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Showing 1–24 of 165 matching trials from the live ClinicalTrials.gov search.
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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 2, 2026, 4:15 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 2, 2026, 4:15 PM EDT
Completed Phase 1 Interventional

Phase I Single Dose, Open-Label Pharmacokinetic Study and Single-Blind, Placebo-Controlled Dose Escalation Study of NFC-1 in Adolescents With Attention-Deficit Hyperactivity Disorder

NCT02286817
View directory record Official record
Conditions
Attention-deficit Hyperactivity Disorder (ADHD)
Interventions
NFC-1
Drug
Lead sponsor
Aevi Genomic Medicine, LLC, a Cerecor company
Industry
Eligibility
12 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Sep 2, 2026, 4:15 PM 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 2, 2026, 4:15 PM 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 2, 2026, 4:15 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 2, 2026, 4:15 PM EDT
Completed Phase 1 Interventional

Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients

NCT01044654
View directory record Official record
Conditions
HIV Infection, HIV Infections
Interventions
SB-728-T
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Los Angeles, California • Newport Beach, California • San Francisco, California + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2015 · Synced Sep 2, 2026, 4:15 PM EDT
Terminated Phase 2 Interventional

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Sep 2, 2026, 4:15 PM EDT
Completed Phase 2 Interventional Results available

An Investigational Drug, PF-02341066, Is Being Studied In Patients With Advanced Non-Small Cell Lung Cancer With A Specific Gene Profile Involving The Anaplastic Lymphoma Kinase (ALK) Gene

NCT00932451
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung
Interventions
PF-02341066
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,069 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
140
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Bakersfield, California + 71 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2017 · Synced Sep 2, 2026, 4:15 PM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Recruiting Phase 2 Interventional

PhII Randomized CAPecitabine + ELAcestrant vs. Capecitabine Alone in ER+ Breast Cancer (CAPELA)

NCT07222215
View directory record Official record
Conditions
Estrogen-receptor-positive Breast Cancer, Metastatic Breast Cancer, Breast Cancer, Hormone Receptor Positive Breast Cancer, Human Epidermal Growth Factor 2 Negative Carcinoma of Breast, HER2- Breast Cancer, ESR1 Gene Mutation, ER Wildtype, Breast Neoplasms
Interventions
Capecitabine, Elacestrant
Drug
Lead sponsor
Kristina A. Fanucci
Other
Eligibility
18 Years and older
Enrollment
297 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT06003387
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
4
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Active, not recruiting Phase 2 Interventional

Panitumumab With or Without Trametinib in Treating Patients With Stage IV Colorectal Cancer

NCT03087071
View directory record Official record
Conditions
EGFR NP_005219.2:p.S492R, KRAS Gene Mutation, MAP2K1 Gene Mutation, Metastatic Colorectal Adenocarcinoma, Refractory Colorectal Adenocarcinoma, Stage IV Colorectal Cancer AJCC v7, Stage IVA Colorectal Cancer AJCC v7, Stage IVB Colorectal Cancer AJCC v7
Interventions
Laboratory Biomarker Analysis, Panitumumab, Trametinib
Other · Biological · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Completed No phase listed Observational

Association of Genes to Resistance to Weight Loss in Obese Patients

NCT00858221
View directory record Official record
Conditions
Obesity, Metabolic Syndrome, Dyslipidemia
Interventions
Not listed
Lead sponsor
Interleukin Genetics, Inc.
Industry
Eligibility
21 Years to 70 Years
Enrollment
824 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Danville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 19, 2009 · Synced Sep 2, 2026, 4:15 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT03368742
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-001
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 17 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Terminated Phase 1 Interventional

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCT06747273
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Interventions
SRP-9004
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
2
States / cities
Columbus, Ohio • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Sep 2, 2026, 4:15 PM EDT
Completed Phase 1 Interventional

Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59

NCT03144999
View directory record Official record
Conditions
Dry Age-related Macular Degeneration
Interventions
AAVCAGsCD59
Biological
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
50 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 2, 2026, 4:15 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 2, 2026, 4:15 PM 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 2, 2026, 4:15 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098
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
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Sacramento, California • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Sep 2, 2026, 4:15 PM EDT
Active, not recruiting Phase 3 Interventional

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

NCT07480564
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
2 Years to 3 Years · Female only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
3
States / cities
Birmingham, Alabama • Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

NCT05788536
View directory record Official record
Conditions
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Interventions
DB-OTO
Genetic
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
9
States / cities
Los Angeles, California • San Diego, California • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 2, 2026, 4:15 PM EDT
Completed Not applicable Interventional Results available

Study in Recipients of Renal Transplant Allograft to Evaluate the Impact of Two Immunosuppressive Regimens

NCT01653847
View directory record Official record
Conditions
End Stage Renal Failure With Renal Transplant
Interventions
Tacrolimus with MMF, Group 2: Tacrolimus with Everolimus.
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 70 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Mar 1, 2021 · Synced Sep 2, 2026, 4:15 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)

NCT06980948
View directory record Official record
Conditions
Chronic Neuropathic Pain
Interventions
ST-503, Sham (No Treatment)
Genetic · Procedure
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
11
States / cities
Scottsdale, Arizona • Little Rock, Arkansas • La Jolla, California + 8 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 2, 2026, 4:15 PM EDT