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Myelodysplastic Syndromes, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenström Macroglobulinemia
- Interventions
- Fludarabine, Busulfan, Total Body Irradiation (TBI), Donor Lymphocyte Infusion (DLI), Cyclophosphamide (CY), Tacrolimus, Mycophenolate mofetil, Allogeneic hematopoietic stem cell transplantation, Peripheral blood stem cell transplantation (PBSCT)
- Drug · Radiation · Biological + 2 more
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2022
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Fanconi Anemia
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland