Clinical trials: Mutation
Acute Myeloid Leukemia, TP53 Gene Mutation
Axatilimab Combined With Decitabine/Venetoclax for the Treatment of TP53-mutated AML
Axatilimab combined with Decitabine/Venetoclax for the treatment of TP53-mutated/deleted AML patients
- Trial name
- NCT07511062
- Testing
- Decitabine, Venetoclax, Axatilimab
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Northside Hospital, Inc.
Solid Tumor (Phase I), RAS Mutation
A Study of AN4035 in Advanced Cancers With RAS Mutations and High CEACAM5 Expression
The goal of this clinical trial is to determine whether AN4035 is safe and tolerable in people with advanced or metastatic solid tumors that have Rat Sarcoma oncogene (RAS) mutated solid tumors and high levels of the CEACAM5…
- Conditions
- Solid Tumor (Phase I), RAS Mutation, CEACAM5
- Trial name
- NCT07686445
- Testing
- Intravenous AN4035, Intravenous AN4035 followed by intravenous EGFR Inhibitor.
- Where
- 2 sites in Australia
- Volunteers
- 228 people
- Sponsor
- Adlai Nortye Biopharma Co., Ltd.
Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera
Flonoltinib Maleate Oral Regimens in Patients With Myelofibrosis
The goal of this clinical trial is to learn which of three different doses of Flonoltinib Maleate taken by mouth daily works best to treat adult patients with myelofibrosis in whom the most common approved therapy has failed to…
- Conditions
- Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera, Myelofibrosis Transformation in Essential Thrombocythemia, Myelofibrosis With High Molecular Risk Mutations
- Trial name
- FM-MF-02 (NCT07750574)
- Testing
- Flonoltinib Maleate orally
- Volunteers
- 105 people
- Sponsor
- Chengdu Zenitar Biomedical Technology Co., Ltd
Ovarian Neoplasms, BRCA 1 Gene Mutation
Ovarian Cancer Liquid Biopsy for Early Assessment & Detection in Individuals With BRCA1/2 Pathogenic Variants
The goal of this clinical study is to evaluate the feasibility and acceptability of the Galleri multi-cancer early detection blood test in people with BRCA1 or BRCA2 gene changes who are at high risk for ovarian cancer.
- Conditions
- Ovarian Neoplasms, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation, Serous Tubal Intraepithelial Carcinoma
- Trial name
- OC-LEAD (NCT07764744)
- Testing
- Galleri Multi-Cancer Early Detection (MCED) Blood Test
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- Weill Medical College of Cornell University
BRAF NP_004324.2:p.V600E, BRAF V600K Mutation Present
Dabrafenib, Trametinib, and IMRT in Treating Patients With BRAF Mutated Anaplastic Thyroid Cancer
This trial studies how well dabrafenib, trametinib, and intensity modulated radiation therapy (IMRT) work together in treating patients with BRAF mutated anaplastic thyroid cancer.
- Conditions
- BRAF NP_004324.2:p.V600E, BRAF V600K Mutation Present, Thyroid Gland Anaplastic Carcinoma
- Trial name
- NCT03975231
- Testing
- Dabrafenib, Intensity-Modulated Radiation Therapy, Trametinib
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- City of Hope Medical Center
Advanced Non-small Cell Lung Cancer, HER2 Mutation
A Study to Learn More About How Well Sevabertinib Works and How Safe it is Compared With Standard Treatment, in Participants Who Have Advanced Non-small Cell Lung Cancer (NSCLC) With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)
Researchers are looking for a better way to treat people who have advanced non-small cell lung cancer (NSCLC) with specific genetic changes called human epidermal growth factor receptor 2 (HER2) mutations.
