Clinical trials: Genetic Mutation
Malignant Brain Tumors
Phase I Study of HSK42360 in Malignant Brain Tumors With BRAF V600 Mutation
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK of HSK42360 when given orally in pediatric patients with active BRAF V600 mutation recurrent malignant brain tumors.
- Trial name
- NCT07158710
- Testing
- HSK42360
- Where
- 6 sites in China
- Volunteers
- 159 people
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Diffuse Large B Cell Lymphoma
Dose Escalation Study in Patients With Relapsed or Refractory DLBCL and MyD88 L265P Mutation
Recent reports have identified a specific oncogenic mutation L265P of the MYD88 gene in approximately 30% of the patients with the activated B-cell (ABC) type of Diffuse Large B Cell Lymphoma (DLBCL).
- Trial name
- NCT02252146
- Testing
- IMO-8400
- Where
- 12 sites in United States
- Volunteers
- 6 people
- Sponsor
- Idera Pharmaceuticals, Inc.
VHL - Von Hippel-Lindau Syndrome, VHL Gene Mutation
A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)
This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.
- Conditions
- VHL - Von Hippel-Lindau Syndrome, VHL Gene Mutation, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03401788
- Testing
- Belzutifan
- Where
- 11 sites in United States, Denmark and 2 more
- Volunteers
- 50 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Acute Myeloid Leukemia, Relapsed Adult AML
Ivosidenib Expanded Access Program in Relapsed/Refractory AML With an IDH1 Mutation
To provide access to ivosidenib monotherapy to patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation.
- Conditions
- Acute Myeloid Leukemia, Relapsed Adult AML, Relapsed Pediatric AML
- Trial name
- NCT03245424
- Testing
- ivosidenib (AG-120)
- Sponsor
- Agios Pharmaceuticals, Inc.
EGFR Mutation Positive Non-small Cell Lung Cancer, EGFR Mutated Non-small Cell Lung Cancer Patients
Study of NXP900 With Osimertinib in Subjects With Advanced, EGFR-Mutated Non-Small Cell Lung Cancer
This is a multi-center, open label, Phase 1b study of NXP900 in combination with osimertinib in subjects with advanced, progressing, EGFR-mutated non-small cell lung cancer (NSCLC)
- Trial name
- NCT07315113
- Testing
- NXP900, Osimertinib
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Nuvectis Pharma, Inc.
Melanoma
HL-085 in NRAS-mutated Advanced Melanoma
This was an open-label, single-arm, multi-center phase II clinical study, aimed at investigating the efficacy and safety of HL-085 capsule in the treatment of advanced melanoma patients with NRAS mutation.
- Trial name
- NCT05217303
- Testing
- HL-085
- Where
- 2 sites in China
- Volunteers
- 100 people
- Sponsor
- Shanghai Kechow Pharma, Inc.
Ovarian Cancer, Small Cell Lung Cancer
A Study of LG00313112 in Participants With Advanced Solid Malignancies Harboring a TP53 Y220C Mutation
This is a first-in-human, Phase 1/2, open-label study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of LG00313112 in participants with advanced solid malignancies harboring a TP53 Y220C…
- Conditions
- Ovarian Cancer, Small Cell Lung Cancer, Non Small Cell Lung Cancer, Colorectal Cancer
- Trial name
- NCT07752875
- Testing
- LG00313112
- Volunteers
- 250 people
- Sponsor
- LG Chem
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Newly Diagnosed Locally Advanced or Metastatic Non-small Cell Lung Cancer
Epidemiological and Scientific Evaluation of EGFR Mutation Status in Patients With Newly Diagnosed Locally Advanced or m
The primary objective of the study is to collect epidemiological data on EGFR mutation status (M+, M-) in a population of predominantly Caucasian ethnicity and to correlate EGFR mutation status with clinico-pathological…
- Trial name
- REASON (NCT00997230)
- Where
- 118 sites in Germany
- Volunteers
- 4,243 people
- Sponsor
- AstraZeneca
Advanced or Metastatic KRAS-mutant Tumor in Pancreatic Adenocarcinoma, Advanced or Metastatic KRAS-mutant Tumor in Colorectal Cancer
Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of WEF-001 as Monotherapy in Advanced KRAS-Mutant Solid Tumours.
This is a study designed to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of WEF-001 as monotherapy in patients with Advanced KRAS-mutant solid tumours.
- Conditions
- Advanced or Metastatic KRAS-mutant Tumor in Pancreatic Adenocarcinoma, Advanced or Metastatic KRAS-mutant Tumor in Colorectal Cancer, Advanced or Metastatic KRAS-mutant Tumor in Non Small Cell Lung Cancer, Advanced Solid Tumors
- Trial name
- NCT07148128
- Testing
- WEF-001
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 110 people
- Sponsor
- Auricula 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
- 141 sites in United States, Australia and 15 more
- Volunteers
- 480 people
- Sponsor
- ArriVent BioPharma, Inc.
