Clinical trials: Gene, open to volunteers
Non Small Cell Lung Caner
AI-Based DeepGEM Tool for Predicting Gene Mutations in NSCLC Patients: A Randomized Controlled Study
This prospective, multicenter, randomized controlled trial aims to evaluate the clinical utility of DeepGEM, an artificial intelligence (AI)-based mutation prediction tool based on histopathological whole-slide images, in…
- Trial name
- NCT07110259
- Testing
- DeepGEM-guided Molecular Testing and Treatment, Standard Diagnostic Pathway
- Volunteers
- 950 people
- Sponsor
- Jianxing He
Autism Spectrum Disorder
MRI Study of Subjects with ASD, Their Relatives and TD
This project is structured around a central study called "Study of genetic factors involved in autism and related conditions ("Genes and Autism" study, sponsor: INSERM).
- Trial name
- NCT05470088
- Testing
- Neuroimaging (MRI)
- Where
- 2 sites in France
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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.
Single Cell Sequencing Technology, Gene Expression Profiling
Host Response to Infection by Direct Analysis of Leukocyte Single Cell-type Gene Expression/transcript Abundance, Direct LS-TA. a Prospective Study Will Evaluate the Performance of Direct LS-TA in Triage Febrile Patients Into Major Categories of Infections: Viral, Bacterial or Active Tuberculosis.
Febrile illness is a common condition and it is crucial to have an early triage of patients according to various aetiologies to enable appropriate treatment.
- Conditions
- Single Cell Sequencing Technology, Gene Expression Profiling, Gene Expression, Infection
- Trial name
- NCT06846645
- Testing
- No intervention
- Where
- Hong Kong
- Volunteers
- 200 people
- Sponsor
- Chinese University of Hong Kong
Lumbar Spinal Stenosis, Cardiac Amyloidosis
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- NCT06034405
- Testing
- Tc99-PYP or Tc99-HDMP Scan
- Where
- 6 sites in United States
- Volunteers
- 1,663 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
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
Thyroid Cancer, Papillary
Ultrasound-Guided Microwave Ablation Vs. Surgery for Low-Risk PapilaryThyroid Carcinoma
This study aims to investigate the efficacy and prognosis of ultrasound-guided and gene-based microwave ablation (MWA) versus surgical treatment in patients with low-risk papillary thyroid carcinoma (PTC).
- Trial name
- NCT06725576
- Testing
- Ablation
- Where
- China
- Volunteers
- 201 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Non-small Cell Lung Cancer, EGFR Gene Mutation
Nintedanib Plus EGFR TKI In EGFR-mutated Non-small Cell Lung Cancer Patients
The purpose of this study is to evaluate the efficacy and safety of Nintedanib with EGFR-TKI in participants with advanced EGFR-TKI-resistant non-small cell lung cancer
- Conditions
- Non-small Cell Lung Cancer, EGFR Gene Mutation, EGFR-TKI Resistant Mutation
- Trial name
- NCT06071013
- Testing
- Nintedanib, gefitinib, erlotinib, afatinib, osimertinib
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- China Medical University Hospital
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.
KLF4 Methylation, Epigenetic
KLF4 Epigenetic Modulation
The study investigates the epigenetic regulation of KLF4 during the progression from colorectal polyps to adenomas and carcinomas.
- Trial name
- POLYEP (NCT07901335)
- Where
- Italy
- Volunteers
- 170 people
- Sponsor
- Azienda USL Reggio Emilia - IRCCS
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Invasive Mammary Carcinoma, Metastatic Breast Cancer
Gene Signatures to Guide HR+MBC Therapy in a Diverse Cohort
This is an open-label, multicenter, two-arm Phase II clinical trial that will evaluate the impact of 2nd line chemotherapy (i.e.
- Trial name
- INSIGHT (NCT05693766)
- Testing
- Capecitabine, Endocrine-therapy, MammoPrint ® and BluePrint assays
- Where
- 4 sites in United States
- Volunteers
- 64 people
- Sponsor
- Sonya Reid
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
Patients Must Have Histologically or Cytologically Confirmed Locally Advanced or Metastatic NSCLC, EGFR Mutation-negative and ALK Rearrangement-negative
Efficacy and Safety of Ivonescimab Monotherapy as First-Line Treatment for PD-L1-Positive, Driver Gene-Negative, Locally Advanced or Metastatic NSCLC: A Multicenter, Prospective, Real-World Cohort Study
The treatment decisions, assessment schedule, and study procedures in this research will adhere to the routine clinical practice of each participating center.
- Conditions
- Patients Must Have Histologically or Cytologically Confirmed Locally Advanced or Metastatic NSCLC, EGFR Mutation-negative and ALK Rearrangement-negative, Eastern Cooperative Oncology Group Performance Status (ECOG PS) Score of 0 to 2, No Prior Systemic Therapy Received for the Advanced or Metastatic Disease
- Trial name
- PROMISE (NCT07696832)
- Testing
- Ivonescimab Injection
- Where
- China
- Volunteers
- 265 people
- Sponsor
- Guangdong Association of Clinical Trials
Severe Combined Immunodeficiency
Autologous Gene Therapy for Artemis-Deficient SCID
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene.
- Trial name
- NCT03538899
- Testing
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, San Francisco
Idiopathic Parkinson's Disease (PD)
Study on the Incidence of Malignant Neoplasms in Patients With Parkinson's Disease and Heterozygous Mutation of the GBA Gene
This multicenter retrospective observational study investigates the incidence of malignant neoplasms in Parkinson's disease (PD) patients carrying heterozygous pathogenic variants in the GBA1 gene.
- Trial name
- ONCOGBA (NCT06814431)
- Where
- 4 sites in Italy
- Volunteers
- 3,000 people
- Sponsor
- Azienda USL Reggio Emilia - IRCCS
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