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ClinicalTrials.gov public records Last synced Sep 17, 2026, 5:14 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Therapy, Gene Clear all
Active, not recruiting Phase 1 Interventional

A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration

NCT06825858
View directory record Official record
Conditions
Age-Related Macular Degeneration
Interventions
KH658
Drug
Lead sponsor
Chengdu Origen Biotechnology Co., Ltd.
Industry
Eligibility
50 Years to 85 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
3
States / cities
Phoenix, Arizona • Lemont, Illinois • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Phase I/II Study of Autologous T Cells to Express T-Cell Receptors (TCRs) in Subjects With Solid Tumors

NCT05194735
View directory record Official record
Conditions
Gynecologic Cancer, Colorectal Cancer, Pancreatic Cancer, Non-small Cell Lung Cancer, Cholangiocarcinoma, Ovarian Cancer, Endometrial Cancer, Ovarian Carcinoma, Ovary Neoplasm, Squamous Cell Lung Cancer, Adenocarcinoma of Lung, Adenosquamous Cell Lung Cancer
Interventions
Neoantigen specific TCR-T cell drug product, Aldesleukin (IL-2)
Biological
Lead sponsor
Alaunos Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 17, 2026, 5:14 AM EDT
Withdrawn Phase 3 Interventional

Olaparib Maintenance Treatment Versus Placebo in Patients With PSR Ovarian Cancer Who Are in CR or PR to Platinum-based Chemotherapy and Whose Tumours Carry sBRCAm or HRR-associated Genes Mutations

NCT02392676
View directory record Official record
Conditions
Platinum Sensitive Relapsed Ovarian Cancer
Interventions
OLAPARIB, PLACEBO
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 96 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
15
States / cities
Mobile, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 1, 2016 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 1 Interventional

AAVrh10-PCCA Gene Therapy for Propionic Acidemia

NCT07643844
View directory record Official record
Conditions
Propionic Acidemia
Interventions
AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
6 Months to 2 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 17, 2026, 5:14 AM 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, 5:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

NCT07748403
View directory record Official record
Conditions
Wilson Disease, Wilson's Disease, Wilsons Disease
Interventions
PM577a
Drug
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Chicago, Illinois • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Inhibitor Masking Device & Sodium Channel, Voltage Gated, Type IX Alpha Subunit (SCN9) Gene Expression

NCT01412918
View directory record Official record
Conditions
Tinnitus
Interventions
The Inhibitor™ Tinnitus Masking Device
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 28, 2016 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 1 Interventional

A Study to Learn About the Study Medicine PF-07985045 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Change in a Gene.

NCT06704724
View directory record Official record
Conditions
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
Interventions
PF-07985045, Gemcitabine, Nab-paclitaxel, Cetuximab, Fluorouracil, Oxaliplatin, Leucovorin, Bevacizumab, Pembrolizumab, Sasanlimab, pemetrexed, Cisplatin, Paclitaxel, Carboplatin, PF-07284892
Drug · Combination Product
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
12
States / cities
Fayetteville, Arkansas • Rogers, Arkansas • Springdale, Arkansas + 6 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Primary Brain Tumors

NCT00002824
View directory record Official record
Conditions
Brain and Central Nervous System Tumors
Interventions
gene therapy, chemotherapy, ganciclovir, conventional surgery
Biological · Drug · Procedure
Lead sponsor
Abramson Cancer Center at Penn Medicine
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2019 · Synced Sep 17, 2026, 5:14 AM EDT
Completed No phase listed Observational

Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews

NCT03054389
View directory record Official record
Conditions
Hemophilia B
Interventions
Qualitative Interview - Participants/ Patients, Qualitative Interview - Investigators and Study Coordinators
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 4 Interventional

D-Cycloserine for Serine Palmitoyltransferase Inhibition

NCT07542548
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
D-cycloserine
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
6 Years to 6 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

NCT04127578
View directory record Official record
Conditions
Parkinson Disease
Interventions
LY3884961, Methylprednisolone, Sirolimus
Biological · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
35 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
11
States / cities
Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1 Interventional Results available

Ipilimumab +/- Vaccine Therapy in Treating Patients With Locally Advanced, Unresectable or Metastatic Pancreatic Cancer

NCT00836407
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
Ipilimumab, Pancreatic Cancer Vaccine
Drug · Biological
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 23, 2020 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

NCT05419492
View directory record Official record
Conditions
Dravet Syndrome
Interventions
ETX101
Drug
Lead sponsor
Encoded Therapeutics
Industry
Eligibility
6 Months to 17 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2033
U.S. locations
11
States / cities
San Francisco, California • Aurora, Colorado • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Lung-MAP: AZD4547 as Second-Line Therapy in Treating FGFR Positive Patients With Recurrent Stage IV Squamous Cell Lung Cancer

NCT02965378
View directory record Official record
Conditions
FGFR1 Gene Amplification, FGFR1 Gene Mutation, FGFR2 Gene Amplification, FGFR2 Gene Mutation, FGFR3 Gene Amplification, FGFR3 Gene Mutation, Recurrent Squamous Cell Lung Carcinoma, Stage IV Squamous Cell Lung Carcinoma AJCC v7
Interventions
Docetaxel, FGFR Inhibitor AZD4547, Laboratory Biomarker Analysis
Drug · Other
Lead sponsor
SWOG Cancer Research Network
Network
Eligibility
25 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1083
States / cities
Birmingham, Alabama • Anchorage, Alaska • Fairbanks, Alaska + 672 more
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 2 Interventional Results available

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

NCT03233204
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma, Malignant Glioma, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Malignant Germ Cell Tumor, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Wilms Tumor
Interventions
Olaparib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
111
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 2 Interventional

E7 TCR-T Cell Immunotherapy for Human Papillomavirus (HPV) Associated Cancers

NCT05686226
View directory record Official record
Conditions
Cervical Cancer, Throat Cancer, Oropharynx Cancer, Anal Cancer, Vulva Cancer, Vaginal Cancer, Penile Cancer, Metastatic Cancer, HPV-Related Malignancy, HPV-Related Carcinoma, HPV-Related Cervical Carcinoma, HPV-Related Squamous Cell Carcinoma, HPV-Related Adenocarcinoma, HPV Positive Oropharyngeal Squamous Cell Carcinoma, HPV-Associated Vaginal Adenocarcinoma, HPV-Related Adenosquamous Carcinoma, HPV-Related Endocervical Adenocarcinoma, HPV-Related Anal Squamous Cell Carcinoma, HPV-Related Penile Squamous Cell Carcinoma, HPV-Related Vulvar Squamous Cell Carcinoma, HPV Positive Rectal Squamous Cell Carcinoma
Interventions
E7 TCR-T cells, Aldesleukin
Biological · Drug
Lead sponsor
Christian Hinrichs
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
3
States / cities
Bethesda, Maryland • New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Colon Cancer That Has Spread to the Liver

NCT00012155
View directory record Official record
Conditions
Colorectal Cancer, Metastatic Cancer
Interventions
NV1020
Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 25, 2013 · Synced Sep 17, 2026, 5:14 AM EDT
Not listed Phase 1Phase 2 Interventional

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

NCT03315078
View directory record Official record
Conditions
X-Linked Combined Immunodeficiency Diseases
Interventions
CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan
Biological · Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 40 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Sep 17, 2026, 5:14 AM EDT
Not listed Phase 1 Interventional

Treatment of a Single Patient With CRD-TMH-001

NCT05514249
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
CRD-TMH-001
Drug
Lead sponsor
Cure Rare Disease, Inc
Other
Eligibility
18 Years to 28 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 2 Interventional

RP2/RP3 in Combination With Atezolizumab and Bevacizumab for the Treatment of Patients With CRC

NCT05733611
View directory record Official record
Conditions
Refractory Metastatic Colorectal Cancer, pMMR, MSS
Interventions
RP2, RP3, atezolizumab, bevacizumab
Biological
Lead sponsor
Replimune, Inc.
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
2
States / cities
Los Angeles, California • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Feb 1, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1 Interventional

Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

NCT05441514
View directory record Official record
Conditions
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Cobimetinib, Enasidenib Mesylate
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Gene Therapy for IGHMBP2-Related Diseases

NCT05152823
View directory record Official record
Conditions
SMARD1, CMT2S
Interventions
Gene Therapy
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
2 Months to 14 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated No phase listed Observational

Characterizing Fatigue Experienced by Cancer Patients Receiving Primary Treatment and Cancer Survivors

NCT01231932
View directory record Official record
Conditions
Cancer, Fatigue, Prostate Cancer, Breast Cancer, Cancer Survivor
Interventions
Not listed
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
18 Years to 120 Years
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 18, 2025 · Synced Sep 17, 2026, 5:14 AM EDT