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Showing 625–648 of 2,559 matching trials from the live ClinicalTrials.gov search.
Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
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
Graft vs Host Disease, Myelodysplastic Syndromes, Leukemia, Leukemia, Myeloid, Leukemia, Myelomonocytic, Chronic, Leukemia, Lymphocytic, Lymphoma, Lymphoma, Mantle-cell, Lymphoma, Non-Hodgkin, Hodgkin Disease
Interventions
RFT5-SMPT-dgA, Isolex system
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Graft Versus Host Disease, Myelodysplastic Syndrome, Myeloproliferative Neoplasm
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Biospecimen Collection, Bone Marrow Biopsy, Computed Tomography, Cyclophosphamide, Echocardiography, Fludarabine, Melphalan, Multigated Acquisition Scan, Questionnaire Administration, Tacrolimus, Vedolizumab
Procedure · Drug · Other + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Duarte, California
Conditions
Multiple Myeloma, Non-Hodgkin's Lymphoma
Interventions
Enterade, Placebo, Standard Supportive Care
Dietary Supplement · Other
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Leukemia
Interventions
filgrastim, recombinant interferon alfa, sargramostim, therapeutic autologous lymphocytes, carmustine, cyclophosphamide, etoposide, gemcitabine hydrochloride, melphalan, bone marrow ablation with stem cell support, in vitro-treated peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
Up to 120 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
Interventions
filgrastim, graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine, fludarabine phosphate, mycophenolate mofetil, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 45 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Multiple Myeloma, CLL / SLL, Non Hodgkin's Lymphoma, Waldenstroms, Mantle Cell Lymphoma
Interventions
Fludarabine, Total Body Irradiation, Infusion of Stem Cells
Drug · Radiation · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years to 70 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Ganglioneuroblastoma, High Risk Neuroblastoma
Interventions
Autologous Hematopoietic Stem Cell Transplantation, Carboplatin, Cisplatin, Cyclophosphamide, Dexrazoxane, Dinutuximab, Doxorubicin, Etoposide, External Beam Radiation Therapy, Isotretinoin, Melphalan, Sargramostim, Thiotepa, Topotecan, Vincristine
Procedure · Drug · Biological + 1 more
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 30 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Boston, Massachusetts + 4 more
Conditions
Acute Myeloid Leukemia in Remission, Primary Myelofibrosis, Primary Myelofibrosis, Prefibrotic Stage, Secondary Acute Myeloid Leukemia, Secondary Myelofibrosis
Interventions
Allogeneic Bone Marrow Transplantation, Allogeneic Hematopoietic Stem Cell Transplantation, Fludarabine Phosphate, Laboratory Biomarker Analysis, Melphalan, Peripheral Blood Stem Cell Transplantation, Pharmacological Study, Ruxolitinib Phosphate, Sirolimus, Tacrolimus
Procedure · Drug · Other
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2015
U.S. locations
1
States / cities
Duarte, California
Conditions
Respiratory Syncytial Virus Infections
Interventions
JNJ-53718678 250 mg, Placebo, JNJ-53718678 125 mg
Drug
Lead sponsor
Janssen Sciences Ireland UC
Industry
Eligibility
13 Years to 75 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
9
States / cities
Los Angeles, California • Detroit, Michigan • Minneapolis, Minnesota + 6 more
Conditions
Non-Malignant Hematologic Disorders
Interventions
Melphalan, Thiotepa, Clofarabine, Fludarabine, Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®), CliniMACS reagents
Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year to 69 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
New York, New York
Conditions
Recurrent Melanoma, Stage IV Melanoma
Interventions
cyclophosphamide, fludarabine phosphate, therapeutic autologous lymphocytes, in vitro-treated peripheral blood stem cell transplantation, gp100 antigen, MART-1 antigen, incomplete Freund's adjuvant, filgrastim, aldesleukin
Drug · Biological · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent
Interventions
Myeloablative Chemotherapy, Stem Cell Rescue
Procedure
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2010
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm
Interventions
Best Practice, Interview, Survey Administration, Virtual Reality
Other · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Buffalo, New York
Conditions
Knee Osteoarthritis
Interventions
Arthroscopic debridement with stem cell transplantation, Arthroscopic debridement only
Procedure
Lead sponsor
University of Colorado, Denver
Other
Eligibility
35 Years to 70 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
3
States / cities
Santa Monica, California • Stanford, California • Denver, Colorado
Conditions
Leukemia
Interventions
5-azacitidine, Allogeneic Hematopoietic Cell Transplantation (HCT)
Drug · Procedure
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years to 68 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, melphalan, methylprednisolone, bone marrow ablation with stem cell support, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2006
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Multiple Myeloma
Interventions
IV busulfan, bortezomib, Autologous Hematopoietic Stem Cell Transplant (HSCT)
Drug · Procedure
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
11
States / cities
Tucson, Arizona • Atlanta, Georgia • Maywood, Illinois + 7 more
Conditions
Refractory Multiple Myeloma, Smoldering Multiple Myeloma, Stage I Multiple Myeloma, Stage II Multiple Myeloma, Stage III Multiple Myeloma
Interventions
total marrow irradiation, melphalan, peripheral blood stem cell transplantation, filgrastim, fluorescence in situ hybridization, cytogenetic analysis, cyclophosphamide, autologous-autologous tandem hematopoietic stem cell transplantation, lenalidomide
Radiation · Drug · Procedure + 2 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
Up to 70 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
Duarte, California
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
Drug · Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2013
U.S. locations
1
States / cities
New York, New York
Conditions
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Interventions
autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
Biological · Device
Lead sponsor
Daniel Bauer
Other
Eligibility
13 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Stem Cell Transplant Complications
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
60 Years to 85 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Omaha, Nebraska
Conditions
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan, Cyclophosphamide, Filgrastim, Fludarabine, Mycophenolate Mofetil, Sorafenib, Tacrolimus
Procedure · Drug · Biological
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Houston, Texas
Conditions
Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Acute Myelogenous Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome
Interventions
VCL-CB01, Phosphate-buffered Saline (PBS)
Biological · Other
Lead sponsor
Astellas Pharma Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
22
States / cities
Tucson, Arizona • Duarte, California • Denver, Colorado + 17 more