Clinical trials: Mutation Abnormality
BRAF V600E Mutation Positive, Metastatic Colorectal Cancer
A Retrospective Non Interventional Study on First Line Treatment for Patients With BRAFV600E Mutant Metastatic Colorectal Cancer (mCRC)
The presence of a BRAFV600E mutation is considered a marker of poor prognosis in patients with mCRC, and findings from clinical trials have largely remained inconclusive regarding the efficacy of first line treatments for…
- Trial name
- CAPSTAN CRC (NCT04317599)
- Testing
- Non interventional
- Where
- 34 sites in Austria, Belgium and 5 more
- Volunteers
- 274 people
- Sponsor
- Pierre Fabre Medicament
Cystic Fibrosis
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance…
- Trial name
- NCT00457821
- Testing
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg
- Where
- 15 sites in United States, Canada and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Malignant Melanoma
A Study of Vemurafenib (Zelboraf) in Chinese Participants With BRAF V600 Mutation-Positive Unresectable or Metastatic Melanoma
This open-label, multicenter study will evaluate the pharmacokinetics, safety and efficacy of vemurafenib in Chinese participants with BRAF V600 mutation-positive unresectable or metastatic melanoma.
- Trial name
- NCT01910181
- Testing
- Vemurafenib
- Where
- 2 sites in China
- Volunteers
- 46 people
- Sponsor
- Hoffmann-La Roche
Cancer
Study of Selective BRAF Kinase Inhibitor Dabrafenib Monotherapy Twice Daily and in Combination With Dabrafenib Twice Daily and Trametinib Once Daily in Combination Therapy in Subjects With BRAF V600E Mutation Positive Metastatic (Stage IV) Non-small Cell Lung Cancer.
This was a Phase II, multicenter, non-randomized, open-label study to assess the efficacy, safety, and tolerability of dabrafenib administered as a single agent and in combination with trametinib in stage IV disease to subjects…
- Trial name
- NCT01336634
- Testing
- Dabrafenib, Trametinib
- Where
- 50 sites in United States, France and 9 more
- Volunteers
- 177 people
- Sponsor
- Novartis Pharmaceuticals
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia in Remission
Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic Malignancies
This pilot clinical trial studies the side effects of irradiated donor cells following stem cell transplant in controlling cancer in patients with hematologic malignancies.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia in Remission, Hematopoietic Cell Transplantation Recipient, JAK2 Gene Mutation
- Trial name
- NCT03272633
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Irradiated Allogeneic Cells
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rutgers, The State University of New Jersey
Pro-opiomelanocortin (POMC) Deficiency Obesity
Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type…
- Trial name
- NCT02896192
- Testing
- Setmelanotide
- Where
- 7 sites in United States, Belgium and 5 more
- Volunteers
- 15 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Cystic Fibrosis
PTC Study to Evaluate Ataluren in Combination With Ivacaftor
The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with nonsense mutation cystic fibrosis
- Trial name
- NCT03256968
- Testing
- Ataluren
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Cystic Fibrosis (CF)
: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva.
- Trial name
- NCT03445793
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Jewish Health
Non-Small Cell Lung Cancer
A Study of Osimertinib With or Without Chemotherapy Versus Chemotherapy Alone as Neoadjuvant Therapy for Patients With EGFRm Positive Resectable Non-Small Cell Lung Cancer
This is a Phase III, randomised, controlled, 3-arm, multi-centre study of neoadjuvant osimertinib as monotherapy or in combination with chemotherapy, versus SoC chemotherapy alone, for the treatment of patients with resectable…
- Trial name
- NeoADAURA (NCT04351555)
- Testing
- Osimertinib, Cisplatin, Carboplatin
- Where
- 158 sites in United States, Austria and 23 more
- Volunteers
- 358 people
- Sponsor
- AstraZeneca
Schizophrenia, Bipolar Disorder
Targeting a Genetic Mutation in Glycine Metabolism With D-cycloserine
The purpose of this study is to assess the efficacy of d-cycloserine (DCS) as an augmentation strategy in two psychotic patients with a triplication (4 copies) of the glycine decarboxylase (GLDC) gene.
- Trial name
- DCS (NCT02304432)
- Testing
- D-cycloserine
- Volunteers
- 2 people
- Sponsor
- Mclean Hospital
Lung Cancer
Iressa Re-Challenge in Advanced NSCLC EGFR M+ Patients Who Responded to Gefitinib USed as 1st Line or Previous Treatment
the primary objective is to characterise the impact of gefitinib on the Response Evaluation Criteria in Solid Tumours (RECIST) based assessments; objective response rate (ORR ; confirmed complete response(CR) or partial response…
- Trial name
- ICARUS (NCT01530334)
- Testing
- Gefitinib 250mg
- Where
- 24 sites in Italy
- Volunteers
- 61 people
- Sponsor
- AstraZeneca
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.
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
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
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.
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