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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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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 9, 2026, 9:50 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Covid-19 Long Haul Preventative and Health Promotion Care Clinical Trial Acceleration Program.

NCT06441955
View directory record Official record
Conditions
COVID-19, Long Haul
Interventions
Ritonavir-Boosted Nirmatrelvir (Paxlovid), Physiological Evaluation, Moderna COVID-19 Vaccine, Biopsychological, Behavioral (e.g., Psychotherapy, Lifestyle Counseling), Genetic (including gene transfer, stem cell and recombinant DNA), Multidisciplinary approach
Drug · Diagnostic Test · Biological + 3 more
Lead sponsor
Well- Konnect Healthcare Services and Research Firm
Network
Eligibility
18 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 1 Interventional Results available

Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee

NCT00599248
View directory record Official record
Conditions
Osteoarthritis, Knee
Interventions
TissueGene-C, Placebo
Biological
Lead sponsor
Kolon TissueGene, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
Baltimore, Maryland • Arlington, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2017 · Synced Sep 9, 2026, 9:50 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155
View directory record Official record
Conditions
Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
375 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2031
U.S. locations
14
States / cities
Duarte, California • Gainesville, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Non-small Cell Lung Cancer That Cannot Be Surgically Removed

NCT00003649
View directory record Official record
Conditions
Lung Cancer
Interventions
Ad5CMV-p53 gene
Biological
Lead sponsor
Eastern Cooperative Oncology Group
Network
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2007
U.S. locations
3
States / cities
Nashville, Tennessee • Green Bay, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 21, 2023 · Synced Sep 9, 2026, 9:50 AM 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 9, 2026, 9:50 AM EDT
Recruiting Phase 1Phase 2 Interventional

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

NCT05821959
View directory record Official record
Conditions
Sensorineural Hearing Loss, Bilateral
Interventions
AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
Combination Product
Lead sponsor
Akouos, Inc.
Industry
Eligibility
Not listed
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
6
States / cities
San Francisco, California • Iowa City, Iowa • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

NCT04850118
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-hRPGRco, Control
Biological · Drug
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
19
States / cities
Scottsdale, Arizona • Los Angeles, California • Jacksonville, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Phase 3 Interventional

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620
View directory record Official record
Conditions
Rett Syndrome
Interventions
NGN-401
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years and older · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
13
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Completed No phase listed Observational

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

NCT02946879
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
Interventions
AAV OPTIRPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Sep 9, 2026, 9:50 AM EDT
Recruiting Phase 1 Interventional

B7-H3.CD28Z.CART in Solid Tumors

NCT07358260
View directory record Official record
Conditions
Pediatric Solid Tumor, Neuroblastoma
Interventions
B7-H3.CD28Z.CART, Fludarabine, Cyclophosphamide
Biological · Drug
Lead sponsor
Robbie Majzner
Other
Eligibility
9 Months to 30 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Energy Balance Interventions in Increasing Physical Activity in Breast Cancer Gene Positive Patients, Lynch Syndrome-Positive Patients, CLL Survivors or High-Risk Family Members

NCT02194387
View directory record Official record
Conditions
Body Mass Index 25 or Greater, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Breast Carcinoma, Cancer Survivor, Chronic Lymphocytic Leukemia, Fatigue, Health Status Unknown, Lynch Syndrome, Ovarian Carcinoma, Overweight
Interventions
Dietary Intervention, Internet-Based Intervention, Laboratory Biomarker Analysis, Questionnaire Administration, Telephone-Based Intervention
Dietary Supplement · Other · Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
337 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

NCT06876363
View directory record Official record
Conditions
X-Linked Chronic Granulomatous Disease
Interventions
EN-374
Genetic
Lead sponsor
Ensoma
Industry
Eligibility
3 Months and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
Los Angeles, California • San Francisco, California • St. Petersburg, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Terminated Phase 2 Interventional Results available

Trial of Radiation and Gene Therapy Before Nivolumab for Metastatic Non-Small Cell Lung Carcinoma and Uveal Melanoma

NCT02831933
View directory record Official record
Conditions
Lung Squamous Cell Carcinoma Stage IV, Nonsquamous Nonsmall Cell Neoplasm of Lung, Metastatic Uveal Melanoma
Interventions
ADV/HSV-tk, Valacyclovir, SBRT, nivolumab
Biological · Drug · Radiation
Lead sponsor
Eric Bernicker, MD
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 20, 2023 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

NCT00976352
View directory record Official record
Conditions
Pompe Disease
Interventions
rAAV1-CMV-GAA (study agent) Administration, RMST
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 13, 2018 · Synced Sep 9, 2026, 9:50 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
View directory record Official record
Conditions
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2030
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 22, 2025 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116
View directory record Official record
Conditions
Hemophilia B, Hemophilia A
Interventions
Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
13
States / cities
Palo Alto, California • San Francisco, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 5, 2025 · Synced Sep 9, 2026, 9:50 AM EDT
Enrolling by invitation Phase 2 Interventional

Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic Retinopathy

NCT05296447
View directory record Official record
Conditions
Diabetic Retinopathy, DR
Interventions
Standard of Care, Aflibercept
Drug
Lead sponsor
AbbVie
Industry
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
20
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 9, 2026, 9:50 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Long-term Follow-up Study of Patients Who Received EG110A, a Non-replicative Herpes Simplex Virus 1-derived Gene Therapy

NCT07227285
View directory record Official record
Conditions
Urinary Bladder, Overactive, Spinal Cord Injury
Interventions
Urodynamics, EG110A, Patient-reported Outcome
Other · Drug
Lead sponsor
Cyllene Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
4
States / cities
Downey, California • Ann Arbor, Michigan • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 9, 2026, 9:50 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Gene Therapy Using Anti-Her-2 Cells to Treat Metastatic Cancer

NCT00924287
View directory record Official record
Conditions
Metastatic Cancer
Interventions
in vitro tumor reactive, chimeric T cell receptor (CAR ) gene-transduced PBL plus IV aldesleukin, Cyclophosphamide, Fludarabine, Mesna
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2015 · Synced Sep 9, 2026, 9:50 AM EDT
Completed No phase listed Observational

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

NCT03597399
View directory record Official record
Conditions
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
12 Months and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
10
States / cities
Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Sep 9, 2026, 9:50 AM EDT
Completed Phase 1 Interventional

Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma

NCT00186862
View directory record Official record
Conditions
Neuroblastoma
Interventions
Interleukin-2
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 2, 2008 · Synced Sep 9, 2026, 9:50 AM EDT