Clinical trials: Gene Mutations
Carcinoma, Non-Small-Cell Lung
A Study of LY2875358 in Participants With Non-Small Cell Lung Cancer With Activating Epidermal Growth Factor Receptor Mutations
The primary purpose of this study is to compare the efficacy of the study drug LY2875358, given together with erlotinib, against erlotinib, alone.
- Trial name
- Balise (NCT01897480)
- Testing
- LY2875358, Erlotinib
- Where
- 45 sites in Denmark, France and 7 more
- Volunteers
- 168 people
- Sponsor
- Eli Lilly and Company
Transthyretin Gene Mutations, Transthyretin Amyloidosis
Transthyretin Amyloidosis Outcome Survey (THAOS)
THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
- Trial name
- THAOS (NCT00628745)
- Testing
- None. Observational Study.
- Where
- 113 sites in United States, Argentina and 23 more
- Volunteers
- 6,718 people
- Sponsor
- Pfizer
Colorectal Cancer
Panitumumab and RAS, Diagnostically-useful Gene Mutation for mCRC
The purpose of this study is to verify the efficacy of mFOLFOX6 + panitumumab combination therapy and mFOLFOX6 + bevacizumab combination therapy in first-line treatment of chemotherapy-naive patients with KRAS/NRAS wild-type…
- Trial name
- PARADIGM (NCT02394795)
- Testing
- oxaliplatin (OXA), levofolinate calcium (l-LV), 5-FU, panitumumab, oxaliplatin (OXA), levofolinate calcium (l-LV), 5-FU, bevacizumab
- Where
- 155 sites in Japan
- Volunteers
- 823 people
- Sponsor
- Takeda
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.
BRAF or NRAS Mutant Metastatic Melanoma
A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma
The study will assess the safety and efficacy of single-agent MEK162 in adult patients with locally advanced and unresectable or metastatic malignant cutaneous melanoma, harboring BRAFV600E or NRAS mutations.
- Trial name
- NCT01320085
- Testing
- MEK162
- Where
- 25 sites in United States, Germany and 3 more
- Volunteers
- 183 people
- Sponsor
- Pfizer
Solid Tumor Malignancy
Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR…
- Trial name
- NCT03822117
- Testing
- Pemigatinib
- Where
- 90 sites in United States, Denmark and 9 more
- Volunteers
- 111 people
- Sponsor
- Incyte Corporation
Non-Small Cell Lung Cancer, NSCLC
First-In-Human Study of STX-721/PFL-721 in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring EGFR or HER2 Exon 20 Insertion Mutations
Study STX-721-101/PFL-721CI101 is an open label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetic (PK) exposure, and preliminary antitumor activity of STX-721/PFL-721 in participants with non-small cell lung…
- Conditions
- Non-Small Cell Lung Cancer, NSCLC, EGFR/HER2 Exon 20 Insertion Mutation
- Trial name
- NCT06043817
- Testing
- STX-721/PFL-721 (Escalated), STX-721/PFL-721 (3 dose levels), STX-721/PFL-721 (RP2D)
- Where
- 32 sites in United States, Australia and 6 more
- Volunteers
- 345 people
- Sponsor
- Pierre Fabre Medicament
NALP3 Mutation
Safety, Efficacy, Pharmacokinetics, Pharmacodynamics of ACZ885 in Patients With NALP3 Mutations and Clinical Symptoms
This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are…
- Trial name
- NCT00487708
- Testing
- canakinumab
- Where
- 10 sites in France, Germany and 3 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
- Trial name
- NCT02971839
- Testing
- VX-561, IVA
- Where
- 14 sites in United States
- Volunteers
- 11 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non-small Cell Lung Cancer With EGFR-Activating Mutations
ARCHER1050: A Study of Dacomitinib vs. Gefitinib in 1st-Line Treatment Of Advanced NSCLC.
This is a multinational, multicenter, randomized, open-label, Phase 3 study comparing the efficacy and safety of treatment with dacomitinib (PF-00299804) to treatment with gefitinib in patients with locally advanced or metastatic…
- Trial name
- NCT01774721
- Testing
- Dacomitinib (PF-00299804), Gefitinib
- Where
- 52 sites in China, Hong Kong and 5 more
- Volunteers
- 452 people
- Sponsor
- Pfizer
NUT Midline Carcinoma, Triple Negative Breast Cancer
A Dose-Finding Study of Birabresib (MK-8628), a Small Molecule Inhibitor of the Bromodomain and Extra-Terminal (BET) Proteins, in Adults With Selected Advanced Solid Tumors (MK-8628-003)
Open-label, phase I, non-randomized, multicentric study of single-agent birabresib (MK-8628) (formerly known as OTX015) administered according to two distinct regimens to participants with selected advanced tumors.
