Clinical trials: Gene Mutation
Non Small Cell Lung Cancer, Medullary Thyroid Cancer
Study of TPX-0046, A RET/SRC Inhibitor in Adult Subjects With Advanced Solid Tumors Harboring RET Fusions or Mutations
A phase 1/2, first-in-human, open-label study to determine the safety, tolerability, PK, and preliminary efficacy of the novel RET/SRC inhibitor TPX-0046 in adult subjects with advanced or metastatic solid tumors harboring RET…
- Conditions
- Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET Gene Mutation, Metastatic Solid Tumor
- Trial name
- NCT04161391
- Testing
- TPX-0046
- Where
- 21 sites in United States, South Korea
- Volunteers
- 41 people
- Sponsor
- Turning Point Therapeutics, Inc.
Intraoperative Staple Line Bleeding as Measured by the Provided Bleeding Severity Scale
Endostapler Hemostasis Study
The goal of this prospective, multi-center, post-market study is to measure AEON™ Endostapler performance with the EASY/THICK MODE feature for laparoscopic bariatric surgery against the Echelon Flex™ Powered Stapler system from…
- Trial name
- NCT04149925
- Testing
- AEON Endostapler, Echelon Flex Powered Stapler
- Where
- 3 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Lexington Medical Inc.
Cystic Fibrosis
A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)
- Trial name
- NCT03525548
- Testing
- VX-445/TEZ/IVA, TEZ/IVA, IVA
- Where
- 44 sites in United States, Belgium and 2 more
- Volunteers
- 113 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
C3 Glomerulonephritis, C3 Glomerulopathy
A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471
The primary purpose of this study was to evaluate the efficacy of 12 months of oral ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based on histologic scoring and proteinuria.
- Conditions
- C3 Glomerulonephritis, C3 Glomerulopathy, Immune Complex Membranoproliferative Glomerulonephritis, IC-MPGN
- Trial name
- NCT03459443
- Testing
- Danicopan
- Where
- 13 sites in United States, Australia and 3 more
- Volunteers
- 22 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Advanced or Metastatic Solid Tumors, FGFR Mutations
Efficacy and Safety of Pemigatinib in Participants With Solid Tumors With FGFR Mutations or Translocations (FIGHT-208)
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 tumors harboring activating FGFR mutations or…
- Conditions
- Advanced or Metastatic Solid Tumors, FGFR Mutations, FGFR Translocations
- Trial name
- NCT04003623
- Testing
- Pemigatinib
- Where
- 11 sites in United States
- Volunteers
- 1 people
- Sponsor
- Incyte Corporation
FGFR Gene Amplification, FGFR1 Gene Amplification
BGJ398 in Treating Patients With FGFR Positive Recurrent Head and Neck Cancer
This phase IIa trial studies how well the experimental drug, BGJ398 (infigratinib), works in treating patients with fibroblast growth factor receptor (FGFR) 1-3 translocated, mutated, or amplified head and neck cancer that has…
- Conditions
- FGFR Gene Amplification, FGFR1 Gene Amplification, FGFR2 Gene Amplification, FGFR2 Gene Mutation
- Trial name
- NCT02706691
- Testing
- BGJ398
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Chicago
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance…
- Trial name
- NCT02516410
- Testing
- VX-661 plus ivacaftor combination, Ivacaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 168 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Primary Hyperparathyroidism
Evaluating Alternative Medical Therapies in Primary Hyperparathyroidism
This study will evaluate whether blocking the mineralocorticoid receptor, alone, or in combination with the calcimimetic cinacalcet, can lower parathyroid hormone and calcium levels in primary hyperparathyroidism.
- Trial name
- NCT02525796
- Testing
- eplerenone, amiloride, Cinacalcet
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Brigham and Women's Hospital
Cystic Fibrosis
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00458341
- Testing
- Ataluren
- Where
- 3 sites in Belgium, France
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
NLRC4-MAS, XIAP Deficiency
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, leucine-rich repeat and…
- Trial name
- NCT03113760
- Testing
- Tadekinig alfa, 0.9% sodium chloride
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- AB2 Bio Ltd.
Opioid Dependency
Transdermal Electroacupuncture for Opioid Detoxification
This single-blind, randomized clinical trial tests whether electroacupuncture, provided as an adjunctive treatment, improves outcomes among patients receiving inpatient opioid detoxification from opioids.
- Trial name
- NCT00742170
- Testing
- Electroacupuncture
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Mclean Hospital
Cystic Fibrosis (CF)
Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
- Trial name
- NCT04853368
- Testing
- ABBV-576, Galicaftor, Navocaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- AbbVie
Non-Small Cell Lung Cancer
A Study of Osimertinib With or Without Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor Non-Small Cell Lung Cancer (FLAURA2)
The reason for the study is to find out if an experimental combination of an oral medication called osimertinib (TAGRISSO®) when used in combination with chemotherapy is more effective than giving osimertinib alone for the…
- Trial name
- FLAURA2 (NCT04035486)
- Testing
- Osimertinib, Pemetrexed/Carboplatin, Pemetrexed/Cisplatin
- Where
- 153 sites in United States, Argentina and 19 more
- Volunteers
- 587 people
- Sponsor
- AstraZeneca