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ClinicalTrials.gov public records Last synced Sep 29, 2026, 7:57 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Lentiviral Gene Transfer Clear all
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Study Participant Treated With Lentiviral-Based Genetically Modified Autologous Cell Product ,AGT103-T

NCT05529342
View directory record Official record
Conditions
HIV
Interventions
Gene modified therapy
Biological
Lead sponsor
American Gene Technologies International Inc.
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2038
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced Sep 29, 2026, 7:57 PM EDT
Withdrawn Phase 2Phase 3 Interventional

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

NCT04140539
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
OTL-101
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced Sep 29, 2026, 7:57 PM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 19, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Active, not recruiting Phase 2 Interventional

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

NCT05353647
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

NCT02999984
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
Genetic · Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 3 Interventional Results available

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

NCT03207009
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Sep 29, 2026, 7:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

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

NCT01306019
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 27, 2026 · Synced Sep 29, 2026, 7:57 PM EDT
Completed No phase listed Observational

CART-meso Long-term Follow-up

NCT02388828
View directory record Official record
Conditions
Subjects Who Have Received Lentiviral-based CART-meso Therapy
Interventions
lentiviral-based CART meso therapy
Biological
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 28, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Temporarily Not Available No phase listed Expanded access

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT07640815
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 55 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1 Interventional

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

NCT02022696
View directory record Official record
Conditions
Adenosine Deaminase Deficiency, ADA-SCID
Interventions
Lentiviral Gene Transfer
Genetic
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Year to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 26, 2017 · Synced Sep 29, 2026, 7:57 PM EDT
Recruiting No phase listed Observational

Lentiviral Gene Therapy (Zamtocabtagene Autoleucel) LTFU

NCT06116110
View directory record Official record
Conditions
Non Hodgkin Lymphoma, Refractory Diffuse Large B Cell Lymphoma (DLBCL), Relapsed Diffuse Large B Cell Lymphoma, High Grade B-cell Lymphoma, Central Nervous System Lymphoma, Primary Mediastinal B-cell Lymphoma (PMBCL)
Interventions
Long-term Follow-Up
Other
Lead sponsor
Miltenyi Biomedicine GmbH
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
11
States / cities
Gilbert, Arizona • Stanford, California • New Haven, Connecticut + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 29, 2026, 7:57 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 4, 2024 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional

Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

NCT02140554
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
lovo-cel
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Terminated Phase 1 Interventional

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 3 Interventional Results available

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

NCT02906202
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Sep 29, 2026, 7:57 PM EDT
Withdrawn Phase 1 Interventional

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

NCT02378922
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 26, 2018 · Synced Sep 29, 2026, 7:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of PMT Therapy of hPAP

NCT05761899
View directory record Official record
Conditions
Hereditary Pulmonary Alveolar Proteinosis
Interventions
Gene-Corrected Macrophages administered by bronchoscopic instillation
Combination Product
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2038
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Active, not recruiting Phase 2 Interventional

Gene Therapy for Fanconi Anemia, Complementation Group A

NCT04248439
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Stanford, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

NCT01745120
View directory record Official record
Conditions
β-thalassemia Major
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
4
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated May 7, 2019 · Synced Sep 29, 2026, 7:57 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT01896102
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Sep 29, 2026, 7:57 PM EDT
Terminated Phase 1 Interventional

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

NCT04525352
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 12, 2022 · Synced Sep 29, 2026, 7:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

Autologous Gene Therapy for Artemis-Deficient SCID

NCT03538899
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
Drug · Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Months and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2038
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Sep 29, 2026, 7:57 PM EDT