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Conditions: Gaucher Disease
Interventions: AVR-RD-02; Drug
Lead sponsor: AVROBIO; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 4
States / cities: San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more

Conditions: Myocardial Infarction, Coronary Artery Disease
Interventions: Intra-coronary infusion; Drug
Lead sponsor: Emory University; Other
Eligibility: 18 Years to 75 Years
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2013
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Multiple Myeloma
Interventions: One Autologous Transplant, Non-Myeloablative Allogeneic Transplant, Second Autologous Transplant, Thalidomide, Dexamethasone, Observation; Procedure · Drug · Behavioral
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Up to 70 Years
Enrollment: 710 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2013
U.S. locations: 36
States / cities: Birmingham, Alabama • Phoenix, Arizona • Duarte, California + 28 more

Conditions: Severe Combined Immunodeficiency Disease, X-linked
Interventions: CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs; Genetic · Drug · Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 24 Months · Male only
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2034
U.S. locations: 3
States / cities: San Francisco, California • Memphis, Tennessee • Seattle, Washington

Conditions: Breast Neoplasm, Neoplasm Metastasis
Interventions: Monoclonal Antibody 3A1, Monoclonal Antibody 95-5-49, Monoclonal Antibody 95-6-22, Baxter Isolex 3001 Stem Cell Selection System, Ceprate SC; Drug · Device
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 107 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1996 – 2014
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: MPS-IH (Hurler Syndrome)
Interventions: Experimental: OTL-203, Active Comparator: Allo-HSCT; Genetic
Lead sponsor: Orchard Therapeutics; Industry
Eligibility: 28 Days to 30 Months
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2031
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Non-Hodgkin Lymphoma, Hodgkin Disease
Interventions: G-CSF plus plerixafor, rituximab; Drug · Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Severe Combined Immunodeficiency
Interventions: Gene-Transduced Autologous CD34+ Stem Cells; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Months to 20 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Liver Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions: filgrastim, carboplatin, cyclophosphamide, etoposide, autologous bone marrow transplantation, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: Up to 35 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2005
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Lymphoma
Interventions: bleomycin sulfate, carmustine, cyclophosphamide, dacarbazine, doxorubicin hydrochloride, etoposide, vinblastine, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: SWOG Cancer Research Network; Network
Eligibility: 15 Years to 65 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2005
U.S. locations: 47
States / cities: Birmingham, Alabama • La Jolla, California • San Francisco, California + 29 more

Conditions: Chronic Granulomatous Disease
Interventions: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 55 Years · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2014
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Glioblastoma Multiforme (WHO Grade IV), Anaplastic Astrocytoma (WHO Grade III)
Interventions: 06Benzylguanine, MGMT P140K, Meltenyi CliniMacs; Drug · Genetic · Device
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 5 Years to 55 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2012
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Anemia, Sickle Cell
Interventions: Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Duarte, California

Conditions: Leukemia
Interventions: filgrastim, cyclophosphamide, dexamethasone, etoposide, peripheral blood stem cell transplantation, radiation therapy; Biological · Drug · Procedure + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 17 Years to 65 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1995 – 2002
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Transfusion Dependent Beta-thalassemia
Interventions: ST-400 Investigational product; Genetic
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 6
States / cities: Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more

Conditions: Relapsing-Remitting Multiple Sclerosis
Interventions: Granulocyte-colony stimulating factor (G-CSF) and prednisone, Carmustine, etoposide, cytarabine, and melphalan (BEAM), Autologous hematopoietic stem cell transplant; Drug · Procedure
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 60 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2015
U.S. locations: 4
States / cities: Columbus, Ohio • Houston, Texas • Seattle, Washington

Conditions: Systemic Lupus Erythematosus
Interventions: Leukapheresis, Non-myeloablative high dose immunosuppressive therapy conditioning (HDIT), Autologous CD34+HPC transplantation (HSCT), Plasmapheresis, Rabbit anti-thymocyte globulin, Methylprednisolone, Growth colony stimulating factor (G-CSF), Corticosteroids, Mycophenolate mofetil, Azathioprine, Intravenous immunoglobulin, Methotrexate, Rituximab, Leflunomide; Procedure · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 60 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2005
U.S. locations: 5
States / cities: La Jolla, California • Los Angeles, California • Chicago, Illinois + 2 more

Conditions: Beta-Thalassemia
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania

Conditions: Relapsing Multiple Sclerosis, Relapsing Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis
Interventions: Autologous Hematopoietic Stem Cell Transplantation, Best Available Therapy (BAT); Procedure · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 55 Years
Enrollment: 156 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2029
U.S. locations: 23
States / cities: Palo Alto, California • Aurora, Colorado • Evanston, Illinois + 18 more

Conditions: X-linked Severe Combined Immunodeficiency (XSCID)
Interventions: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 50 Years · Male only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Multiple Myeloma, Amyloidosis
Interventions: Melphalan, Pegfilgrastim, Autologous Hematopoietic Progenitor Cell Transplant; Device · Drug · Procedure
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years to 75 Years
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Lysosomal Storage Diseases, Cystinosis
Interventions: CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST); Genetic
Lead sponsor: University of California, San Diego; Other
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: La Jolla, California

Conditions: Lymphoma, Non-Hodgkin, Hodgkin Disease
Interventions: AMD3100, G-CSF, Apheresis; Drug · Procedure
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 18 Years to 75 Years
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Lymphoma
Interventions: rituximab, bortezomib, carmustine, cisplatin, cyclophosphamide, cytarabine, dexamethasone, doxorubicin hydrochloride, etoposide, melphalan, thalidomide, autologous-autologous tandem hematopoietic stem cell transplantation, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: SWOG Cancer Research Network; Network
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2008
U.S. locations: 29
States / cities: Hartford, Connecticut • Beech Grove, Indiana • Richmond, Indiana + 21 more

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Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Intra-coronary Infusion of Bone Marrow Derived Autologous CD34+ Selected Cells in Patients With Acute Myocardial Infarction

Stem Cell Transplantation in Individuals With Multiple Myeloma (BMT CTN 0102)

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

Chemotherapy and Progenitor Cell Transplantation to Treat Inflammatory Breast Cancer

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

The Effect of Rituximab on Mobilization With AMD3100 (Plerixafor) Plus G-CSF in Patients With Relapsed or Refractory Non-Hodgkin Lymphoma (NHL) or Hodgkin Disease (HD)

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

Peripheral Stem Cell Transplantation Plus Chemotherapy in Treating Patients With Malignant Solid Tumors

S9901 Combination Chemotherapy With or Without Peripheral Stem Cell Transplantation in Treating Men With Stage III or Stage IV Hodgkin's Disease

Gene Therapy for Chronic Granulomatous Disease

Treatment of Newly Diagnosed Brain Tumors With Chemotherapy and Radiation Using Cells Modified for Chemoprotection and an Experimental Drug to Decrease the Tumor Cell Resistance to Chemotherapy

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

High-Dose Chemotherapy Followed by Total-Body Irradiation and Peripheral Stem Cell Transplantation in Treating Patients With Chronic Lymphocytic Leukemia

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study

Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant (LIST)

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant.

Stem Cell Gene Therapy for Cystinosis

Intravenous AMD3100 for Collection of Autologous Peripheral Blood Stem Cells in Patients With Lymphoma

S0629, Observation or Combination Chemotherapy, Bortezomib, Thalidomide, and Rituximab Followed By Two Autologous Peripheral Blood Stem Cell Transplants in Treating Patients With Waldenstrom Macroglobulinemia