Clinical trials: Gene
OTC Deficiency
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
- Trial name
- NCT05345171
- Testing
- DTX301, Oral Corticosteroids, Sodium Acetate
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 37 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Genomic
Harmonized Micronutrient Project
The purpose of this trial is to better define how individuals with different genetic make-ups will require different interventions to improve their methylation potential and nutrient status.
- Trial name
- NCT01823744
- Testing
- micronutrient supplementation
- Where
- Brazil
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Esophageal Neoplasms
KCNA3 and OTOP2 Gene Methylation Combined Detection Kit (Fluorescent PCR Method)
The goal of this observational study is to test the clinical efficacy of "KCNA3 and OTOP2 gene methylation combined detection kit (fluorescence PCR method)"in esophageal cancer and high-grade esophageal neoplasia auxiliary…
- Trial name
- NCT05680077
- Testing
- KCNA3 and OTOP2 Gene Methylation Combined Detection Kit (Fluorescent PCR Method)
- Where
- China
- Volunteers
- 1,116 people
- Sponsor
- Wuhan Ammunition Life-tech Co., Ltd
Non-Small Cell Lung Cancer, ALK Gene Rearrangement Positive
Expanded Access to Ensartinib for Participants With ALK+ NSCLC
This is an open-label, multicenter, intermediate-sized expanded access treatment protocol to the existing IND 111,695 for ensartinib (X-396).
- Trial name
- NCT04146571
- Testing
- Ensartinib
- Where
- 4 sites in United States
- Sponsor
- Xcovery Holdings, Inc.
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
β-thalassemia Major
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT05776173
- Testing
- BD211
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Advanced or Recurrent Colorectal Cancer
Panitumumab for Intravenous Infusion 100 mg and 400 mg Special Drug Use Surveillance "Survey on Unresectable, Advanced or Recurrent Colorectal Cancer With Wild-type KRAS Gene" (All-patient Surveillance)
The efficacy and safety of panitumumab (Vectibix) in the routine clinical setting will be studied. Specifically, information will be collected on the following as events of interest: skin disorders, interstitial lung disease…
- Trial name
- NCT02089737
- Testing
- Panitumumab
- Volunteers
- 3,091 people
- Sponsor
- Takeda
Peripheral Arterial Disease(PAD), Arterial Occlusive Disease
Follow-up Study Using Gene Therapy for Critical Limb Ischemia (NL003-CLI-III-L)
The goal of this observational study is to learn about the long-term effects of Donaperminogene Seltoplasmid Injection (NL003) in participants who have been received drug NL003 or placebo at least one dose from the parent phase…
- Conditions
- Peripheral Arterial Disease(PAD), Arterial Occlusive Disease, Arteriosclerosis Obliterans, Thromboangiitis Obliterans
- Trial name
- NCT07023965
- Testing
- NL003
- Where
- 5 sites in China
- Volunteers
- 542 people
- Sponsor
- Beijing Northland Biotech. Co., Ltd.
Esophageal Squamous Cell Carcinoma
Z650 in Advanced Esophageal Squamous Cell Carcinoma With EGFR Over Expression or Gene Amplification
The purpose of this study is to evaluate the safety and efficacy of Z650 in Advanced Esophageal Squamous Cell Carcinoma patients With EGFR Over expression or Gene Amplification.
- Trial name
- NCT03888092
- Testing
- Z650
- Where
- China
- Volunteers
- 81 people
- Sponsor
- Sunshine Lake Pharma Co., Ltd.
Huntington's Disease
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study.
- Trial name
- NCT04120493
- Testing
- intra-striatal rAAV5-miHTT
- Where
- 12 sites in United States
- Volunteers
- 43 people
- Sponsor
- UniQure Biopharma B.V.
HER2 Gene Mutation, Advanced Solid Tumors
HEAT Trial (HER2 Antibody Therapy With Lutetium-177)
This is a first-in-human, Phase 0/1, open-label study of177Lu-RAD202 to determine the recommended dose(s) for future exploration of 177Lu-RAD202 in participants with HER2 expressing advanced solid tumours.
