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Conditions: Granulomatous Disease, Chronic, X-linked
Interventions: Lentiviral G1XCGD Gene Therapy; Biological
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 23 Months and older · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2024
U.S. locations: 3
States / cities: Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts

Conditions: Graft Vs Host Disease, Graft-versus-host-disease
Interventions: CliniMacs®; Device
Lead sponsor: Christopher Dvorak; Other
Eligibility: Up to 30 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2035
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Hematologic Malignancies
Interventions: L-leucyl-L-leucine Methyl Ester (LLME), Fludarabine, Cytarabine, Cyclophosphamide, Tacrolimus, Mesna, Granulocyte Macrophage Colony-Stimulating Factor (GM-CSF), Hematopoietic stem cell transplantation (HSCT); Drug · Biological · Procedure
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University; Other
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria, Hodgkin's Lymphoma, Non-Hodgkin Lymphoma
Interventions: Systematic chemotherapy and antibodies, Allogeneic stem cell transplantation, Miltenyi CliniMACS; Drug · Procedure · Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 2 Years to 21 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Lymphoma
Interventions: filgrastim, busulfan, melphalan, thiotepa, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: OHSU Knight Cancer Institute; Other
Eligibility: Up to 70 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2016
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Chronic Granulomatous Disease
Interventions: Busulfan, Alemtuzumab, Cyclophosphamide, Sirolimus, Total Body Irradiation, Allogeneic peripheral blood stem cell; Drug · Radiation · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 4 Years to 65 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2034
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Kidney Transplantation, Kidney Disease, Kidney Failure
Interventions: Alemtuzumab, Mycophenolate mofetil, Sirolimus, Tacrolimus, Donor bone marrow stem cell infusion, Kidney transplant; Drug · Procedure
Lead sponsor: University of Miami; Other
Eligibility: 18 Years to 65 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Peripheral T-Cell Lymphoma
Interventions: Gemcitabine, Navelbine, Doxorubicin Hydrochloride Liposome Injection, Granulocyte-colony stimulating factor (G-CSF), Pegfilgrastim, Cyclophosphamide, Vincristine, Leucovorin, Methotrexate, Doxorubicin Hydrochloride, Cytarabine, Etoposide, Carmustine, Denileukin diftitox; Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 70 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2016
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Diffuse Large B Cell Lymphoma
Interventions: Blinatumomab, Autologous stem cell transplant, Carmustine, Etoposide, Cytarabine, Melphalan, Peripheral blood draws; Drug · Procedure
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Sickle Cell Disease
Interventions: ATG, fludarabine, cyclophosphamide, Total body irradiation, Stem cell infusion, Sirolimus, mycophenolate mofetil; Drug · Radiation · Procedure
Lead sponsor: University of Illinois at Chicago; Other
Eligibility: 16 Years to 60 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2029
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Acute Myeloid Leukemia, Myelodysplastic Syndromes
Interventions: Interferon gamma-1b, Donor Leukocyte Infusion (DLI); Drug · Biological
Lead sponsor: Sawa Ito, MD; Other
Eligibility: 18 Years and older
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 3
States / cities: St Louis, Missouri • Pittsburgh, Pennsylvania • Seattle, Washington

Conditions: Multiple Myeloma
Interventions: GPC-100, Propranolol, G-CSF; Drug
Lead sponsor: GPCR Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 10
States / cities: La Jolla, California • Gainesville, Florida • Indianapolis, Indiana + 7 more

Conditions: Sensorineural Hearing Loss
Interventions: Autologous Stem Cells; Genetic
Lead sponsor: James Baumgartner, MD; Other
Eligibility: 6 Weeks to 6 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Orlando, Florida

Conditions: Lymphoma, B-cell, Lymphoma, Large B-Cell, Diffuse, Lymphoma, Follicular, Grade 3b, Follicular Lymphoma, Grade 3b
Interventions: denintuzumab mafodotin, rituximab, ifosfamide, carboplatin, etoposide; Drug
Lead sponsor: Seagen Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 81 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 29
States / cities: Little Rock, Arkansas • Duarte, California • San Diego, California + 24 more

Conditions: Scleroderma, Systemic, Sclerosis, Autoimmune Disease
Interventions: mHSCT, cyclophosphamide; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 69 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2016
U.S. locations: 15
States / cities: Duarte, California • Los Angeles, California • Lexington, Kentucky + 10 more

Conditions: Sickle Cell Disease
Interventions: nula-cel Drug Product; Genetic
Lead sponsor: Kamau Therapeutics; Industry
Eligibility: 12 Years to 50 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 6
States / cities: Los Angeles, California • Palo Alto, California • St Louis, Missouri + 2 more

Conditions: Hematologic Malignancies
Interventions: Allogeneic donor TCRαβ+/CD19+ cell-depleted peripheral blood mononuclear cells; Device
Lead sponsor: University of Wisconsin, Madison; Other
Eligibility: 18 Years to 80 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: Sickle Cell Disease
Interventions: Plerixafor, Busulfan, BIVV003; Biological · Drug · Genetic
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 5
States / cities: Oakland, California • Sacramento, California • Atlanta, Georgia + 2 more

Conditions: Beta-Thalassemia
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania

Conditions: Leukemia, Solid Tumors
Interventions: Clinimacs Cell Separation System, conditioning chemotherapy, DLI; Device · Drug · Other
Lead sponsor: University of Wisconsin, Madison; Other
Eligibility: 6 Months to 25 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: CML (Chronic Myelogenous Leukemia), CLL (Chronic Lymphocytic Leukemia), AML (Acute Myelogenous Leukemia), Acute Lymphocytic Leukemia, MDS (Myelodysplastic Syndrome)
Interventions: Miltenyi system; Combination Product
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 75 Years
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Anemia, Sickle Cell
Interventions: Siplizumab, Exchange Transfusion, Total Body Irradiation, Stem Cell Infusion, Cyclophosphamide 50mg, Mesna, Sirolimus Oral Tablet, Rituximab or Biosimilar; Drug · Procedure
Lead sponsor: Columbia University; Other
Eligibility: 18 Years to 50 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: New York, New York

Conditions: Amyotrophic Lateral Sclerosis
Interventions: RAPA-501 Autologous T stem cells; Biological
Lead sponsor: Rapa Therapeutics LLC; Industry
Eligibility: 18 Years and older
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions: Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D); Biological · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 149 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2029
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

A Feasibility Study Using CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem Cell Transplant

Research Study to Determine if an Experimental Agent, LLME Can Decrease the Incidence and Severity of Graft-Versus-Host-Disease (GVHD) Following Blood (Hematopoietic) Stem Cell Transplantation

Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies

Busulfan, Melphalan, and Thiotepa in Treating Patients Who Are Undergoing an Autologous Stem Cell Transplant for Hodgkin's or Non-Hodgkin's Lymphoma

Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

Using Donor Stem Cells and Alemtuzumab to Prevent Organ Rejection in Kidney Transplant Patients

Treatment of PTCL With Aggressive Induction Therapy Followed by Autologous SCT Using Denileukin Diftitox (Ontak)

Blinatumomab Consolidation Post Autologous Stem Cell Transplantation in Patients With Diffuse Large B-Cell Lymphoma (DLBCL)

Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation

Ph2, Study to Assess the Safety and Efficacy of GPC 100 and Propranolol With and Without G-CSF for the Mobilization of Stem Cells in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplant

Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss

Treatment Study of Denintuzumab Mafodotin (SGN-CD19A) Plus RICE Versus RICE Alone for Diffuse Large B-Cell Lymphoma

Scleroderma: Cyclophosphamide or Transplantation

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Prophylactic TCRaB+ and CD19+ Depleted Donor Lymphocyte Infusion After Allogeneic Stem Cell Transplant in High-Risk Patients With Hematologic Malignancies

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Haploidentical Transplant With NK Cell Infusion for Pediatric Acute Leukemia and Solid Tumors

Stem Cell Transplantation for Patients With Cancers of the Blood

Siplizumab for Sickle Cell Disease Transplant

RAPA-501 Therapy for ALS

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis