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Showing 1–24 of 1,024 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 3 Interventional

Somatropin Treatment in Patients With SHOX Deficiency and Turner Syndrome

NCT00190658
View directory record Official record
Conditions
Failure to Thrive
Interventions
Somatropin (rDNA origin) for injection
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
3 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2010
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 19, 2010 · Synced Sep 3, 2026, 2:27 AM EDT
Recruiting Phase 1 Interventional

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

NCT07148089
View directory record Official record
Conditions
Catecholaminergic Polymorphic Ventricular Tachycardia
Interventions
SGT-501
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
4
States / cities
Boston, Massachusetts • Rochester, Minnesota • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 3, 2026, 2:27 AM 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 3, 2026, 2:27 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011, SPK-8016
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
9
States / cities
Boston, Massachusetts • Madison, Mississippi • Kansas City, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Sep 3, 2026, 2:27 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926, Placebo
Genetic · Other
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2039
U.S. locations
17
States / cities
Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Active, not recruiting No phase listed Observational

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study

NCT06162585
View directory record Official record
Conditions
Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
Interventions
Gene Therapy product-MCO-010
Biological
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
5
States / cities
Beverly Hills, California • Pensacola, Florida • Fargo, North Dakota + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2025 · Synced Sep 3, 2026, 2:27 AM EDT
Terminated No phase listed Observational

Setting up a Blood Bank for Gene Therapy in HIV-Infected Infants

NCT00000917
View directory record Official record
Conditions
HIV Infections, Pregnancy
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Day to 6 Months
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
8
States / cities
La Jolla, California • Long Beach, California • Los Angeles, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2008 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

A Phase 1, Open-Label, Dose-Escalation & Expanded Cohort, Continuous IV Infusion, Multi-center Study of the Safety, Tolerability,PK & PD of EPZ-5676 in Treatment Relapsed/Refractory Patients With Leukemias Involving

NCT01684150
View directory record Official record
Conditions
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Disorders
Interventions
EPZ-5676
Drug
Lead sponsor
Epizyme, Inc.
Industry
Eligibility
18 Years to 90 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
6
States / cities
Scottsdale, Arizona • Chicago, Illinois • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2024 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 4 Interventional

Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy

NCT07632768
View directory record Official record
Conditions
Cystic Fibrosis (CF), Pancreatic Insufficiency, Pancreas Disease, Exocrine Pancreatic Insufficiency, Exocrine Pancreatic Insufficiency in Subjects With Cystic Fibrosis, CFTR Gene Mutation
Interventions
Discontinuation of Pancreatic Enzyme Replacement Therapy
Drug
Lead sponsor
Indiana University
Other
Eligibility
0 Years to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

NCT00018018
View directory record Official record
Conditions
Severe Combined Immunodeficiency Syndrome
Interventions
CD34+ cells transduced with ADA retrovir
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Sep 3, 2026, 2:27 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

NCT04120493
View directory record Official record
Conditions
Huntington's Disease
Interventions
intra-striatal rAAV5-miHTT, Imitation (sham) surgery
Genetic · Other
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
25 Years to 65 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
12
States / cities
Birmingham, Alabama • Tucson, Arizona • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Sep 3, 2026, 2:27 AM EDT
Recruiting Phase 1Phase 2 Interventional

Super-selective Intra-arterial Repeated Infusion of Cetuximab for the Treatment of Newly Diagnosed Glioblastoma

NCT02861898
View directory record Official record
Conditions
Glioblastoma, Brain Cancer, Brain Neoplasm, Brain Tumor, Brain Neoplasm, Malignant, EGFR Gene Overexpression, GBM
Interventions
Intra-arterial Cetuximab, Intra-arterial Mannitol
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Recruiting Phase 2 Interventional

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
3
States / cities
La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Gene Therapy Plus Radiation Therapy in Treating Patients With Non-Small Cell Lung Cancer

NCT00004225
View directory record Official record
Conditions
Lung Cancer
Interventions
Ad5CMV-p53 gene, radiation therapy
Biological · Radiation
Lead sponsor
Eastern Cooperative Oncology Group
Network
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 6, 2010 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 3 Interventional Results available

A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

NCT02752035
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
Interventions
gilteritinib, azacitidine
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Los Angeles, California • Orange, California • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

NCT04408625
View directory record Official record
Conditions
Frontotemporal Dementia
Interventions
LY3884963, Methylprednisolone, Optional Sirolimus, Optional Prednisone
Biological · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
30 Years to 85 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
4
States / cities
Maitland, Florida • Orlando, Florida • Burlington, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

CAR T Cell Receptor Immunotherapy Targeting VEGFR2 for Patients With Metastatic Cancer

NCT01218867
View directory record Official record
Conditions
Metastatic Cancer, Metastatic Melanoma, Renal Cancer
Interventions
Anti-VEGFR2 CAR CD8 plus PBL, Cyclophosphamide, Aldesleukin, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Gene Therapy to Improve Wound Healing in Patients With Diabetes

NCT00065663
View directory record Official record
Conditions
Wounds and Injuries, Diabetes, Diabetic Foot Ulcers, Foot Wounds
Interventions
GAM501
Genetic
Lead sponsor
Tissue Repair Company
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
2
States / cities
Phoenix, Arizona • San Diego, California
Source: ClinicalTrials.gov public record
Updated Nov 19, 2007 · Synced Sep 3, 2026, 2:27 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 3, 2026, 2:27 AM EDT
Recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807
View directory record Official record
Conditions
Phenylketonurias
Interventions
NGGT002
Genetic
Lead sponsor
NGGT INC.
Industry
Eligibility
18 Years to 55 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
5
States / cities
Orange, California • Minneapolis, Minnesota • Morristown, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

A Study of LOXO-260 in Cancer Patients With a Change in a Particular Gene (RET) That Has Not Responded to Treatment

NCT05241834
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung, Thyroid Neoplasms
Interventions
LOXO-260
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
12 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 3, 2026, 2:27 AM 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 3, 2026, 2:27 AM EDT
Recruiting Early Phase 1 Interventional

A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion

NCT04655404
View directory record Official record
Conditions
High Grade Glioma, Diffuse Intrinsic Pontine Glioma
Interventions
Larotrectinib, Larotrectinib surgical
Drug · Procedure
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Up to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2036
U.S. locations
10
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 3, 2026, 2:27 AM EDT
Terminated Phase 1 Interventional

Wild Type p53 Adenovirus for Oral Premalignancies

NCT00410865
View directory record Official record
Conditions
Mouth Cancer
Interventions
INGN 201
Genetic
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 6, 2011 · Synced Sep 3, 2026, 2:27 AM EDT