- Trial name
- RAD202 (NCT06824155)
- Testing
- 177Lu-RAD202
- Where
- 5 sites in Australia
- Volunteers
- 30 people
- Sponsor
- Radiopharm Theranostics, Ltd
EGFR Gene Mutation
Clinical Trial of YH25448 in Patients with EGFR Mutation Positive Advanced NSCLC
YH25448 is an oral, highly potent, mutant-selective and irreversible EGFR Tyrosine-kinase inhibitors (TKIs) targets both the T790M mutation and activating EGFR mutations while sparing wild type-EGFR.
- Trial name
- NCT03046992
- Testing
- YH25448
- Where
- 16 sites in South Korea
- Volunteers
- 224 people
- Sponsor
- Yuhan Corporation
Frontotemporal Dementia
A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.
- Trial name
- NCT04374136
- Testing
- Part 1 Blinded - AL001, Part 2 (OLE Treatment) - AL001
- Where
- 44 sites in United States, Argentina and 14 more
- Volunteers
- 119 people
- Sponsor
- Alector Inc.
Merkel Cell Carcinoma, Basal Cell Carcinoma
Study to Investigate the Efficacy and Safety of RP1 in Adult Patients With Organ Transplants and Advanced Skin Malignancies
The purpose of this study is to assess the safety and efficacy of RP1 (administered into the tumor) in 90 patients who have received an organ transplant in the past and currently have skin cancer.
- Conditions
- Merkel Cell Carcinoma, Basal Cell Carcinoma, Locally Advanced Cutaneous Squamous Cell Carcinoma, Cancer
- Trial name
- ARTACUS (NCT04349436)
- Testing
- RP1, intra-tumoral injection, oncolytic virus
- Where
- 24 sites in United States
- Volunteers
- 69 people
- Sponsor
- Replimune, Inc.
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.
Biliary Tract Cancer
A Post-marketing Observational Study of Tasfygo in Participants With Unresectable Biliary Tract Cancer With Fibroblast Growth Factor Receptor 2 (FGFR2) Fusion Gene Positivity Who Progressed After Chemotherapy
The primary purpose of this study is to investigate the safety of Tasfygo.
- Trial name
- NCT06793709
- Testing
- No Intervention
- Where
- Japan
- Volunteers
- 60 people
- Sponsor
- Eisai Co., Ltd.
Advanced Solid Tumor, Esophageal Cancer
A Study of PRT3789 in Combination With Pembrolizumab in Patients With Advanced or Metastatic Solid Tumors With a SMARCA4 Mutation
This is a Phase 2 an open-label, multi-center study to determine the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and anti-tumor activity of PRT3789 in combination with pembrolizumab in patients with…
- Conditions
- Advanced Solid Tumor, Esophageal Cancer, Metastatic Solid Tumor, Non-small Cell Lung Cancers
- Trial name
- NCT06682806
- Testing
- PRT3789, pembrolizumab
- Where
- 9 sites in United States, Spain
- Volunteers
- 6 people
- Sponsor
- Prelude Therapeutics Incorporated
Acute Myeloid Leukemias
A Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Acute Myeloid Leukemias
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of revumenib in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML)…
- Trial name
- NCT06226571
- Testing
- Revumenib, Chemotherapy Regimen, HiDAC
- Where
- 48 sites in United States, Australia and 4 more
- Volunteers
- 96 people
- Sponsor
- Syndax Pharmaceuticals
Non Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma
Safety, PK/PD, and Clinical Activity of KT-413 in Adult Patients with Relapsed or Refractory B-cell NHL
This Phase 1a/1b study will evaluate the safety, tolerability and the pharmacokinetics/ pharmacodynamics (PK/ PD) of KT-413 in patients with R/R NHL.
- Conditions
- Non Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma, DLBCL, MYD88 Gene Mutation
- Trial name
- NCT05233033
- Testing
- KT-413
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Kymera Therapeutics, Inc.
Lung Cancer, ROS1 Gene Rearrangement
X-396 Capsule in Advanced NSCLC Patients With ROS1 Gene Rearrangement
To assess treatment effectiveness and safety of oral X-396 capsule (Ensartinib) administered to Chinese patients with Advanced Non-Small Cell Lung Cancer (NSCLC) that is confirmed to be positive for a c-ROS Oncogene (ROS1)…
- Trial name
- NCT03608007
- Testing
- X-396 Capsule
- Where
- China
- Volunteers
- 69 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.