Clinical trials: Gene Mutations
Acute Myeloid Leukemia, Leukemia
A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 Status
Patients received oral AC220 daily for 14 days to study the side effects, tolerability and best dose for treating relapsed or refractory acute myeloid leukemia, regardless of FLT3 status.
- Conditions
- Acute Myeloid Leukemia, Leukemia, Myelodysplastic Syndrome, AML
- Trial name
- NCT00462761
- Testing
- AC220
- Where
- 5 sites in United States, Georgia
- Volunteers
- 76 people
- Sponsor
- Daiichi Sankyo
Mutations in Exons 18 to 21
Pathomolecular Analysis of Rare EGFR Mutations in Advanced NSCLC
While current mutational analyses comprise exons 19 and 21 in which the majority of EGFR-mutations occur, this study aims at investigating the relevance of exon 18 and 20 mutations.
- Trial name
- NCT01215474
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- Provitro GmbH
Primary Ciliary Dyskinesia
A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD
This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
- Trial name
- NCT06600425
- Testing
- RCT1100
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- ReCode Therapeutics
Pancreas Cancer
Safety and Efficacy of the Therapeutic Vaccine GI-4000 in Combination With Gemcitabine Versus Placebo for the Treatment of Non-metastatic, Post-resection Pancreas Cancer
The GI-4000 therapeutic vaccine or placebo will be injected under the skin of post-resection, non-metastatic pancreas cancer patients.
- Trial name
- NCT00300950
- Testing
- GI-4000, Gemcitabine
- Where
- 28 sites in United States, India
- Volunteers
- 176 people
- Sponsor
- GlobeImmune
Non-small Cell Lung Cancer
An Open Study of ASP8273 in Patients With Non-Small-Cell Lung Cancer (NSCLC) Who Have Epidermal Growth Factor Receptor (EGFR) Mutations
Purpose of the study is to determine the following in patients with non-small cell lung cancer (NSCLC) harboring EGFR activating mutations. the safety and tolerability of ASP8273. the pharmacokinetics (PK) of ASP8273.
- Trial name
- NCT02192697
- Testing
- ASP8273
- Where
- 14 sites in Japan, South Korea and 1 more
- Volunteers
- 124 people
- Sponsor
- Astellas Pharma Inc
Carcinoma, Non-Small-Cell Lung
Study of Capmatinib and Spartalizumab/Placebo in Advanced NSCLC Patients With MET Exon 14 Skipping Mutations
A double-blind, placebo controlled, randomized, phase II study evaluating the efficacy and safety of capmatinib (INC280) and spartalizumab (PDR001) combination therapy versus capmatinib and placebo as first line treatment for…
- Trial name
- NCT04323436
- Testing
- Spartalizumab, Capmatinib
- Where
- 15 sites in United States, Belgium and 7 more
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer
Phase II Single Arm Study of AZD9291 to Treat NSCLC Patients in Asia Pacific
A Phase II, Open Label, Single-arm Study to Assess the Safety and Efficacy of AZD9291 in Asia Pacific Patients with Locally Advanced/Metastatic Non-Small Cell Lung Cancer whose Disease has Progressed with Previous Epidermal…
- Trial name
- AURA17 (NCT02442349)
- Testing
- AZD9291
- Where
- 30 sites in Australia, China and 1 more
- Volunteers
- 171 people
- Sponsor
- AstraZeneca
AML, MDS
A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 and/or Ras Mutations
This is a Phase 1/2a dose-escalation study of E6201, a dual mitogen-activated protein kinase/extracellular-signal regulated kinase 1 (MEK1) and FMS-like tyrosine kinase 3 (FLT3) inhibitor, in subjects with advanced hematologic…
- Conditions
- AML, MDS, CMML
- Trial name
- NCT02418000
- Testing
- E6201
- Where
- 4 sites in United States
- Volunteers
- 27 people
- Sponsor
- Spirita Oncology, LLC
Romano-Ward Syndrome, Long QT Syndrome
Evaluation of the Effects of KCNQ1 Mutation on Insulin Tolerance and Obsessive Compulsive Features in Long QT Romano-Ward Syndrome Patients.
