Clinical trials: Gene, open to volunteers
Heart Failure With Reduced Ejection Fraction
Study Assessing Left Ventricular Administration of a Genetic Medicine Directing Organ Regeneration in Heart Failure
This clinical trial investigates the safety and preliminary effectiveness of YAP101, a gene therapy designed to improve heart function in adults with ischemic heart failure and reduced ejection fraction (HFrEF).
- Trial name
- SALVADOR-HF (NCT06831825)
- Testing
- YAP101 (AAV9-Sav-shRNA)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Medley Therapeutics
Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm
Evaluating the Effects of Tasimelteon Vs. Placebo in Delayed Sleep-Wake Phase Disorder (DSWPD) and the CRY1Δ11 Variant
This is a double-blind, randomized, two-period crossover study to evaluate the efficacy and safety of a single oral dose of tasimelteon and matching placebo in male and female subjects with DSWPD and the CRY1Δ11 variant.
- Conditions
- Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm, Chronobiology Disorders, Gene Mutations and Other Alterations Nec
- Trial name
- NCT06701396
- Testing
- Tasimelteon
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Vanda Pharmaceuticals
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Recurrent High Grade Gliomas, Glioblastoma (GBM)
A Study of SRN-101 Gene Therapy in Adults With Recurrent High-Grade Glioma (ALERT-1)
ALERT-1 is a Phase 1/2 study evaluating the safety and preliminary anti-tumor activity of SRN-101, an investigational gene therapy, in adults with recurrent high-grade glioma. High-grade glioma is a type of brain cancer.
- Conditions
- Recurrent High Grade Gliomas, Glioblastoma (GBM), Astrocytoma, Oligodendroglioma
- Trial name
- ALERT-1 (NCT07902037)
- Testing
- SRN-101
- Volunteers
- 40 people
- Sponsor
- Siren Biotechnology
Locally Advanced or Metastatic Solid Tumors, Locally Advanced or Metastatic Non-small Cell Lung Cancer
A Study of XZP-5955 Tablets in Patients With NTRK or ROS1 Fusion Positive Locally Advanced or Metastatic Solid Tumors
A phase I/II study to examine the safety, tolerability, pharmacokinetics and efficacy of XZP-5955 tablets in patients with advanced solid tumors harboring NTRK or ROS1 gene fusion
- Trial name
- NCT04996121
- Testing
- XZP-5955 tablets
- Where
- China
- Volunteers
- 360 people
- Sponsor
- Xuanzhu Biopharmaceutical Co., Ltd.
Prostate Cancer
A Study of Metastases Free Survival With Saruparib vs Placebo Added to a Standard RT/ADT in Men With High-risk Prostate Cancer With a BRCA Mutation
The purpose of the study is to demonstrate superiority of Saruparib (AZD5305) relative to placebo added to a standard radiation therapy (RT) + androgen deprivation therapy (ADT) regimen by assessment of metastases-free survival…
- Trial name
- EvoPAR-PR02 (NCT06952803)
- Testing
- Saruparib, Abiraterone + Prednisolone/Prednisone, Androgen Deprivation Therapy (ADT)
- Where
- 346 sites in United States, Argentina and 26 more
- Volunteers
- 700 people
- Sponsor
- AstraZeneca
Familial Chylomicronemia Syndrome (FCS)
CS-121 APOC3 Base Editing in FCS
This is an open-label, single-arm, dose-escalation Phase I clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid…
- Trial name
- NCT07176923
- Testing
- CS-121
- Where
- China
- Volunteers
- 15 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Atherosclerosis, Cardiovascular Disease
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
The purpose of this study is to measure the change in bad cholesterol (LDL-C) with VERVE-102 compared to placebo in adults who have plaque buildup in their arteries or are at high risk of developing it.
- Conditions
- Atherosclerosis, Cardiovascular Disease, Hypercholesterolemia
- Trial name
- HEART-3 (NCT07839936)
- Testing
- VERVE-102
- Where
- United States
- Volunteers
- 175 people
- Sponsor
- Verve Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
PDL1 Gene Mutation, Non Small Cell Lung Cancer
177Lu-anti-PD-L1 sdAb in Metastatic Solid Tumors
This is a Phase 0/1, First-in-Human (FIH), study to evaluate safety, tolerability of 177Lu-RAD204 in participants with selected solid tumors, and to determine the MTD and/or recommended dose for future exploration.
- Conditions
- PDL1 Gene Mutation, Non Small Cell Lung Cancer, Small Cell Lung Cancer ( SCLC ), TNBC, Triple Negative Breast Cancer
- Trial name
- NCT06305962
- Testing
- 177Lu-RAD204
- Where
- 6 sites in Australia
- Volunteers
- 30 people
- Sponsor
- Radiopharm Theranostics, Ltd
Knee Arthritis Osteoarthritis, Knee Arthritis, Osteoarthritis
Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis
The goal of this clinical trial is to learn if GNSC-001 works to treat knee osteoarthritis in adults. It will also learn about the safety of GNSC-001. The main questions it aims to answer are: 1.
- Conditions
- Knee Arthritis Osteoarthritis, Knee Arthritis, Osteoarthritis, Knee Arthritis, Knee Arthritis Disease
- Trial name
- RAPHAEL (NCT07831577)
- Testing
- Gene Therapy GNSC-001
- Volunteers
- 240 people
- Sponsor
- Genascence Corporation
Mesial Temporal Lobe Epilepsy
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).
- Trial name
- GenTLE (NCT06063850)
- Testing
- AAV9-hSyn1-miGRIK2
- Where
- 18 sites in United States
- Volunteers
- 12 people
- Sponsor
- UniQure Biopharma B.V.
Neovascular Age-related Macular Degeneration
Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
- Trial name
- NCT06141460
- Testing
- RRG001
- Where
- China
- Volunteers
- 48 people
- Sponsor
- Shanghai Refreshgene Technology Co., Ltd.
Advanced Cholangiocarcinoma, FGFR2 Fusions
Study of Futibatinib in Patients With Advanced Cholangiocarcinoma With FGFR2 Fusion or Rearrangement
This is an open-label, multinational, randomized Phase 2 study confirming the clinical benefit of 20 mg futibatinib and evaluating the safety and efficacy of 16 mg futibatinib in previously treated CCA harboring FGFR2 gene…
- Conditions
- Advanced Cholangiocarcinoma, FGFR2 Fusions, Gene Rearrangement
- Trial name
- FOENIX-CCA4 (NCT05727176)
- Testing
- TAS-120
- Where
- 65 sites in United States, Argentina and 11 more
- Volunteers
- 120 people
- Sponsor
- Taiho Oncology, Inc.
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation
Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia
- Trial name
- AUGMENT-101 (NCT04065399)
- Testing
- revumenib, cobicistat
- Where
- 57 sites in United States, Australia and 9 more
- Volunteers
- 447 people
- Sponsor
- Syndax Pharmaceuticals
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
LCA5
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration…
- Trial name
- NCT05616793
- Testing
- AAV8.hLCA5
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Opus Genetics, Inc
Chronic Myelogenous Leukemia
A Clinical Trial to Evaluate the Safety and Tolerability of TQB3911 Tablets in Patients With BCR::ABL Fusion Gene Positive Leukemia
Phase I clinical trial to explore the safety, tolerability, and initial efficacy of TQB3911 tablets in BCR::ABL fusion gene positive leukemia.
- Trial name
- NCT06672263
- Testing
- TQB3911 tablets
- Where
- 5 sites in China
- Volunteers
- 120 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.