Non-Small Cell Lung Cancer
DS-8201a in Human Epidermal Growth Factor Receptor 2 (HER2)-Expressing or -Mutated Non-Small Cell Lung Cancer
The primary objective of this trial is to evaluate the efficacy of trastuzumab deruxtecan in HER2-overexpressing and/or HER2-mutated advanced NSCLC participants.
- Trial name
- DESTINY-Lung01 (NCT03505710)
- Testing
- Trastuzumab deruxtecan
- Where
- 21 sites in United States, France and 3 more
- Volunteers
- 181 people
- Sponsor
- Daiichi Sankyo
KRAS G12C Mutation, KRAS G12S Mutation
A Study to Investigate the Safety and Efficacy of KQB365 as Monotherapy and in Combination in Participants With Advanced Solid Malignancies
The goal of this clinical trial is to learn if KQB365 works to treat advanced solid tumor cancer in adults. It will also learn about the safety of KQB365.
- Conditions
- KRAS G12C Mutation, KRAS G12S Mutation, Solid Tumor Malignancies, CRC (Colorectal Cancer)
- Trial name
- NCT06720987
- Testing
- KQB365, Cetuximab, KQB198
- Where
- 11 sites in United States
- Volunteers
- 140 people
- Sponsor
- Kumquat Biosciences Inc.
Advanced KRAS G12D Mutant Solid Tumors
Phase I Study of HRS-4642 in Patients With Advanced Solid Tumors Harboring KRAS G12D Mutation
The study is being conducted to evaluate the safety and tolerability of HRS-4642 in patients with advanced solid tumors harboring KRAS G12D mutation.To estimate the maximum tolerated dose (MTD) and/or a biologically active dose…
- Trial name
- NCT05533463
- Testing
- HRS-4642
- Where
- China
- Volunteers
- 102 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Advanced or Metastatic NSCLC
Phase 1/2 Study of HS-10375 in Patients with Advanced or Metastatic Non-Small-Cell Lung Cancer(NSCLC)
HS-10375 is an oral, highly selective, small molecular inhibitor of EGFR C797S. This study will evaluate the safety, tolerability, pharmacokinetics and clinical activity of HS-10375 in Chinese advanced or metastatic NSCLC.
- Trial name
- NCT05435248
- Testing
- HS-10375
- Where
- China
- Volunteers
- 354 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses.
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Trial name
- AURORA (NCT04123626)
- Testing
- QR-1123
- Where
- 5 sites in United States
- Volunteers
- 11 people
- Sponsor
- ProQR Therapeutics
Melanoma and Brain Metastases
Study to Evaluate Treatment of Dabrafenib Plus Trametinib in Subjects With BRAF Mutation-Positive Melanoma That Has Metastasized to the Brain
This is a multi-cohort, open label, Phase II study with Dabrafenib (GSK2118436) and Trametinib (GSK1120212) combination therapy in subject with BRAF mutation-positive melanoma that has metastasized to the brain.
- Trial name
- NCT02039947
- Testing
- Dabrafenib, Trametinib
- Where
- 47 sites in United States, Australia and 5 more
- Volunteers
- 127 people
- Sponsor
- Novartis Pharmaceuticals
Grade 2 Astrocytoma or Oligodendroglioma With an IDH1 or IDH2 Mutation
HMPL-306 in Participants With Postoperative Residual or Recurrent Grade 2 Glioma Harboring IDH1 or IDH2 Mutations
This study is a randomized, double-blind, placebo-controlled, multicenter Phase III clinical trial evaluating the efficacy and safety of HMPL-306 in participants with postoperative residual or recurrent grade 2 gliomas carrying…
- Trial name
- NCT07912398
- Testing
- HMPL-306 tablet
- Volunteers
- 210 people
- Sponsor
- Hutchmed
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Characteristics and Impacts of X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) in Boys: An Observational International Study
This is an observational, multicentre, international study over a 2-year follow-up period. The aim of this study is to understand how XLHED affects the lives of young male patients and their families over time.
- Trial name
- EdeReaLife (NCT07096206)
- Where
- 2 sites in France, Germany
- Volunteers
- 27 people
- Sponsor
- Pierre Fabre Medicament
Hemophilia A, Hemophilia B
Study of Ataluren (PTC124) in Hemophilia A and B
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting.
- Trial name
- NCT00947193
- Testing
- Ataluren
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
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