Clinical trials: Gene Mutations
Ovarian Cancer
A Study of Adavosertib (MK-1775) in Combination With Paclitaxel and Carboplatin Versus Paclitaxel and Carboplatin Alone for Participants With Platinum-Sensitive Ovarian Tumors With the P53 Gene Mutation (MK-1775-004)
This is a study of the safety and efficacy of adavosertib in combination with paclitaxel plus carboplatin in the treatment of ovarian, fallopian tube, and primary peritoneal tumors with the P53 mutation.
- Trial name
- NCT01357161
- Testing
- adavosertib, paclitaxel, carboplatin
- Volunteers
- 136 people
- Sponsor
- Merck Sharp & Dohme LLC
Malignant Neoplasm
KEYMAKER-U01 Substudy 01J: A Study of Pembrolizumab Plus MK-1084 in Participants With Non-Small Cell Lung Cancer (NSCLC) With Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) G12C Mutations (MK-3475-01J/KEYMAKER-U01J)
Researchers want to learn if using a study medicine called MK-1084 can help treat Non-Small Cell Lung Cancer (NSCLC). MK-1084 is a type of treatment called targeted therapy for the Kirsten rat sarcoma viral oncogene homolog…
- Trial name
- NCT07252739
- Testing
- MK-1084, Pembrolizumab, Cetuximab
- Where
- 39 sites in United States, Chile and 11 more
- Volunteers
- 140 people
- Sponsor
- Merck Sharp & Dohme LLC
Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
Phase 3 Study of BGJ398 (Oral Infigratinib) in First Line Cholangiocarcinoma With FGFR2 Gene Fusions/Translocations
Infigratinib is an oral drug which selectively binds to fibroblast growth factor receptor (FGFR) 2 and is being developed to treat participants with FGFR2 mutated cholangiocarcinoma.
- Trial name
- NCT03773302
- Testing
- BGJ398, Gemcitabine, Cisplatin
- Where
- 116 sites in United States, Australia and 13 more
- Volunteers
- 48 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Non-Small Cell Lung Cancer, EGFR Gene Mutation
D-0316 Versus Icotinib in Patients With Locally Advanced or Metastatic EGFR Sensitising Mutation Positive NSCLC
To assess the efficacy and safety of D-0316 versus Icotinib, a standard of care epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), in patients with locally advanced or Metastatic Non Small Cell Lung Cancer…
- Trial name
- NCT04206072
- Testing
- D-0316 Capsule, Icotinib Hydrochloride Tablets
- Where
- 2 sites in China
- Volunteers
- 362 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Carcinoma, Non Small Cell Lung
A Study of Pemetrexed and Gefitinib Versus Gefitinib in Non-Small Cell Lung Cancer (NSCLC)
The purpose of this study is to compare the combination of pemetrexed and gefitinib versus gefitinib alone, in terms of progression-free survival.
- Trial name
- NCT01469000
- Testing
- Gefitinib, Pemetrexed
- Where
- 25 sites in China, Japan and 2 more
- Volunteers
- 195 people
- Sponsor
- Eli Lilly and Company
Non-GCB/ABC Diffuse Large B-Cell Lymphoma, With and Without MyD88 and/or CD79B Mutations
Study of Mivavotinib (CB-659) in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)
Study CX-659-401 is a multicenter, open-label, phase 2 study of mivavotinib to evaluate the single-agent activity of mivavotinib in patients with relapsed/refractory non-GCB/ABC DLBCL, incorporating ctDNA-based next-generation…
- Trial name
- NCT05319028
- Testing
- Mivavotinib
- Where
- 7 sites in United States
- Volunteers
- 2 people
- Sponsor
- Calithera Biosciences, Inc
Hepatitis C, Chronic
An Observational Study on Long-Term Persistence of Resistant Mutations And Durability of Sustained Virological Response in Patients With Chronic Hepatitis C Treated With Direct Acting Antiviral (DAA)- Containing Regimens
This observational long-term follow-up study will assess the persistence of direct acting antiviral (DAA) resistant mutations and the durability of sustained virological response in patients with chronic hepatitis C who have…
- Trial name
- NCT01168856
- Where
- 126 sites in United States, Australia and 13 more
- Volunteers
- 734 people
- Sponsor
- Hoffmann-La Roche
Isocitrate Dehydrogenase Gene Mutation
A Study of HMPL-306 in Advanced Solid Tumors With IDH Mutations
An open label single-arm clinical trial to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of HMPL-306 in advanced or metastatic solid tumors with IDH mutation.
- Trial name
- NCT04762602
- Testing
- HMPL-306
- Where
- 11 sites in United States, Spain
- Volunteers
- 42 people
- Sponsor
- Hutchmed
Gaucher Disease, Type 2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2).
- Trial name
- NCT04411654
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Prevail Therapeutics
Leukemia, Myeloid, Acute
A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy
The purpose of this study are 1. to determine the recommended combination dose of AG-120 and AG-221 separately when administered with azacitidine and, 2.
