Clinical trials: Gene Mutations
Advanced NSCLC
AST2818 in Advanced Non-Small Cell Lung Cancer With EGFR Mutations
This study is conducted to assess the safety, tolerability and preliminary efficacy of AST2818 in patients with advanced Non Small Cell Lung Cancer (NSCLC).
- Trial name
- NCT03127449
- Testing
- Alflutinib
- Where
- China
- Volunteers
- 116 people
- Sponsor
- Allist Pharmaceuticals, Inc.
NSCLC
BBP-398 in Combination With Osimertinib in Locally Advanced or Metastatic NSCLC Patients With EGFR Mutations
This is an open-label, non-randomized, multi-cohort, multi-center Phase Ia/Ib study for BBP-398 in combination with Osimertinib to evaluate the safety, tolerability, pharmacokinetics, determine MTD and/or RP2D, and anti-cancer…
- Trial name
- NCT06032936
- Testing
- BBP-398, osimertinib
- Where
- 5 sites in China
- Volunteers
- 4 people
- Sponsor
- LianBio LLC
Solid Tumor
Dose Escalation Study of ARQ 736 in Adult Subjects With Advanced Solid Tumors Harboring BRAF and/or NRAS Mutations
This is an open-label, Phase 1, dose escalation study of oral ARQ 736 administered to subjects with advanced solid tumors harboring the mutation.
- Trial name
- NCT01225536
- Testing
- ARQ 736
- Where
- 4 sites in United States, Italy
- Volunteers
- 24 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Non-Small Cell Lung Cancer (NSCLC)
Study of Capmatinib in Chinese Adult Patients With Advanced Non-small Cell Lung Cancer Harboring MET Exon 14 Skipping Mutation
The purpose of the study was to learn whether the study treatment (capmatinib), which already shows efficacy and safety in non-Chinese patients, could help Chinese patients with controlling their lung cancer in a safe way.
- Trial name
- GeoMETry-C (NCT04677595)
- Testing
- Capmatinib
- Where
- 17 sites in China
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Non Small Cell Lung Cancer, Colorectal Cancer
A Dose Escalation Study of SHP2 Inhibitor in Patients With Solid Tumors Harboring KRAS of EGFR Mutations
A Phase 1 dose escalation study in patients with advanced solid tumors harboring KRAS or EGFR mutations to determine the maximum tolerated dose and recommended Phase II dose of HBI-2376 and characterize its pharmacokinetic…
- Conditions
- Non Small Cell Lung Cancer, Colorectal Cancer, Pancreatic Cancer, Solid Tumor
- Trial name
- NCT05163028
- Testing
- HBI-2376
- Where
- 12 sites in United States, Puerto Rico
- Volunteers
- 42 people
- Sponsor
- HUYABIO International, LLC.
Non Small Cell Lung Cancer, EGFR-TKI Resistant Mutation
Human EGFR Mutations Quantitative Detection Kit (Real-time Fluorescent PCR Method)
In this clinical trial, investigators select FFPE and plasma samples of non-small cell lung cancer which are used for quantitative detection of four kinds of EGFR(Epidermal Growth Factor Receptor) mutations.
- Conditions
- Non Small Cell Lung Cancer, EGFR-TKI Resistant Mutation, EGFR-TKI Sensitizing Mutation
- Trial name
- NCT02661009
- Volunteers
- 1,000 people
- Sponsor
- GenoSaber
Acute Myeloid Leukemia
Study of CEP-701 (Lestaurtinib) in Patients With Acute Myeloid Leukemia (AML)
The purpose of the study is to determine whether CEP-701 given in sequence with induction chemotherapy increases the proportion of patients with relapsed acute myeloid leukemia (AML) who achieve a second complete remission (CR).
