Clinical trials: Gene Mutations
Solid Tumor
Dose Escalation Study of ARQ 736 in Adult Subjects With Advanced Solid Tumors Harboring BRAF and/or NRAS Mutations
This is an open-label, Phase 1, dose escalation study of oral ARQ 736 administered to subjects with advanced solid tumors harboring the mutation.
- Trial name
- NCT01225536
- Testing
- ARQ 736
- Where
- 4 sites in United States, Italy
- Volunteers
- 24 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Lung Cancer
Laboratory Study Using Samples From Patients With Non-Small Cell Lung Cancer Treated on Clinical Trial CASE-2507
RATIONALE: Studying samples of blood and tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and RNA and identify biomarkers related to cancer.
- Trial name
- NCT00907699
- Testing
- DNA analysis, RNA analysis, fluorescence in situ hybridization
- Sponsor
- Case Comprehensive Cancer Center
Advanced Cancer, Breast Cancer
First-in-Human Study of OKI-219 in Advanced Solid Tumors and Advanced Breast Cancer
OKI-219-101 is a Phase 1a/1b, open-label, multicenter, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of OKI-219 as monotherapy and in combination…
- Conditions
- Advanced Cancer, Breast Cancer, Advanced Solid Tumors, PI3K Gene Mutation
- Trial name
- PIKture-01 (NCT06239467)
- Testing
- OKI-219, Fulvestrant, Trastuzumab
- Where
- 33 sites in United States, Belgium and 4 more
- Volunteers
- 200 people
- Sponsor
- OnKure, Inc.
Non-Small Cell Lung Carcinoma, Advanced Non-Small Cell Squamous Lung Cancer
Whole Body HER3 Quantification With Radiolabelled Patritumab Deruxtecan (HER3-DXd) PET/CT
Activity of patritumab deruxtecan (U3-1402; HER3-DXd) has been shown in a phase I/II study in patients with HER3 expressing breast cancer as well as in a phase I study in patients with EGFR TKI refractory EGFR mutation positive…
- Conditions
- Non-Small Cell Lung Carcinoma, Advanced Non-Small Cell Squamous Lung Cancer, EGFR Gene Mutation
- Trial name
- NCT06222489
- Testing
- 89Zr-Patritumab deruxtecan
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- The Netherlands Cancer Institute
Hemophilia
Genetic Susceptibility to Factor VIII Inhibitors
This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII.
- Trial name
- NCT00344435
- Where
- Sweden
- Volunteers
- 1,187 people
- Sponsor
- National Cancer Institute (NCI)
Diabetes Mellitus
Efficacy and Safety Study of Sulfonylureas in Neonatal Diabetes Mellitus
The aim of our trial is to try to switch patients with permanent neonatal diabetes mellitus due to a Kir6.2 or SUR1 activating mutation from subcutaneous insulin to oral glibenclamide therapy.
- Trial name
- GLIDKIR6-2 (NCT00610038)
- Testing
- glibenclamide
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Lung Cancer, Non-squamous, Non-small Cell
Beamion 44: A Study to Test How Well Zongertinib is Tolerated by People With Advanced Non-small Cell Lung Cancer With HER2 Mutations When Given in Combination With Chemotherapy With or Without Pembrolizumab
This study is open to adults with a type of lung cancer called HER2-mutant non-squamous non-small cell lung cancer (NSCLC) that is advanced or has spread.
- Trial name
- NCT07486817
- Testing
- Zongertinib, Cisplatin, Carboplatin
- Where
- 17 sites in Australia, China and 5 more
- Volunteers
- 60 people
- Sponsor
- Boehringer Ingelheim
Solid Tumor Malignancy
Efficacy and Safety of Pemigatinib in Previously Treated Locally Advanced/Metastatic or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or Translocations (FIGHT-207)
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 tumor malignancies harboring activating FGFR…
- Trial name
- NCT03822117
- Testing
- Pemigatinib
- Where
- 90 sites in United States, Denmark and 9 more
- Volunteers
- 111 people
- Sponsor
- Incyte Corporation
HER2 Positive Breast Neoplasms, Hormone Receptor Positive Breast Neoplasms
A Safety Study of SGN-LIV1A in Breast Cancer Patients
This study will examine the safety and tolerability of ladiratuzumab vedotin (LV) in patients with metastatic breast cancer. LV will be given alone or in combination with trastuzumab.
- Conditions
- HER2 Positive Breast Neoplasms, Hormone Receptor Positive Breast Neoplasms, Triple Negative Breast Neoplasms, HER2 Mutations Breast Neoplasms
- Trial name
- NCT01969643
- Testing
- ladiratuzumab vedotin, Trastuzumab
- Where
- 38 sites in United States
- Volunteers
- 290 people
- Sponsor
- Seagen Inc.
Metastatic Colorectal Cancer
Pemetrexed and Gemcitabine for Treatment Resistant Patients With Metastatic Colorectal Cancer and KRAS Mutations
This study aims to investigate the efficacy and safety of the combination of pemetrexed and gemcitabine in heavily pre-treated, chemotherapy resistant colorectal cancer patients with KRAS mutations.
- Trial name
- PG (NCT01109615)
- Testing
- Pemetrexed, Gemcitabine
- Where
- Denmark
- Volunteers
- 40 people
- Sponsor
- Vejle Hospital
Cerebral Arteriovenous Malformations
Liquid Biopsies for Detecting Somatic Mutations in Sporadic Cerebral Arteriovenous Malformations.
Cerebral arteriovenous malformations (CAVMs) are abnormal vessels located on the surface of the brain or within the cerebral parenchyma, causing abnormal communication between the arterial and venous networks, without the…
- Trial name
- BioMAV2 (NCT07103304)
- Testing
- Search for activating somatic genetic mutations
- Where
- 3 sites in France
- Volunteers
- 16 people
- Sponsor
- University Hospital, Rouen
Subjects With NSCLC With an EGFR Activating Mutation
A Study for Subjects Who Are Participating in an Astellas-sponsored ASP8273 Study
The purpose of the study is to evaluate long-term safety and tolerability of continuous treatment with ASP8273 in subjects who are participating in an Astellas-sponsored ASP8273 study which has completed, at a minimum, the…
- Trial name
- NCT03042013
- Testing
- ASP8273
- Sponsor
- Astellas Pharma Global Development, Inc.
Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm
Evaluating the Effects of Tasimelteon Vs. Placebo in Delayed Sleep-Wake Phase Disorder (DSWPD) and the CRY1Δ11 Variant
This is a double-blind, randomized, two-period crossover study to evaluate the efficacy and safety of a single oral dose of tasimelteon and matching placebo in male and female subjects with DSWPD and the CRY1Δ11 variant.
- Conditions
- Sleep Wake Disorders, Sleep Disorders, Circadian Rhythm, Chronobiology Disorders, Gene Mutations and Other Alterations Nec
- Trial name
- NCT06701396
- Testing
- Tasimelteon
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Vanda Pharmaceuticals
Breast Cancer Risk, Ovarian Cancer Risk
Population Based Germline Testing for Early Detection and Prevention of Cancer
PROTECT-C is a research study offering genetic testing to people to see whether they have a genetic change that increases their risk of breast, ovary, bowel, and/or womb cancer.
- Conditions
- Breast Cancer Risk, Ovarian Cancer Risk, Cancer Gene Mutation
- Trial name
- PROTECT-C (NCT07498829)
- Testing
- Genetic testing for Cancer Susceptibility Genes (CSGs) (BRCA1, BRCA2, RAD51C, RAD51D, BRIP1, PALB2, MLH1, MSH2, MSH6) and personalised breast and ovarian cancer risk
- Where
- United Kingdom
- Volunteers
- 6,000 people, healthy volunteers welcome
- Sponsor
- Queen Mary University of London
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results
Genetic mutations have closely linked to the pathogenesis and prognostication of myeloid cancers. In addition, a number of molecularly targeted agents have been developed in recent years.
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Aplastic Anemia
- Trial name
- NCT04060485
- Where
- Taiwan
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Acute Pancreatitis
The Association Between Post-ERCP Acute Pancreatitis and Various Genetic Mutations
Pancreatitis remains the most common complication of ERCP, with the reported incidence ranging from 2% to 9%. Although 80% of cases are mild, a significant number of patients may develop severe pancreatitis, that means additional…
- Trial name
- NCT02928718
- Testing
- ERCP
- Where
- South Korea
- Volunteers
- 75 people
- Sponsor
- Seoul National University Hospital
Lung Neoplasms, Carcinoma, Non-Small-Cell Lung
(SYMPHONY) Phase 1/2 Study Targeting EGFR Resistance Mechanisms in NSCLC
This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and anticancer activity of BLU-945, a selective EGFR inhibitor, as…
- Conditions
- Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Neoplasms
- Trial name
- NCT04862780
- Testing
- BLU-945, osimertinib
- Where
- 26 sites in United States, Canada and 8 more
- Volunteers
- 177 people
- Sponsor
- Blueprint Medicines Corporation
Non-Small Cell Lung Cancer
Efficacy and Resistance Mechanisms of IP in NSCLC With Leptomeningeal Metastases
This is a prospective interventional study clinical study to investigate the efficacy and resistance mechanisms of intrathecal pemetrexed in patients with driver gene mutations advanced NSCLC with leptomeningeal metastases.
- Trial name
- EPIC (NCT06440954)
- Testing
- Intrathecal pemetrexed
- Where
- China
- Volunteers
- 220 people
- Sponsor
- Hunan Province Tumor Hospital
Sickle Cell Disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult…
- Trial name
- SPARKLE (NCT06439082)
- Testing
- Crizanlizumab
- Where
- 36 sites in United States, Brazil and 3 more
- Volunteers
- 354 people
- Sponsor
- Novartis Pharmaceuticals
Parkinson Disease, GBA Gene Mutation
Ambroxol as a Disease-modifying Treatment in GBA-PD
The present multicenter, randomized, double-blind, placebo-controlled clinical trial will investigate whether the prolonged administration of high-dose oral Ambroxol over 52 weeks is safe, tolerable, able to change…
- Trial name
- AMBITIOUS (NCT05287503)
- Testing
- Ambroxol Hydrochloride
- Where
- 3 sites in Italy
- Volunteers
- 65 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
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