Clinical trials: Genetic Mutation
Cancer
Study to Determine the Effectiveness of GSK1120212 in BRAF Mutation-positive Melanoma Previously Treated With or Without a BRAF Inhibitor
MEK113583 is a Phase II open-label, multi-site study to investigate the objective response rate, safety, and pharmacokinetics of GSK1120212 in subjects with BRAF mutation-positive melanoma who were previously treated with or…
- Trial name
- NCT01037127
- Testing
- GSK1120212
- Where
- 10 sites in United States, Australia
- Volunteers
- 97 people
- Sponsor
- GlaxoSmithKline
Tumors With CDK4/6 Pathway Activation
LEE011 for Patients With CDK4/6 Pathway Activated Tumors (SIGNATURE)
The purpose of this signal seeking study was to determine whether treatment with LEE011 demonstrates sufficient efficacy in CDK4/6 pathway activated solid tumors and/or hematologic malignancies to warrant further study.
- Trial name
- SIGNATURE (NCT02187783)
- Testing
- LEE011
- Where
- 61 sites in United States
- Volunteers
- 106 people
- Sponsor
- Novartis Pharmaceuticals
Diffuse Astrocytoma, Anaplastic Astrocytoma
Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors
The purpose of this study was to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma (LGG) or relapsed or refractory high…
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT02684058
- Testing
- Dabrafenib, trametinib, Carboplatin
- Where
- 57 sites in United States, Argentina and 18 more
- Volunteers
- 151 people
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Cancer
A Study to Evaluate Chemotherapy Plus Osimertinib Against Chemotherapy Plus Placebo in Patients With Non-small Cell Lung Cancer (NSCLC)
The study will evaluate the efficacy and safety of treatment with chemotherapy in combination with osimertinib compared to chemotherapy in combination with placebo in patients whose disease has progressed extracranially following…
- Trial name
- COMPEL (NCT04765059)
- Testing
- Osimertinib (AZD9291) pemetrexed cisplatin or carboplatin
- Where
- 40 sites in United States, China and 4 more
- Volunteers
- 98 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
Trikafta in Cystic Fibrosis Patients
This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta, and who are not expected to be approved for CFTR modulator treatment in the…
- Trial name
- NCT03506061
- Testing
- Trikafta
- Where
- 2 sites in United States
- Volunteers
- 42 people
- Sponsor
- Emory University
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Afatinib as a Potential Targeted Treatment in Cancers With HER2 Genetic Changes (MATCH-Subprotocol B)
This phase II MATCH treatment trial identifies the effects of afatinib in patients whose cancer has genetic changes called HER2 mutations.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439136
- Testing
- Afatinib Dimaleate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Cancer Institute (NCI)
Non-small Cell Lung Cancer
Erlotinib Therapy and Subsequent Development of Mechanisms of Secondary Resistance in Patients With NSCLC
Erlotinib is a drug which targets non small cell lung cancer with a genetic change (mutation) in the epidermal growth factor receptor (EGFR).
- Trial name
- NCT00997334
- Testing
- Erlotinib
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- David M. Jackman, MD
Parkinson, Parkinson Disease
Parkinson Disease and DBS: Cognitive Effects in GBA Mutation Carriers
Every year, approximately 9,000 Parkinson disease (PD) patients undergo deep brain stimulator (DBS) placement into the subthalamic nucleus (STN-DBS).