- Trial name
- NCT00079482
- Testing
- CEP-701, high-dose cytarabine, Mitozantrone, Etoposide, Cytarabine (combination Chemotherapy)
- Where
- 81 sites in United States, Australia and 11 more
- Volunteers
- 224 people
- Sponsor
- Cephalon
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
X-Linked Retinitis Pigmentosa
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
- Trial name
- NCT05874310
- Testing
- FT-002
- Where
- 2 sites in China
- Volunteers
- 18 people
- Sponsor
- Frontera Therapeutics
Carcinoma, Non-Small-Cell Lung, ErbB Receptors
Afatinib in EGFR+NSCLC (Recurrent or Stage IV) - Patients With Poor Performance Status (ECOG 2 or 3)
There is a medical need for improving treatment of poor performance status patients with EGFR driver mutations and documenting safety and tolerability of existing agents.
- Trial name
- NCT02695290
- Testing
- Afatinib
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Boehringer Ingelheim
Advanced and Selected Solid Tumors
A Study to Investigate Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) of BKM120 Plus GSK1120212 in Selected Advanced Solid Tumor Patients
This is an open label, dose finding, phase Ib clinical trial to determine the maximum tolerated dose (MTD) and /or recommended phase II dose (RP2D) and schedule for the PI3K (Phosphatidylinositol 3-Kinase) inhibitor BKM120 given…
- Trial name
- NCT01155453
- Testing
- BKM120, GSK1120212
- Where
- 7 sites in United States, Belgium and 3 more
- Volunteers
- 113 people
- Sponsor
- Novartis Pharmaceuticals
Glioma
Study of DS-1001b in Patients With Gene IDH1-Mutated Gliomas
This is a study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of DS-1001b in patients with gliomas that harbor IDH1-R132 mutations.
- Trial name
- NCT03030066
- Testing
- DS-1001b
- Where
- Japan
- Volunteers
- 47 people
- Sponsor
- Daiichi Sankyo
Hypertrophic Cardiomyopathy
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by…
- Trial name
- MyPEAK-1 (NCT05836259)
- Testing
- TN-201
- Where
- 10 sites in United States
- Volunteers
- 30 people
- Sponsor
- Tenaya Therapeutics
Retinitis Pigmentosa
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading…
- Trial name
- NCT03349242
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 140 people
- Sponsor
- MeiraGTx UK II Ltd
Fabry Disease
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A)…
- Trial name
- NCT01218659
- Testing
- migalastat hydrochloride, agalsidase
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 68 people
- Sponsor
- Amicus Therapeutics
Non-Small Cell Lung Cancer
A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer
This open-label, multi-center study will evaluate the progression-free survival and safety of erlotinib in participants with locally advanced or metastatic non-small cell lung cancer with activating mutations in the tyrosine…
- Trial name
- ESSENCE (NCT01667562)
- Testing
- Erlotinib
- Where
- 4 sites in Serbia
- Volunteers
- 375 people
- Sponsor
- Hoffmann-La Roche
Unspecified Adult Solid Tumor, Protocol Specific, Solid Tumor
Safety, Pharmacokinetics and Pharmacodynamics of BEZ235 Plus MEK162 in Selected Advanced Solid Tumor Patients
This is an open label, dose finding, phase Ib clinical trial to determine the maximum tolerated dose (MTD) and/or RP2D of the orally administered PI3K/mTOR inhibitor BEZ235 in combination with the MEK1/2 inhibitor MEK162.
- Trial name
- NCT01337765
- Testing
- BEZ235 + MEK162
- Where
- 6 sites in United States, Australia and 3 more
- Volunteers
- 29 people
- Sponsor
- Pfizer
Bardet-Biedl Syndrome 1, Retinal Degeneration
First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration
The goal of this first in human study is to evaluate the preliminary safety and tolerability of AXV-101 in participants with BBS1.
- Trial name
- AXIS (NCT07269665)
- Testing
- AXV-101
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Axovia Therapeutics
Non-small Cell Lung Cancer
A Phase II Clinical Trial of DZD9008 in Combination With AZD4205 in Standard Treatment Failed NSCLC Patients With EGFR Mutations (WU-KONG21)
DZD9008 in combination with AZD4205 for the treatment of patients with advanced NSCLC with EGFR mutations who have progressed after standard treatment.
- Trial name
- NCT06295432
- Testing
- DZD9008+AZD4205
- Where
- 4 sites in China
- Volunteers
- 30 people
- Sponsor
- Dizal Pharmaceuticals
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.