- Trial name
- SOHO-02 (NCT06452277)
- Testing
- Sevabertinib, Pembrolizumab, Cisplatin
- Where
- 279 sites in United States, Argentina and 34 more
- Volunteers
- 444 people
- Sponsor
- Bayer
Non-Small Cell Lung Cancer With Mutation in Epidermal Growth Factor Receptor
Osimertinib and Cyclosporine A to Enhance Antitumor Efficacy in NSCLC
This single-site, biomarker-enriched, randomized Phase II study will evaluate osimertinib with or without cyclosporine A (CsA) in metastatic epidermal growth factor receptor (EGFR )-mutant non-small cell lung cancer (NSCLC) [exon…
- Trial name
- OCEAN (NCT07874490)
- Testing
- Osimertinib, Cyclosporine A (CsA), ctDNA Biopsy Panel
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- NYU Langone Health
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of…
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Trial name
- KH176-301 (NCT06451757)
- Testing
- Sonlicromanol
- Where
- 10 sites in United States, Denmark and 5 more
- Volunteers
- 220 people
- Sponsor
- Khondrion BV
Metastatic Hepatocellular Carcinoma, Solid Tumors
Safety and Preliminary Anti-Tumor Activity of TYRA-430 in Advanced Hepatocellular Carcinoma and Other Solid Tumors With Activating FGF/FGFR Pathway Aberrations
A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamic (PD), and preliminary antitumor activity of TYRA-430 in cancers with FGF/FGFR pathway aberrations, including locally advanced/metastatic…
- Conditions
- Metastatic Hepatocellular Carcinoma, Solid Tumors, Solid Tumor, Adult, FGFR Gene Amplification
- Trial name
- SURF431 (NCT06915753)
- Testing
- TYRA-430
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 125 people
- Sponsor
- Tyra Biosciences, Inc
Non-Small-Cell Lung Cancer, Metastatic Non-Small-Cell Lung Cancer
Study to Evaluate Efficacy and Safety of Firmonertinib Compared With Investigator's Choice of EGFR Inhibitor as First-Line Treatment in Participants Who Have Locally Advanced or Metastatic NSCLC With EGFR P-Loop and Alpha C-Helix Compressing (PACC) Uncommon Mutations
Global, Phase 3, randomized, multicenter, open-label study evaluating the efficacy and safety of firmonertinib at a dose level of 240 mg QD compared to investigator's choice of osimertinib (80 mg QD) or afatinib (40 mg QD) in…
- Conditions
- Non-Small-Cell Lung Cancer, Metastatic Non-Small-Cell Lung Cancer, Advanced Non-Small-Cell Lung Cancer, EGFR P-Loop and Alpha C-Helix Compressing
- Trial name
- NCT07185997
- Testing
- Firmonertinib, EGFR-TKI inhibitor based on investigator's choice
- Where
- 139 sites in United States, Australia and 15 more
- Volunteers
- 480 people
- Sponsor
- ArriVent BioPharma, Inc.
Epidermal Growth Factor, Epidermal Growth Factor Receptor
A Phase 1 Study of EPI-326 in EGFR-mutant NSCLC and HNSCC
A phase 1 study to determine the safety, tolerability, PK, PD, and preliminary anti-tumor activity of ascending doses of EPI-326 administered to patients with locally advanced or metastatic HNSCC and to patients with any…
- Conditions
- Epidermal Growth Factor, Epidermal Growth Factor Receptor, Epidermal Growth Factor Receptor Gene Mutation, Non Small Cell
- Trial name
- NCT07462377
- Testing
- EPI-326
- Where
- 8 sites in United States, South Korea and 1 more
- Volunteers
- 110 people
- Sponsor
- EpiBiologics
Peroxisome Biogenesis Disorder, Zellweger Spectrum Disorder
Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
The Peroxisome Biogenesis Disorders (PBD) are a group of inherited disorders due to defects in peroxisome assembly causing complex developmental and metabolic sequelae.
- Conditions
- Peroxisome Biogenesis Disorder, Zellweger Spectrum Disorder, RCDP - Rhizomelic Chondrodysplasia Punctata, D-Bifunctional Protein Deficiency
- Trial name
- NCT01668186
- Where
- Canada
- Volunteers
- 244 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant)
An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities
This is a phase 2 open-label, one-arm, clinical study to evaluate the efficacy and safety of DCCR (diazoxide choline) extended-release tablets over 1 year of treatment, in patients with genetic obesities.
