Clinical trials: Gene, open to volunteers
Evaluation, NSCLC
RecistTM Criteria in Evaluating the Efficacy of Targeted Therapy for NSCLC
Investigators established the efficacy evaluation criteria for tumor markers (RecistTM) in the preliminary research. Among patients with advanced non-small cell lung cancer, patients with positive driving genes are more likely to…
- Conditions
- Evaluation, NSCLC, Targeted Therapy
- Trial name
- NCT06142058
- Testing
- RecistTM criteria
- Where
- China
- Volunteers
- 44 people
- Sponsor
- Xueqin Yang
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Multiple Sclerosis
Genes and Environment in Multiple Sclerosis
The purpose of the research study is to identify the genetic, environmental and immune profiles that may increase a person's risk of developing multiple sclerosis (MS).
- Trial name
- GEMS (NCT01353547)
- Where
- 5 sites in United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Acute Myeloid Leukemia
Characterization of T-cell Repertoire in Patients With Acute Myeloid Leukemia Undergoing Donor Stem Cell Transplant
This research trial studies characterization of T-cell repertoire through next-generation sequencing in patients with acute myeloid leukemia undergoing stem cell transplant.
- Trial name
- NCT03921047
- Testing
- Biospecimen Collection, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- University of Southern California
Arrhythmogenic Cardiomyopathy, PKP2-ACM
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Trial name
- SNAPSHOT- PKP2 (NCT06976606)
- Where
- 6 sites in United States
- Volunteers
- 40 people
- Sponsor
- Lexeo Therapeutics
Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations.
- Conditions
- Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1
- Trial name
- NCT05735184
- Testing
- Ziftomenib, Venetoclax, Azacitidine
- Where
- 44 sites in United States
- Volunteers
- 420 people
- Sponsor
- Kura Oncology, Inc.
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
High Grade Glioma, Diffuse Intrinsic Pontine Glioma
A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion
This is a pilot study that will evaluate disease status in children that have been newly diagnosed high-grade glioma with TRK fusion. The evaluation will occur after 2 cycles of the medication (Larotrectinib) have been given.
- Trial name
- NCT04655404
- Testing
- Larotrectinib, Larotrectinib surgical
- Where
- 21 sites in United States, Australia and 3 more
- Volunteers
- 15 people
- Sponsor
- Nationwide Children's Hospital
HEPATITIS B CHRONIC
Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients With Chronic Hepatitis B
This is a Phase 1, open-label, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, and antiviral activity of PBGENE-HBV in adult participants with chronic hepatitis B.
- Trial name
- ELIMINATE-B (NCT06680232)
- Testing
- PBGENE-HBV
- Where
- 7 sites in United States, France and 4 more
- Volunteers
- 45 people
- Sponsor
- Precision BioSciences, Inc.
Childhood Acute Lymphoblastic Leukemia
NUDT15/TPMT Multi-gene Guided 6-MP Dosing in Childhood ALL Maintenance Therapy
This is a prospective, single-center clinical study to evaluate the safety and efficacy of a personalized 6-mercaptopurine (6-MP) dosing strategy guided by NUDT15 and TPMT genotypes in children with Acute Lymphoblastic Leukemia…
- Trial name
- NCT07462299
- Testing
- NUDT15/TPMT genotype-guided 6-MP dosing
- Where
- China
- Volunteers
- 110 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Mutations Affecting the COL5A Gene, Aneurysm Aortic
Fibroblast Line Evaluation for COL5A eXpression and Thoracic Aortic Aneurysms: in Vitro Characterization of Cutaneous Fibroblasts in Adult Patients
This study examines adults with COL5A gene mutations to understand why some develop aortic aneurysms while others do not. Participants provide a small skin biopsy, and researchers analyze fibroblasts to evaluate collagen…
- Trial name
- FLEX5 project (NCT07282717)
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS Policlinico S. Donato
Leber's Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- GOLD (NCT04912843)
- Testing
- NR082 injection
- Where
- China
- Volunteers
- 102 people
- Sponsor
- Wuhan Neurophth Biotechnology Limited Company
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Idiopathic Infantile Hypercalcaemia - Severe Form, Genetic Disease
Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
This study evaluates the efficacy of rifampin in the treatment of hypercalcemia and/or hypercalciuria in participants with at least one inactivating mutation of the CYP24A1 gene.
- Conditions
- Idiopathic Infantile Hypercalcaemia - Severe Form, Genetic Disease, Hypercalcemia, Idiopathic, of Infancy, Hypercalciuric Hypercalcemia
- Trial name
- RICHH (NCT03301038)
- Testing
- Rifampin
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Philadelphia
Huntington Disease
IMarkHD: in Vivo Longitudinal Imaging of HD Pathology
iMarkHD is an adaptive, longitudinal positron emission tomography (PET) and magnetic resonance (MR) imaging study in Huntington's disease (HD) that aims to assess abnormal molecular, functional, and structural changes in…
- Trial name
- NCT03434548
- Testing
- PET imaging, Multi-modal MRI imaging
- Where
- United Kingdom
- Volunteers
- 113 people, healthy volunteers welcome
- Sponsor
- King's College London
BRCA Mutation, Ovarian Cancer
Prediction of Germline BRCA 1/2 Genes From Healthy Ovaries
The project aims at enhancing performance metrics and prospectively validating a radiogenomics model based on ovarian US images for predicting germline breast cancer susceptibility gene 1 and/or 2 (BRCA) status in women with…
- Conditions
- BRCA Mutation, Ovarian Cancer, Ultrasound Therapy; Complications
- Trial name
- PROBE-II (NCT05769517)
- Testing
- Germinal BRCA
- Where
- Italy
- Volunteers
- 6,465 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Pancreatic Ductal Adenocarcinoma (PDAC), Colorectal Cancer (CRC)
Advanced Therapies for Liver Metastases
Liver metastases (MTS) are the main cause of death for patients affected by colorectal carcinoma (CRC) and pancreatic ductal adenocarcinoma (PDAC), thus representing the major unmet clinical need for these malignancies.
- Conditions
- Pancreatic Ductal Adenocarcinoma (PDAC), Colorectal Cancer (CRC), Liver Metastasis, IPMN, Pancreatic
- Trial name
- LiMeT (NCT04622423)
- Where
- Italy
- Volunteers
- 625 people, healthy volunteers welcome
- Sponsor
- IRCCS San Raffaele
FGFR2 Gene Fusion/Rearrangement, Other Solid Tumors, Adult
A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement
The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma)…
- Trial name
- ReFocus202 (NCT07359820)
- Testing
- Lirafugratinib
- Where
- 19 sites in United States, France and 3 more
- Volunteers
- 30 people
- Sponsor
- Elevar Therapeutics
Hemophilia B
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05641610
- Testing
- ZS801
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Systemic Lupus Erythematosus
" TREX1 Gene Mutations and Their Role in Systemic Lupus Erythematosus
Systemic lupus erythematosus (SLE) is a multisystem autoimmune disease characterized by diverse clinical manifestations, prominently involving the skin.
- Trial name
- NCT07397728
- Testing
- TREX1 gene polymorphisms
- Where
- Egypt
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- South Valley University
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