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ClinicalTrials.gov public records Last synced Sep 17, 2026, 2:40 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

NCT05293626
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NR082 Injection, Injection needle
Drug · Device
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 3, 2024 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 3 Interventional Results available

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03505099
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
onasemnogene abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 42 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654
View directory record Official record
Conditions
Gaucher Disease, Type 2
Interventions
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Genetic · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
0 Months to 24 Months
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 17, 2026, 2:40 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 17, 2026, 2:40 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Boost GVAX Pancreas Vaccine With or Without CY in Patients With Pancreas Cancer

NCT01088789
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
Cyclophosphamide, Cyclophosphamide Pill, GVAX Pancreas Vaccine
Drug · Biological
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2026
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 25, 2025 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 1 Interventional

Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis

NCT05835895
View directory record Official record
Conditions
Osteoarthritis, Knee, Osteo Arthritis Knee, Knee Osteoarthritis
Interventions
GNSC-001, transient immune-modulation, Placebo
Genetic · Drug
Lead sponsor
Genascence Corporation
Industry
Eligibility
40 Years to 75 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
9
States / cities
Mobile, Alabama • La Mesa, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2025 · Synced Sep 17, 2026, 2:40 PM EDT
Completed No phase listed Observational

Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

NCT06667414
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 60 Years
Enrollment
574 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
13
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Bioequivalence ANDA SNP Clinical Study - Raloxifene and Single Nucleotide Polymorphisms

NCT06062810
View directory record Official record
Conditions
Breast Cancer
Interventions
Raloxifene - Usual, Raloxifene - Study
Drug
Lead sponsor
Han Xu, M.D., Ph.D., FAPCR, Sponsor-Investigator, IRB Chair
Industry
Eligibility
24 Years to 64 Years · Female only
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 1 Interventional

Redirected High Affinity Gag-Specific Autologous T Cells for HIV Gene Therapy

NCT00991224
View directory record Official record
Conditions
HIV Infections
Interventions
WT-gag-TCR modified T cells, α/6-gag-TCR modified T cells, STI or Drug Holiday
Biological · Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 9, 2019 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

NCT02652780
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

NCT07818759
View directory record Official record
Conditions
Sanfilippo Syndrome (MPS III)
Interventions
rAAV9-CMV-hNAGLUop
Biological
Lead sponsor
NeuroGT
Industry
Eligibility
6 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2029
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 13, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)

NCT01023048
View directory record Official record
Conditions
Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Interventions
preimplantation diagnosis
Other
Lead sponsor
Natera, Inc.
Industry
Eligibility
Up to 40 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2014 · Synced Sep 17, 2026, 2:40 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study of TAK-500 With or Without Pembrolizumab in Adults With Select Locally Advanced or Metastatic Solid Tumors

NCT05070247
View directory record Official record
Conditions
Pancreatic Cancer, Hepatocellular Cancer, Mesothelioma, Breast Cancer, Gastric Cancer, Esophageal Cancer, Nasopharyngeal Cancer, Kidney Cancer, Squamous Cell Cancer of Head and Neck (SCCHN), Non-small Cell Lung Cancer (NSCLC), Non-squamous
Interventions
TAK-500, Pembrolizumab, Tocilizumab, Dexamethasone
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
12
States / cities
Birmingham, Alabama • Duarte, California • La Jolla, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 1 Interventional

Gene Expression and Tolerability Study of NV1FGF in Patients With Peripheral Artery Occlusive Disease Planned to Undergo Major Amputation

NCT01157143
View directory record Official record
Conditions
Peripheral Arterial Occlusive Disease
Interventions
XRP0038 (NV1FGF)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 4, 2010 · Synced Sep 17, 2026, 2:40 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
ABO-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Sep 17, 2026, 2:40 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

NCT04833907
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV-Olig001-ASPA, Levetiracetam, Prednisone
Drug
Lead sponsor
Myrtelle Inc.
Industry
Eligibility
3 Months to 60 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
BEAM-101
Biological
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
19
States / cities
Birmingham, Alabama • Phoenix, Arizona • Jacksonville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Sep 17, 2026, 2:40 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 17, 2026, 2:40 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

A to Z Study Follow-up: Collection of DNA Data From Buccal Swabs

NCT00866151
View directory record Official record
Conditions
Weight Management, Diet Conditions, Dietary Recommendations, Overweight Management, Obesity
Interventions
Not listed
Lead sponsor
Interleukin Genetics, Inc.
Industry
Eligibility
25 Years to 50 Years · Female only
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 19, 2009 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Clinical Trial of an Alphavirus Replicon Vaccine for Cytomegalovirus (CMV)

NCT00439803
View directory record Official record
Conditions
Cytomegalovirus Infections
Interventions
AVX601, Placebo
Biological
Lead sponsor
AlphaVax, Inc.
Industry
Eligibility
18 Years to 45 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2008
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 9, 2008 · Synced Sep 17, 2026, 2:40 PM EDT
Recruiting Phase 1Phase 2 Interventional

PS-002 for the Treatment of IgA Nephropathy in Adults

NCT07182227
View directory record Official record
Conditions
Immunoglobulin A (IgA) Nephropathy
Interventions
PS-002
Genetic
Lead sponsor
Purespring Therapeutics Limited
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Miami, Florida • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 17, 2026, 2:40 PM EDT
Not listed No phase listed Observational

Whole Genome Sequencing (WGS) on IVF Embryos and Individual Patients

NCT05739890
View directory record Official record
Conditions
Fertility Issues, Single-Gene Defects
Interventions
Embryo genome sequencing, Parents genome sequencing
Other
Lead sponsor
GenEmbryomics Pty. Ltd
Industry
Eligibility
18 Years to 48 Years
Enrollment
100 participants
Timeline
2023 – 2024
U.S. locations
3
States / cities
New York, New York • Portland, Oregon • Kirkland, Washington
Source: ClinicalTrials.gov public record
Updated Sep 24, 2023 · Synced Sep 17, 2026, 2:40 PM EDT
Completed Phase 3 Interventional Results available

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

NCT03392974
View directory record Official record
Conditions
Hemophilia A
Interventions
Valoctocogene Roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Sep 17, 2026, 2:40 PM EDT