- Trial name
- NCT02677922
- Testing
- AG-120, Azacitidine, AG-221
- Where
- 49 sites in United States, Australia and 12 more
- Volunteers
- 130 people
- Sponsor
- Celgene
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to…
- Trial name
- CHORD (NCT05788536)
- Testing
- DB-OTO
- Where
- 17 sites in United States, Germany and 3 more
- Volunteers
- 36 people
- Sponsor
- Regeneron Pharmaceuticals
Retinitis Pigmentosa, Leber Congenital Amaurosis
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101).
- Trial name
- NCT05203939
- Testing
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose
- Where
- 7 sites in United States
- Volunteers
- 22 people
- Sponsor
- Ocugen
Solid Tumors
Study of RXDX-105, Potent RET Inhibitor in Patients With Advanced Lung Cancer and Other Solid Tumors
This is a first-in-human, multicenter, open-label study consisting of 2 phases. Phase 1 is a dose escalation study of RXDX-105 (formerly known as CEP-32496) in patients with advanced solid tumors aimed at defining the recommended…
- Trial name
- NCT01877811
- Testing
- RXDX-105
- Where
- 14 sites in United States
- Volunteers
- 143 people
- Sponsor
- Hoffmann-La Roche
Solid Tumor, Breast Tumor
Niraparib in the Treatment of Patients With Advanced PALB2 Mutated Tumors
The purpose of this study is to further evaluate the efficacy and safety of niraparib in patients with locally advanced or metastatic solid tumors and a pathogenic or likely pathogenic tumor PALB2 (tPALB2) mutation.
- Conditions
- Solid Tumor, Breast Tumor, Colon Tumor, Malignant, Lung Tumor
- Trial name
- PAVO (NCT05169437)
- Testing
- Niraparib
- Where
- 80 sites in United States
- Volunteers
- 22 people
- Sponsor
- Tempus AI
Pro-opiomelanocortin (POMC), Proprotein Convertase Subtilisin/Kexin Type 1 (PCSK1) and Leptin Receptor (LepR) Gene Mutations
Genetic Testing and Phenotypic Characterization of Severely Obese Pediatric and Adult Volunteers
The purpose of this screening study is to identify people who have a rare genetic cause of obesity - specifically three genetic variants (a change in the DNA structure) of the POMC, PCSK1 and LepR genes that are currently known…
- Trial name
- NCT02849977
- Where
- 58 sites in United States, Canada and 5 more
- Volunteers
- 5,966 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Non-Small Cell Lung Cancer
A Study of Erlotinib (Tarceva) Versus Gemcitabine/Cisplatin as First-line Treatment in Patients With Non-small Cell Lung Cancer With EGFR Mutations
This open-label, randomized, parallel arm study assessed the efficacy and safety of Tarceva (erlotinib) versus gemcitabine/cisplatin combination chemotherapy as first-line treatment in patients with stage IIIB/IV non-small cell…
- Trial name
- NCT01342965
- Testing
- Erlotinib, Chemotherapy
- Where
- 28 sites in China, Malaysia and 1 more
- Volunteers
- 217 people
- Sponsor
- Hoffmann-La Roche
Human Immunodeficiency Virus Infection
A Safety and Efficacy Study of Amdoxovir in HIV-1 Treatment-experienced Subjects.
This is a double-blind Phase 2a study to test the safety and efficacy of an investigational HIV drug, amdoxovir (300 mg or 500 mg twice daily) compared with tenofovir DF 300 mg once daily in HIV-1 infected antiretroviral…
- Trial name
- NCT01737359
- Testing
- amdoxovir 300 mg bid, amdoxovir 500 mg bid, tenofovir DF 300 mg qd
- Where
- 6 sites in Argentina
- Volunteers
- 2 people
- Sponsor
- RFS Pharma, LLC
Non-Small Cell Lung Cancer, Squamous Non-small-cell Lung Cancer
A Study of Sapanisertib in Relapsed/Refractory NFE2L2-Mutated and Wild-Type Squamous Non-Small Cell Lung Cancer
This is a multicenter, randomized, open-label Phase 2 study of sapanisertib in biomarker-defined populations of sqNSCLC. Patients with NFE2L2 (the name for gene encoding the protein called NRF2)-mutated or wild-type sqNSCLC…
- Conditions
- Non-Small Cell Lung Cancer, Squamous Non-small-cell Lung Cancer, Squamous Non-Small Cell Neoplasm of Lung, NFE2L2 Gene Mutation
- Trial name
- NCT05275673
- Testing
- sapanisertib
- Where
- 14 sites in United States
- Volunteers
- 7 people
- Sponsor
- Faeth Therapeutics
Factor V Leiden, Prothrombin G20210A
Engagement Study for Participants With Factor V Leiden and Prothrombin G20210A Mutations
This study will enrol individuals who have, or may have, Factor V Leiden or Prothrombin G20210A mutations, which are genetic changes linked to an increased risk of blood clots.
- Trial name
- NCT07584265
- Where
- United Kingdom
- Volunteers
- 100 people
- Sponsor
- Richmond Research Institute
Advanced Solid Tumor
QTX3046 in Patients With KRAS G12D Mutations
Phase 1 study to determine the safety and tolerability of QTX3046 as a single agent or in combination with cetuximab.
- Trial name
- NCT06428500
- Testing
- QTX3046, Cetuximab
- Where
- 5 sites in United States
- Volunteers
- 240 people
- Sponsor
- Quanta Therapeutics