- Conditions
- NUT Midline Carcinoma, Triple Negative Breast Cancer, Non-small Cell Lung Cancer With Rearranged ALK Gene/Fusion Protein or KRAS Mutation, Castrate-resistant Prostate Cancer
- Trial name
- NCT02259114
- Testing
- Birabresib
- Volunteers
- 47 people
- Sponsor
- Oncoethix GmbH, a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Non-small Cell Lung Cancer (NSCLC)
A Study to Learn About the Tests Looking for a Gene Mutation in Adults With Lung Cancer in China (ELEGANT)
The main aim of this study is to examine the percentage of people with lung cancer who carry a certain gene mutation (epidermal growth factor receptor exon 20 insertions - EGFR ex20ins) and their frequency in Chinese participants…
- Trial name
- ELEGANT (NCT05737849)
- Testing
- No Intervention
- Where
- China
- Sponsor
- Takeda
Advanced Non-Small Cell Lung Cancer With MET Mutations
CNS Dose Escalation/Expansion of Tepotinib in MET-driven NSCLC
This study will look at how effective the study drug (tepotinib) is at stopping the growth and spread of lung cancer with central nervous system metastasis.
- Trial name
- NCT04739358
- Testing
- Tepotinib
- Where
- 5 sites in United States
- Volunteers
- 2 people
- Sponsor
- Criterium, Inc.
Advanced Solid Tumors Harboring RAS Mutations or Amplifications
A Study of HRS-2329 in Participants With Advanced Solid Tumors Harboring RAS Mutations or Amplifications
This is an open-label, multi-center phase I clinical study to evaluate the safety, tolerability, and pharmacokinetics of HRS-2329 in participants with advanced solid tumors harboring RAS mutations or amplifications.
- Trial name
- NCT07189949
- Testing
- HRS-2329 Tablet
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Non-Squamous Non-Small Cell Lung Cancer
A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor Mutations
This single arm, open-label study will evaluate the efficacy and safety of erlotinib (Tarceva) in participants with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR)…
- Trial name
- NCT01260181
- Testing
- Erlotinib
- Where
- 9 sites in Portugal
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Breast Cancer Metastatic, BRCA 1 Gene Mutation
Assessment of the Efficacy and Safety of Olaparib Monotherapy Versus Physicians Choice Chemotherapy in the Treatment of Metastatic Breast Cancer Patients With Germline BRCA1/2 Mutations.
This open label, randomised, controlled, multi-centre phase III study will assess the efficacy and safety of single agent olaparib vs standard of care based on physician's choice of capecitabine, vinorelbine or eribulin in…
- Conditions
- Breast Cancer Metastatic, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation
- Trial name
- OlympiAD (NCT02000622)
- Testing
- Olaparib, Physician's choice chemotherapy
- Where
- 171 sites in United States, Bulgaria and 17 more
- Volunteers
- 302 people
- Sponsor
- AstraZeneca
Locally Advanced or Metastatic Non-Small Cell Lung Cancer, EGFR Exon20 Insertion Mutations
Efficacy and Safety of JMT101 Combined With Osimertinib in Patients With Non-Small Cell Lung Cancer
This study is a phase II, open label, multi-center study to evaluate the efficacy and safety of JMT101 combined with Osimertinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR…
- Trial name
- NCT05132777
- Testing
- JMT101, Osimertinib Mesylate Tablets
- Volunteers
- 155 people
- Sponsor
- Shanghai JMT-Bio Inc.
Locally Advanced or Metastatic EGFR Sensitising Mutation Positive Non-small Cell Lung Cancer
Alflutinib Mesylate Versus Gefitinib in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer （FLAG）
To assess the efficacy and safety of Alflutinib Mesylate versus Gefitinib in patients with locally advanced or Metastatic Non Small Cell Lung Cancer
- Trial name
- NCT03787992
- Testing
- Gefitinib 250 mg
- Where
- China
- Volunteers
- 358 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Acute Myeloid Leukemia With Gene Mutations
Expanded Treatment Protocol for Adults With FLT3-ITD Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) to Receive Quizartinib
An expanded access program (EAP): Allows doctors to give medicine to patients, Before it is approved by health authorities. This EAP is for: Quizartinib Patients with FLT3-ITD mutated AML, AML that has come back, or Is resistant…
- Trial name
- NCT03746912
- Testing
- Quizartinib Dihydrochloride
- Sponsor
- Daiichi Sankyo
POMC Deficiency Obesity, PCSK1 Deficiency Obesity
An Observational,Prospective Natural History Study of Early-Onset Extreme Obesity Due to Bi-Allelic Loss-of-Function Mutations in the POMC, PCSK1 or LEPR Genes
This is an observational study. There are no protocol-defined visits, although patients are expected to have routine office visits approximately every 6 months.
- Conditions
- POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NHS (NCT03621007)
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.