The objectives of the study are to investigate if KCNQ1 mutation in Romano-Ward long QT patients can be associated with changes in insulin regulation and with psychological features of compulsivity, impulsivity and behavioural…
- Conditions
- Romano-Ward Syndrome, Long QT Syndrome, Compulsive Behavior, Healthy Individuals
- Trial name
- PRIME (NCT04715256)
- Testing
- Cognitive assessment of obsessive-compulsive and impulsive behaviours, Genomic analysis, Glucoregulation assessment
- Where
- 2 sites in France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Biotrial
Locally Advanced or Metastatic Non-Small Cell Lung Cancer, EGFR Exon20 Insertion Mutations
Real-World Effectiveness of Osimertinib for Patients With Non-Small Cell Lung Cancer
This study is a retrospective real-world study to evaluate the efficacy of Osimertinib for patients with advanced or metastatic EGFR 20exon insertion mutation (20ins) Non-Small Cell Lung Cancer.
- Trial name
- NCT05513664
- Testing
- Osimertinib
- Where
- 24 sites in China
- Volunteers
- 80 people
- Sponsor
- Shanghai JMT-Bio Inc.
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
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.
Primary Ciliary Dyskinesia
Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects
This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.
- Trial name
- NCT05737485
- Testing
- RCT1100
- Where
- 4 sites in United States, Australia and 2 more
- Volunteers
- 9 people
- Sponsor
- ReCode Therapeutics
HER2 Gene Mutation
IAH0968 in Combination With GC for the Treatment of HER2-Positive Unresectable Advanced/Metastatic Malignant Tumors
The study aims to evaluate the efficacy and safety of IAH0968 in combination with gemcitabine and cisplatin for the treatment of HER2-positive unresectable advanced/metastatic malignant tumors and cholangiocarcinoma.
- Trial name
- NCT05991518
- Testing
- Injection of IAH0968+Gemcitabine+Cisplatin, Gemcitabine+Cisplatin
- Where
- China
- Volunteers
- 136 people
- Sponsor
- SUNHO（China）BioPharmaceutical CO., Ltd.
ALL, Adult, AML, Adult
A Study of BN104 in the Treatment of Acute Leukemia
The Phase I/II trial is to learn the safety, pharmacokinetics, and preliminary efficacy of BN104 taken once daily or twice daily in patients with acute lymphocytic leukemia or acute myeloblastic leukemia.
- Conditions
- ALL, Adult, AML, Adult, Acute Leukaemia, KMT2A Rearrangement
- Trial name
- NCT06052813
- Testing
- BN104 monotherapy, BN104 monotherapy - rp2d
- Where
- China
- Volunteers
- 66 people
- Sponsor
- Institut de Recherches Internationales Servier (I.R.I.S.)
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.
Germline BRCA1/2 Mutations and, Metastatic Adenocarcinoma of the Pancreas
Olaparib in gBRCA Mutated Pancreatic Cancer Whose Disease Has Not Progressed on First Line Platinum-Based Chemotherapy
A Phase III, Randomised, Double Blind, Placebo Controlled, Multicentre Study of Maintenance Olaparib Monotherapy in Patients with gBRCA Mutated Metastatic Pancreatic Cancer whose Disease Has Not Progressed on First Line Platinum…
- Trial name
- POLO (NCT02184195)
- Testing
- Olaparib
- Where
- 103 sites in United States, Australia and 10 more
- Volunteers
- 154 people
- Sponsor
- AstraZeneca
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
Cystic Fibrosis
Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR…
- Trial name
- NCT01946412
- Testing
- Ivacaftor
- Where
- 16 sites in United States, Canada and 1 more
- Volunteers
- 33 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Solid Tumor
A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes
A Phase 2, open-label, single-arm trial to evaluate the response of rucaparib in participants with various solid tumors and with deleterious mutations in Homologous Recombination Repair (HRR) genes.
- Trial name
- LODESTAR (NCT04171700)
- Testing
- Rucaparib
- Where
- 19 sites in United States
- Volunteers
- 83 people
- Sponsor
- pharmaand GmbH