- Conditions
- SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity
- Trial name
- NCT05532020
- Testing
- DCCR (diazoxide choline) extended-release tablets
- Sponsor
- Soleno Therapeutics, Inc.
Non-small Cell Lung Cancer, Histiocytic Neoplasm
Phase 1/2 Trial of S241656 in Selected RAS/MAPK Mutation- Positive Malignancies
BDTX-4933-101 is a first-in-human, open-label, Phase 1/2 dose escalation, dose optimization and expansion study designed to evaluate the safety and tolerability of S241656 as monotherapy and in combination with other anti-cancer…
- Conditions
- Non-small Cell Lung Cancer, Histiocytic Neoplasm, Histiocytosis, BRAF Gene Mutation
- Trial name
- NCT05786924
- Testing
- S241656, FOLFOX6/FOLFOX7, FOLFIRI
- Where
- 49 sites in United States, Australia and 8 more
- Volunteers
- 554 people
- Sponsor
- Institut de Recherches Internationales Servier
Esophageal Cancer, Gastric Adenocarcinoma
A Study of Pembrolizumab With Trastuzumab and Chemotherapy in People With Esophagogastric Cancer
The purpose of this study to find out whether adding trastuzumab and pembrolizumab to standard chemotherapy is an effective treatment for resectable HER2+ esophagogastric cancer.
- Conditions
- Esophageal Cancer, Gastric Adenocarcinoma, HER2 Gene Mutation
- Trial name
- NCT06123338
- Testing
- Pembrolizumab, Trastuzumab, Oxaliplatin
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen
Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen
Study CL3-95031-007 (CHONQUER) is a Phase 3, international, multicenter, double-blind, randomized, placebo-controlled study of orally administered ivosidenib.
- Trial name
- CHONQUER (NCT06127407)
- Testing
- Ivosidenib 500mg
- Where
- 115 sites in United States, Australia and 15 more
- Volunteers
- 136 people
- Sponsor
- Servier Bio-Innovation LLC
Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation
Ivosidenib Plus Durvalumab and Gemcitabine/Cisplatin as First-Line Therapy in Participants With Locally Advanced or Metastatic Cholangiocarcinoma With an IDH1 Mutation
The objective of this study is to investigate the safety, tolerability and preliminary activity of ivosidenib in combination with durvalumab and gemcitabine/cisplatin as first-line therapy in participants with locally advanced…
- Trial name
- NCT06501625
- Testing
- Ivosidenib, Durvalumab (for the first 8, 21-day, cycles), Gemcitabine (for the first 8, 21-day, cycles)
- Where
- 38 sites in United States, Australia and 7 more
- Volunteers
- 55 people
- Sponsor
- Institut de Recherches Internationales Servier
Lung Cancer, Adjuvant
A Study to Assess Zipalertinib Versus Placebo in Participants With Early Stage NSCLC With Uncommon EGFR Mutations, Following Complete Tumor Resection
The purpose of this study is to compare the efficacy of zipalertinib versus placebo in participants with early stage resected non-small cell lung cancer (NSCLC) harboring uncommon epidermal growth factor receptor mutation…
- Conditions
- Lung Cancer, Adjuvant, Post-surgical, EGFR
- Trial name
- REZILIENT4 (NCT07128199)
- Testing
- TAS6417
- Where
- 253 sites in United States, Argentina and 22 more
- Volunteers
- 360 people
- Sponsor
- Taiho Oncology, Inc.
NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations
Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations
This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.
- Trial name
- MASter-1 (NCT04641442)
- Testing
- MAS825
- Where
- 12 sites in United States, Czechia and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
Cardiovascular Disease, Arterial Stiffness
Natural History Study of CADASIL
Background: CADASIL (cerebral autosomal dominant arteriopathy with subcortical infarct and leukoencephalopathy) is a genetic disorder. It causes narrowing of the small blood vessels and can lead to strokes and dementia.
- Conditions
- Cardiovascular Disease, Arterial Stiffness, Germline Mutation in the NOTCH 3 Gene, Pathogenesis of CADASIL
- Trial name
- NCT05072483
- Testing
- MRI
- Where
- United States
- Volunteers
- 155 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)