Clinical trials: Genetic Mutation
Carcinoma, Non-Small-Cell Lung, ErbB Receptors
Afatinib Treatment for Patients With EGFR Mutation Positive NSCLC Who Are Age 70 or Older
Continuous treatment until progression or occurence of intolerable Adverse Event (AE) or end of trial. The end of trial is one year after the last patient has entered the study.
- Trial name
- NCT02514174
- Testing
- Afatinib
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Boehringer Ingelheim
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Trametinib as a Potential Targeted Treatment in Cancers With NF1 Genetic Changes (MATCH-Subprotocol S1)
This phase II MATCH treatment trial identifies the effects of trametinib in patients whose cancer has a has a genetic change called NF1 mutation.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439318
- Testing
- Trametinib Dimethyl Sulfoxide
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Cancer Institute (NCI)
Cystic Fibrosis
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
- Trial name
- KONTINUE (NCT01707290)
- Testing
- Ivacaftor
- Where
- 38 sites in United States, Belgium and 2 more
- Volunteers
- 125 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Carcinoma, Non-Small-Cell Lung
Afatinib as Second-line Therapy for Lung Cancer With Epidermal Growth Factor Receptor (EGFR) Mutation
The objectives of this single-arm, open-label trial are to assess the efficacy and safety of afatinib as second line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harbouring a…
- Trial name
- NCT02208843
- Testing
- Afatinib
- Where
- 21 sites in Egypt, Malaysia and 5 more
- Volunteers
- 60 people
- Sponsor
- Boehringer Ingelheim
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics
Carcinoma, Non-Small-Cell Lung
Study of Oral cMET Inhibitor INC280 in Patients With EGFR Wild-type (wt), Advanced Non-small Cell Lung Cancer (NSCLC) (Geometry Mono-1)
Study to evaluate the efficacy and safety of capmatinib as a single-agent treatment for subjects with advanced/metastatic (stage IIIB or IV) non-small cell lung cancer (NSCLC) who had wild-type epidermal growth factor receptor…
- Trial name
- NCT02414139
- Testing
- Capmatinib
- Where
- 95 sites in United States, Argentina and 18 more
- Volunteers
- 373 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer, Medullary Thyroid Cancer
A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)
This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants…
- Conditions
- Non-Small Cell Lung Cancer, Medullary Thyroid Cancer, Colon Cancer, Any Solid Tumor
- Trial name
- LIBRETTO-001 (NCT03157128)
- Testing
- LOXO-292
- Where
- 85 sites in United States, Australia and 14 more
- Volunteers
- 857 people
- Sponsor
- Eli Lilly and Company
Carcinoma, Non-small Cell Lung
Trial of Dasatinib in Patients With Advanced Cancers Harboring DDR2 Mutation or Inactivating B-RAF Mutation
The purpose of this study is to establish whether patients with malignancy harboring a discoidin domain receptor 2 mutation or an inactivating B-RAF mutation will respond to dasatinib.
- Trial name
- NCT01514864
- Testing
- Dasatinib
- Where
- 24 sites in United States, Brazil and 5 more
- Volunteers
- 19 people
- Sponsor
- Bristol-Myers Squibb
Carcinoma, Non-Small-Cell Lung, Adenocarcinoma
BIBW 2992 (Afatinib) vs Gemcitabine-cisplatin in 1st Line Non-small Cell Lung Cancer (NSCLC)
To investigate the efficacy and safety of BIBW 2992 compared to standard first-line chemotherapy in patients with stage IIIB or IV adenocarcinoma of the lung harbouring an EGFR activating mutation
- Trial name
- NCT01121393
- Testing
- Gemcitabine+Cisplatin, BIBW 2992
- Where
- 36 sites in China, South Korea and 1 more
- Volunteers
- 364 people
- Sponsor
- Boehringer Ingelheim
Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
- Trial name
- HAUSER-OLE (NCT02624869)
- Testing
- Evolocumab
- Where
- 46 sites in United States, Australia and 21 more
- Volunteers
- 163 people
- Sponsor
- Amgen
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Cystic Fibrosis
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance…
- Trial name
- NCT02070744
- Testing
- VX-661, Ivacaftor
- Where
- 23 sites in United States
- Volunteers
- 40 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Trametinib as a Potential Targeted Treatment in Cancers With BRAF Genetic Changes (MATCH-Subprotocol R)
This phase II MATCH treatment trial identifies the effects of trametinib in patients with cancer having genetic changes called BRAF mutations and fusions.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439279
- Testing
- Trametinib Dimethyl Sulfoxide
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Cancers
A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency
This research study is studying a checkpoint kinase 1 (CHK1) inhibitor as a possible treatment for advanced solid tumors that harbor genetic alterations in the homologous repair (HR) pathway, genetic alterations that indicate…
- Trial name
- NCT02873975
- Testing
- LY2606368
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Dana-Farber Cancer Institute
BRCA1 Mutation Carrier, BRCA2 Mutation Carrier
Veliparib in Treating Patients With Persistent or Recurrent Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
This phase II trial studies how well veliparib works in treating patients with epithelial ovarian, fallopian tube, or primary peritoneal cancer that has come back or does not respond to treatment.
- Conditions
- BRCA1 Mutation Carrier, BRCA2 Mutation Carrier, Ovarian Epithelial Tumor, Recurrent Fallopian Tube Carcinoma
- Trial name
- NCT01540565
- Testing
- Laboratory Biomarker Analysis, Veliparib
- Where
- 153 sites in United States
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
Medullary Thyroid Cancer, Infantile Myofibromatosis
A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.
- Conditions
- Medullary Thyroid Cancer, Infantile Myofibromatosis, Infantile Fibrosarcoma, Papillary Thyroid Cancer
- Trial name
- LIBRETTO-121 (NCT03899792)
- Testing
- Selpercatinib
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 36 people
- Sponsor
- Eli Lilly and Company
Advanced Malignant Solid Neoplasm, Cholangiocarcinoma
Infigratinib for the Treatment of Advanced or Metastatic Solid Tumors in Patients With FGFR Gene Mutations
This phase II trial studies how well infigratinib works in treating solid tumors that have spread to other places in the body (advanced or metastatic) in patients with FGFR gene mutations such as FGFR1-3 gene fusions or other…
- Conditions
- Advanced Malignant Solid Neoplasm, Cholangiocarcinoma, Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm
- Trial name
- NCT04233567
- Testing
- Infigratinib
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Sameek Roychowdhury
Schizophrenia, Psychotic Disorders
Acute Glycine Pharmacodynamic Study
The purpose of this study is to use proton magnetic resonance spectroscopy (MRS) at 4 Tesla to measure brain glycine levels noninvasively at baseline and for 2 hours after a single oral dose of a concentrated glycine-containing…
- Trial name
- NCT01610011
- Testing
- Glycine administration
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Mclean Hospital
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA).
- Trial name
- REALM-DCM (NCT03439514)
- Testing
- ARRY-371797 (PF-07265803)
- Where
- 107 sites in United States, Argentina and 8 more
- Volunteers
- 77 people
- Sponsor
- Pfizer