- 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
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Showing 1–24
of 29
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia, Immune Dysregulation, Hemophagocytic Lymphohistiocytosis (HLH), Non Malignant Disorders
- Interventions
- ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
- Biological
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- 6 Months to 50 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2035
- U.S. locations
- 2
- States / cities
- Seattle, Washington
- Conditions
- Primary Immunodeficiency Disorders (PIDs)
- Interventions
- Leniolisib
- Drug
- Lead sponsor
- Pharming Technologies B.V.
- Industry
- Eligibility
- 12 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis, Macrophage Activation Syndrome, Hyperinflammatory Syndromes
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Pittsburgh, Pennsylvania
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF, STAT6 GOF
- Interventions
- Upadacitinib, Placebo
- Drug
- Lead sponsor
- Lisa Satter
- Other
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- St Louis, Missouri • New York, New York • Houston, Texas
- Conditions
- Long COVID-19, Long COVID
- Interventions
- Paxlovid, Ritonavir, Nirmatrelvir-matching placebo
- Drug
- Lead sponsor
- Kanecia Obie Zimmerman
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 964 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency, Primary T-cell Immunodeficiency Disorders
- Interventions
- Busulfan test dose, Fludarabine, Busulfan, Alemtuzumab, Total body Irradiation, Allogeneic HSCT, Tacrolimus (Tacro), Mycophenolate mofetil (MMF), Cyclophosphamide (Cytoxan)
- Drug · Radiation · Procedure
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 4 Years to 69 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Conditions
- Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Up to 99 Years
- Enrollment
- 10,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2027
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
- Interventions
- Hematopoietic stem cell transplant (HSCT)
- Biological
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 5 Years to 40 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Long COVID, Long Covid19
- Interventions
- Experimental: Paxlovid 25 day dosing, Experimental: Paxlovid 15 day dosing, Placebo Comparator: Control
- Drug
- Lead sponsor
- Kanecia Obie Zimmerman
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 963 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 68
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 51 more
- Conditions
- Asthma, Obesity, Metabolic Syndrome
- Interventions
- Bronchoscopy and Nasal Epithelium Brush Sampling
- Procedure
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2029
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Myeloid Malignancy, Bone Marrow Failure Syndrome, Transfusion-dependent Red Blood Cell (RBC) Defect, Congenital Immunodeficiency, Metabolic Disease, Severe Immune Dysregulation
- Interventions
- Alemtuzumab, Busulfan, Fludarabine, Clofarabine
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 3 Months to 30 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Myalgic Encephalomyelitis, Chronic Fatigue Syndrome
- Interventions
- Two-day cardiopulmonary exercise test
- Other
- Lead sponsor
- Ithaca College
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 173 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2023
- U.S. locations
- 3
- States / cities
- Torrance, California • Ithaca, New York • New York, New York
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Interventions
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction, protein expression analysis, reverse transcriptase-polymerase chain reaction, western blotting, chromatography, high performance liquid chromatography, immunoenzyme technique
- Genetic · Other
- Lead sponsor
- OHSU Knight Cancer Institute
- Other
- Eligibility
- 1 Year to 55 Years
- Enrollment
- 213 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1975 – 2016
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- IPEX
- Interventions
- CD4^LVFOXP3
- Biological
- Lead sponsor
- Bacchetta, Rosa, MD
- Other
- Eligibility
- 4 Months to 35 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2037
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Down Syndrome
- Interventions
- Not listed
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 1 Year to 120 Years
- Enrollment
- 700 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026 – 2040
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Common Variable Immunodeficiency (CVID)
- Interventions
- Leniolisib
- Drug
- Lead sponsor
- Pharming Technologies B.V.
- Industry
- Eligibility
- 12 Years to 75 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 5
- States / cities
- Aurora, Colorado • Bethesda, Maryland • Burlington, Massachusetts + 2 more
- 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
- Non-Neoplastic Hematologic and Lymphocytic Disorder
- Interventions
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
- Procedure · Biological · Drug + 2 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Up to 49 Years
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
- Conditions
- Non-Hodgkin Lymphoma, Lymphoma Non-Hodgkin, B-cell Lymphoma, Immune Dysregulation
- Interventions
- Not listed
- Lead sponsor
- Henry M. Jackson Foundation for the Advancement of Military Medicine
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 3
- States / cities
- Bethesda, Maryland • Portsmouth, Virginia
- Conditions
- Immunodeficiency, Hematopoietic Stem Cell Transplantation
- Interventions
- Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF), Tacrolimus, Busulfan, Total Body Irradiation (TBI), Fludarabine, Cyclophosphamide (CY), Post-Transplant Cyclophosphamide (PTCY)
- Procedure · Drug · Radiation
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania