Search | Clinical Trials Finder

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ClinicalTrials.gov public records Last synced Nov 14, 2026, 10:00 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 24 matching trials from the live ClinicalTrials.gov search.

Filters: Intervention: Gene-modified CD34+ cells Clear all

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: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 6
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Human Immunodeficiency Virus
Interventions: Busulfan, Cal-1 modified HSPC, Cal-1 modified CD4+ T lymphocytes; Drug · Biological
Lead sponsor: Calimmune, Inc.; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 2
States / cities: Los Angeles, California • San Francisco, California

Conditions: Leukemia, Myeloma, Myeloproliferative Diseases
Interventions: Fludarabine, Melphalan, Alemtuzumab, Stem Cell infusion, Tacrolimus, Mini Methotrexate, G-CSF, Donor Lymphocyte Infusion (DLI), AP1903, Methylprednisolone, Questionnaire; Drug · Procedure · Behavioral
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 18 Years to 65 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 4
States / cities: Charlotte, North Carolina • Philadelphia, Pennsylvania • Memphis, Tennessee + 1 more

Conditions: Infantile Malignant Osteopetrosis
Interventions: RP-L401; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 1 Month and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Sickle Cell Disease
Interventions: Plerixafor, Busulfan, Gene-modified CD34+ cells, Motixafortide; Drug · Biological
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 18 Years to 24 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Memphis, Tennessee

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: 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: Bone Marrow Suppression, Brain and Central Nervous System Tumors, Drug/Agent Toxicity by Tissue/Organ
Interventions: filgrastim, gene therapy, lomustine, procarbazine hydrochloride, vincristine sulfate, in vitro-treated peripheral blood stem cell transplantation; Procedure · Biological · Drug
Lead sponsor: Indiana University; Other
Eligibility: 5 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2011
U.S. locations: 2
States / cities: Indianapolis, Indiana • Boston, Massachusetts

Conditions: HIV
Interventions: SB-728mR-HSPC Infusion 3 days following busulfan conditioning; Genetic
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 18 Years to 75 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2027
U.S. locations: 5
States / cities: Duarte, California • Los Angeles, California • San Francisco, California + 1 more

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: Pyruvate Kinase Deficiency
Interventions: RP-L301; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years to 50 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: Stanford, California

Conditions: Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions: PM359; Biological
Lead sponsor: Prime Medicine, Inc.; Industry
Eligibility: 6 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 3
States / cities: Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee

Conditions: Pyruvate Kinase Deficiency
Interventions: RP-L301; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years to 55 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Leukocyte Adhesion Defect - Type I
Interventions: RP-L201; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 3 Months and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Fanconi Anemia
Interventions: Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells, Laboratory Biomarker Analysis, Leukapheresis, Methylprednisolone, Plerixafor, Prednisone; Procedure · Biological · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 4 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2024
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: AIDS-Related Hodgkin Lymphoma, AIDS-Related Non-Hodgkin Lymphoma, HIV Infection, Recurrent Hodgkin Lymphoma, Recurrent Non-Hodgkin Lymphoma
Interventions: Gene-Modified HIV-Protected Hematopoietic Stem Cells, Laboratory Biomarker Analysis, Transplant Conditioning; Biological · Other
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 18 Years to 66 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Sickle Cell Disease
Interventions: βAS3-FB vector transduced peripheral blood CD34+ cells; Biological
Lead sponsor: Donald B. Kohn, M.D.; Other
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2025
U.S. locations: 1
States / cities: Los Angeles, California

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: Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Lymphoma, Myelodysplastic Syndromes
Interventions: BPX-501 dose 1, Rimiducid, BPX-501 dose 2, BPX-501 dose 3, BPX-501 dose 4, SCT; Biological · Drug · Procedure
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2032
U.S. locations: 8
States / cities: Atlanta, Georgia • Buffalo, New York • New York, New York + 4 more

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: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 3
States / cities: New York, New York • Charlotte, North Carolina • Nashville, Tennessee

Conditions: Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
Interventions: Exa-cel; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 9
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 4 more

Search by objective public record fields.

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

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection

Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

Gene Editing For Sickle Cell Disease

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

Gene Therapy for Chronic Granulomatous Disease

Combination Chemotherapy Plus Gene Therapy in Treating Patients With CNS Tumors

Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

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

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Gene Therapy for Fanconi Anemia

Gene-Modified HIV-Protected Stem Cell Transplant in Treating Patients With HIV-Associated Lymphoma

Stem Cell Gene Therapy for Sickle Cell Disease

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

Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant

Stem Cell Gene Therapy for Cystinosis

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease