Clinical trials: Mutations, open to volunteers
CML (Chronic Myelogenous Leukemia), Chronic Phase CML
ELVN-001 for the Treatment of Chronic Myeloid Leukemia With and Without T315I Mutation in Japanese Participants
The purpose of this study is to evaluate the safety, tolerability and determine the recommended dose for further clinical evaluation of ELVN-001 in Japanese patients with chronic phase chronic myeloid leukemia with and without…
- Trial name
- CML (NCT06787144)
- Testing
- ELVN-001
- Where
- 4 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Enliven Therapeutics
Non-Small Cell Lung Cancer, Epidermal Growth Factor Receptor
Bolstering Outcomes After Induction With Osimertinib Plus Chemotherapy Through Optimized Site-Directed Primary Tumor Therapy (BOOST Trial)
This is a single-arm, open-label, phase II study evaluating the clinical outcomes of local therapy (surgery or radiotherapy) to the primary tumor in patients with EGFR-mutant advanced non-small cell lung cancer (NSCLC) who have…
- Trial name
- BOOST (NCT07073365)
- Testing
- Local Therapy (Surgery or Radiotherapy)
- Where
- South Korea
- Volunteers
- 70 people
- Sponsor
- Pusan National University Hospital
Solid Tumors Harboring a TP53 Y220C Mutation
A Phase I First-in-Human Study of GenSci128 in Patients With Solid Tumors Harboring a TP53 Y220C Mutation
The purpose of this study is to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of GenSci128 Tablet in Patients with Locally Advanced or Metastatic Solid Tumors Harboring a TP53 Y220C Mutation
- Trial name
- NCT06908434
- Testing
- GenSci128 tablets
- Where
- China
- Volunteers
- 82 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
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.
Chronic Hepatitis B
Molecular Characteristics of Hepatitis B Virus Integration, Mutation, and Drug Resistance
Patients with a confirmed diagnosis of chronic hepatitis B (CHB) who have complete clinical data and retained blood or liver tissue samples will be enrolled.
- Trial name
- HBV IMDR (NCT06779864)
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Beijing Friendship Hospital
Pediatric Cancer, Li-Fraumeni Syndrome
Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer
This prospective, single-arm, multi-center clinical trial aims to explore and evaluate the efficacy and safety of arsenic trioxide combined with chemotherapy for pediatric cancer with p53 mutation.
- Conditions
- Pediatric Cancer, Li-Fraumeni Syndrome, p53 Mutations
- Trial name
- NCT06088030
- Testing
- Arsenic trioxide
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Yang Li
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
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
Gliomas Harboring IDH1 and/or IDH2 Mutations
Phase I Study of HMPL-306 for the Treatment of Gliomas With IDH1 and/or IDH2 Mutations
This study is a multicenter, randomized controlled Phase I clinical study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of HMPL-306 in patients with gliomas harboring IDH1…
- Trial name
- NCT07025018
- Testing
- HMPL-306
- Where
- China
- Volunteers
- 52 people
- Sponsor
- Hutchmed
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
Pancreatic Cancer
A Phase II Study of GFH375 Combined With GFS202A or GFH276 Versus GFH375 in Patients With Previously Treated Locally Advanced Unresectable or Metastatic Pancreatic Cancer Harboring KRAS G12D Mutation
This is a phase 2 study. All subjects are patients with locally advanced unresectable or metastatic pancreatic cancer harboring KRAS G12D mutation who have received at least one prior line of systemic therapy.
- Trial name
- NCT07784374
- Testing
- GFH375, GFH276, GFS202A
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency
Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of…
- Trial name
- ARTEGENE (NCT05071222)
- Testing
- ARTEGENE drug product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Duodenum Cancer, Familial Adenomatous Polyposis
Duodenal Polyposis Classification in FAP
Duodenal cancer is the leading cause of cancer-related mortality in patients with familial adenomatous polyposis (FAP), yet the current Spigelman staging system provides limited predictive accuracy for advanced neoplasia.
- Conditions
- Duodenum Cancer, Familial Adenomatous Polyposis, Duodenal Polyposis, Ampulla of Vater Adenoma
- Trial name
- DRACO (NCT07199127)
- Testing
- DRACO
- Where
- 2 sites in Italy
- Volunteers
- 300 people
- Sponsor
- Fondazione IRCCS Istituto Nazionale dei Tumori, Milano
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood…
- Trial name
- NCT06090669
- Testing
- TruSight Oncology, imatinib
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Inflammatory Disease, Genetic Disease
Molecular Diagnosis of Systemic Autoinflammatory Diseases
Systemic autoinflammatory diseases (SAIDs) are a set of rare clinically and genetically heterogeneous conditions. The project proposes to identify novel genes and specific signatures in subgroups of patients with SAIDs.
- Conditions
- Inflammatory Disease, Genetic Disease, Somatic Mutation, Molecular Sequence Variation
- Trial name
- SAIDiag (NCT05364294)
- Where
- France
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
BRCA1 Mutation, BRCA2 Mutation
BRCA Mutation Carriers' Platform a Multicenter Study
Subjects who carry mutations in breast cancer susceptibility genes 1 and 2 (BRCA1 and BRCA2 genes) are at higher risk of developing cancers.
- Trial name
- BRCA_ENDORSE (NCT07253051)
- Testing
- Registration of clinical datas in the platform
- Where
- 40 sites in Italy
- Volunteers
- 10,000 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Somatic Mutation
Linking Somatic Mutation Rate With Baseline Exposure in East Palestine
The goal of this study is to This research team is conducting this study to develop methods to measure the biological impact of exposure to the chemicals released following the February 3, 2023 train derailment on residents of…
- Trial name
- NCT06357845
- Testing
- Survey, Biological specimen collection, Communications about study updates and future research opportunities
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Case Comprehensive Cancer Center
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