Clinical trials: Genetic Mutation
Melanoma
Safety and Efficacy in Participants With Metastatic BRAF-mutant Melanoma Treated With Encorafenib With and Without Binimetinib in Combination With Nivolumab and Low-dose Ipilimuma
Patients with unresectable or metastatic BRAF-mutant melanoma high-risk patients will be given 450 mg orally (PO) daily (QD) plus binimetinib 45 mg PO twice daily (BID) together with nivolumab administered intravenously (IV) at…
- Trial name
- QUAD01 (NCT04655157)
- Testing
- encorafenib, nivolumab, ipilimumab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Jason J. Luke, MD
Melanoma
Vemurafenib (R05185426) in Poor Performance Status Patients With Unresectable Locally Advanced or Metastatic Melanoma Harboring a V600E/K Mutation
The purpose of this study is to find out what effects, good and/or bad, vemurafenib has on the patient and the melanoma. Specifically, the investigators want to know how well vemurafenib shrinks melanoma.
- Trial name
- NCT01474551
- Testing
- vemurafenib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Alzheimer's Disease
A Study of Crenezumab Versus Placebo in Preclinical Presenilin1 (PSEN1) E280A Mutation Carriers to Evaluate Efficacy and Safety in the Treatment of Autosomal-Dominant Alzheimer's Disease (AD), Including a Placebo-Treated Non-Carrier Cohort
This study consists of 2 periods: [1] Study Period A - evaluating the efficacy and safety of Crenezumab versus Placebo in participants who carry the PSEN1 E280A autosomal-dominant mutation and do not meet the criteria for mild…
- Trial name
- NCT01998841
- Testing
- Crenezumab
- Where
- 4 sites in Colombia
- Volunteers
- 252 people
- Sponsor
- Genentech, Inc.
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase 3 (FLT3) Mutation / Internal Tandem Duplication (ITD)
A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)
The purpose of the phase 1 portion (dose escalation) of the study was to establish an optimally safe and biologically active recommended phase 2 dose (RP2D) and/or to determine maximum tolerated dose (MTD) for gilteritinib in…
- Trial name
- NCT04240002
- Testing
- gilteritinib, fludarabine, cytarabine
- Where
- 7 sites in United States, Germany and 3 more
- Volunteers
- 9 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Neoplasms
An Extension (Rollover) Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Malignancies Previously Enrolled in an Antecedent Vemurafenib Protocol
This open-label, multicenter, non-randomized study provided continued access to vemurafenib for eligible participants with BRAF V600 mutation-positive malignancy, who were previously enrolled and treated in an antecedent…
- Trial name
- NCT01739764
- Testing
- Vemurafenib
- Where
- 83 sites in United States, Belarus and 23 more
- Volunteers
- 215 people
- Sponsor
- Hoffmann-La Roche
Non-Small Cell Lung Cancer, Squamous Non-small-cell Lung Cancer
A Study of Sapanisertib in Relapsed/Refractory NFE2L2-Mutated and Wild-Type Squamous Non-Small Cell Lung Cancer
This is a multicenter, randomized, open-label Phase 2 study of sapanisertib in biomarker-defined populations of sqNSCLC. Patients with NFE2L2 (the name for gene encoding the protein called NRF2)-mutated or wild-type sqNSCLC…
- Conditions
- Non-Small Cell Lung Cancer, Squamous Non-small-cell Lung Cancer, Squamous Non-Small Cell Neoplasm of Lung, NFE2L2 Gene Mutation
- Trial name
- NCT05275673
- Testing
- sapanisertib
- Where
- 14 sites in United States
- Volunteers
- 7 people
- Sponsor
- Faeth Therapeutics
Genetic Disease, Nonsense Mutation
Phase 1 Study of ELX-02 in Healthy Adult Subjects
Phase 1 Multiple Ascending Dose Study in Normal Healthy Volunteers
- Trial name
- NCT03309605
- Testing
- ELX-02
- Where
- Belgium
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Breast Cancer, Metastatic Breast Cancer
ABT-888 and Temozolomide for Metastatic Breast Cancer and BRCA1/2 Breast Cancer
The purpose of this research study is to find out if the combination of ABT-888 and temozolomide is safe and effective in treating patients with metastatic breast cancer.
- Conditions
- Breast Cancer, Metastatic Breast Cancer, BRCA1 Gene Mutation, brca2 Gene Mutation
- Trial name
- NCT01009788
- Testing
- ABT-888, temozolomide
- Where
- 3 sites in United States
- Volunteers
- 63 people
- Sponsor
- Steven J Isakoff, MD, PhD
Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm
Testing Whether Cancers With Specific Mutations Respond Better to Glutaminase Inhibitor, Telaglenastat Hydrochloride, Anti-Cancer Treatment, BeGIN Study
This phase II trial studies how well glutaminase inhibitor telaglenastat hydrochloride (CB-839 HCl) works in treating patients with specific genetic mutations and solid tumors or malignant peripheral nerve sheath tumors that have…
- Conditions
- Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, NF1 Mutation Positive Malignant Peripheral Nerve Sheath Tumor, Unresectable Malignant Solid Neoplasm
- Trial name
- NCT03872427
- Testing
- Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging
- Where
- 31 sites in United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor
Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well ulixertinib works in treating patients with solid tumors that have spread to other places in the body (advanced), non-Hodgkin lymphoma, or histiocytic disorders that have a…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03698994
- Testing
- Pharmacokinetic Study, Ulixertinib
- Where
- 107 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia (AML)
A Global Study of the Efficacy and Safety of Midostaurin + Chemotherapy in Newly Diagnosed Patients With FLT3 Mutation Negative (FLT3-MN) Acute Myeloid Leukemia (AML)
The purpose of this study was to confirm the preliminary evidence from early clinical trials that midostaurin may provide clinical benefit not only to AML patients with the FLT3-mutations but also in FLT3-MN (SR<0.05) AML (FLT3…
- Trial name
- NCT03512197
- Testing
- Midostaurin, Chemotherapy
- Where
- 133 sites in United States, Argentina and 18 more
- Volunteers
- 511 people
- Sponsor
- Novartis Pharmaceuticals
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
ALK Gene Mutation, BRAF Gene Mutation
Recombinant EphB4-HSA Fusion Protein and Pembrolizumab, MK-3475
This phase IIa trial studies how well recombinant EphB4-HSA fusion protein and pembrolizumab work in treating patients with non-small cell lung cancer that has spread to other places in the body or head and neck squamous cell…
- Conditions
- ALK Gene Mutation, BRAF Gene Mutation, EGFR Gene Mutation, Head and Neck Squamous Cell Carcinoma
- Trial name
- NCT03049618
- Testing
- Laboratory Biomarker Analysis, Pembrolizumab, Recombinant EphB4-HSA Fusion Protein
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Southern California
Myoclonus Epilepsies, Progressive
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK
A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due…
- Trial name
- AUT022201 (NCT05873062)
- Testing
- AUT00201
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Autifony Therapeutics Limited
Carcinoma, Non-Small-Cell Lung
Afatinib (BIBW 2992) in Advanced Non-Small Cell Lung Cancer Patients With EGFR Mutation
Primary objective of the trial is to evaluate the safety of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR (Epidermal growth factor receptor) mutation(s) and have never…
- Trial name
- NCT01953913
- Testing
- Afatinib
- Where
- 33 sites in China, Hong Kong and 3 more
- Volunteers
- 542 people
- Sponsor
- Boehringer Ingelheim