- Conditions
- Parkinson, Parkinson Disease, Genetic Predisposition, GBA Gene Mutation
- Trial name
- NCT03234478
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Rutgers, The State University of New Jersey
Locally Advanced or Metastatic EGFR T790M+ NSCLC
Study of AZD9291 Plus MEDI4736 Versus AZD9291 Monotherapy in NSCLC After Previous EGFR TKI Therapy in T790M Mutation Positive Tumours
A Phase III, Multi-Centre, Open Label, Randomized Study to Assess the Efficacy and Safety of AZD9291 in Combination with MEDI4736 versus AZD9291 Monotherapy in patients with Locally Advanced or Metastatic Epidermal Growth Factor…
- Trial name
- CAURAL (NCT02454933)
- Testing
- AZD9291, MEDI4736
- Where
- 13 sites in Canada, South Korea and 1 more
- Volunteers
- 29 people
- Sponsor
- AstraZeneca
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Recurrent Platinum Resistant Ovarian Cancer
Efficacy and Safety Study of Cediranib in Combination With Olaparib in Patients With Recurrent Platinum-Resistant Ovarian Cancer
This is an open label, single arm, multi-center study to assess the efficacy and safety of the combination of cediranib and olaparib tablets in platinum-resistant relapsed high grade serous, high grade endometroid or clear cell…
- Trial name
- CONCERTO (NCT02889900)
- Testing
- cediranib and olaparib
- Where
- 25 sites in United States
- Volunteers
- 62 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an…
- Trial name
- NCT02725567
- Testing
- IVA
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Retinal Disease, Retinitis Pigmentosa
Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
This study was to assess the safety of gene transfer via subretinal administration of rAAV2-VMD2-hMERTK in subjects with MERTK-associated retinitis pigmentosa (RP).
- Trial name
- NCT01482195
- Testing
- Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus
- Where
- Saudi Arabia
- Volunteers
- 6 people
- Sponsor
- King Khaled Eye Specialist Hospital
Advanced Cholangiocarcinoma, Metastatic Cholangiocarcinoma
Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy)
Study AG120-C-005 is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study of orally administered AG-120. Participants, all personnel involved in the evaluation of participants' response to treatment (e.g.…
- Trial name
- ClarIDHy (NCT02989857)
- Testing
- AG-120
- Where
- 49 sites in United States, France and 4 more
- Volunteers
- 187 people
- Sponsor
- Institut de Recherches Internationales Servier
Cystic Fibrosis
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
This study will evaluate the efficacy of ivacaftor treatment in subjects with CF 6 years of age and older who have a 3849 + 10KB C→T or D1152H CFTR mutation.
- Trial name
- NCT03068312
- Testing
- Ivacaftor
- Where
- Israel
- Volunteers
- 38 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations
The purpose of this study is to explore the combination of Ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation
- Trial name
- NCT03256799
- Testing
- Ivacaftor/Ataluren
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Cystic Fibrosis
Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis aged 6 to 11…
- Trial name
- NCT01897233
- Testing
- Lumacaftor, Ivacaftor
- Where
- 20 sites in United States, Canada
- Volunteers
- 62 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non Small Cell Lung Cancer
Phase 2 Study of AUY922 in NSCLC Patients With Exon 20 Insertion Mutations in EGFR
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific cancer.
- Trial name
- NCT01854034
- Testing
- AUY922
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Massachusetts General Hospital
Leukemia, Myeloid, Isocitrate Dehydrogenase
An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation
This is an international, multicenter, open-label, randomized, Phase 3 study comparing the efficacy and safety of AG-221 versus conventional care regimens (CCRs) in subjects 60 years or older with acute myeloid leukemia (AML)…
- Trial name
- IDHENTIFY (NCT02577406)
- Testing
- AG-221, BSC, Azacitidine
- Where
- 93 sites in United States, Australia and 16 more
- Volunteers
- 319 people
- Sponsor
- Celgene
Locally Advanced or Metastatic EGFR Sensitising Mutation Positive Non Small Cell Lung Cancer
AZD9291 Versus Gefitinib or Erlotinib in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer
To assess the efficacy and safety of AZD9291 versus a standard of care epidermal growth factor receptor tyrosine kinase inhibitor in patients with locally advanced or Metastatic Non Small Cell Lung Cancer
- Trial name
- FLAURA (NCT02296125)
- Testing
- Erlotinib 150/100 mg, Gefitinib 250 mg
- Where
- 170 sites in United States, Australia and 28 more
- Volunteers
- 674 people
- Sponsor
- AstraZeneca