Clinical trials: Mutation
Advanced Lung Non-Squamous Non-Small Cell Carcinoma, Recurrent Lung Non-Squamous Non-Small Cell Carcinoma
Combination Treatment (Talazoparib Plus Avelumab) for Stage IV or Recurrent Non-Squamous Non-Small Cell Lung Cancer With STK11 Gene Mutation (A LUNG-MAP Treatment Trial)
This phase II LUNG-MAP treatment trial studies how well combination treatment (talazoparib plus avelumab) works in treating patients with non-squamous non-small cell lung cancer that has an STK11 gene mutation and has come back…
- Conditions
- Advanced Lung Non-Squamous Non-Small Cell Carcinoma, Recurrent Lung Non-Squamous Non-Small Cell Carcinoma, Stage IV Lung Cancer AJCC v8, Stage IVA Lung Cancer AJCC v8
- Trial name
- NCT04173507
- Testing
- Avelumab, Talazoparib, Talazoparib Tosylate
- Where
- 325 sites in United States
- Volunteers
- 47 people
- Sponsor
- SWOG Cancer Research Network
Glioma, BRAF V600E
A Biospecimen Collection Study in BRAF-V600E Mutated Recurrent Gliomas
This is a surgical biospecimen collection study. The purpose of this study is to understand how much of two drugs (dabrafenib and trametinib) are able to penetrate brain tumors and turn off the RAF signaling pathway.
- Conditions
- Glioma, BRAF V600E, Pleomorphic Xantho-Astrocytoma, Glioblastoma
- Trial name
- NCT03593993
- Testing
- Surgical Cohort
- Where
- 5 sites in United States
- Volunteers
- 3 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Advanced or Metastatic Solid Tumors
A Study to Assess Naporafenib (ERAS-254) Administered With Trametinib in Patients With RAS Q61X Mutations
To evaluate the efficacy of naporafenib administered with trametinib in patients with rat sarcoma viral oncogene (RAS) Q61X solid tumors To evaluate the safety and tolerability of naporafenib administered with trametinib in…
- Trial name
- SEACRAFT-1 (NCT05907304)
- Testing
- Naporafenib, Trametinib
- Where
- 29 sites in United States, Australia and 3 more
- Volunteers
- 86 people
- Sponsor
- Erasca, Inc.
Lynch Syndrome
Diagnosis of Lynch Syndrome Based on Next-generation Sequencing in Patients Meeting Chinese Lynch Syndrome Criteria
The purpose of this study is to find out the proportion of patients diagnosed with Lynch syndrome in colorectal cacner patients meeting Chinese Lynch syndrome criteria.
- Trial name
- NCT03046849
- Testing
- next-generation sequencing
- Where
- 3 sites in China
- Volunteers
- 100 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Fabry Disease
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A)…
- Trial name
- NCT01218659
- Testing
- migalastat hydrochloride, agalsidase
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 68 people
- Sponsor
- Amicus Therapeutics
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
Benign Breast Disease, Breast Cancer
Prolactin Receptor and Breast Diseases
Prolactin is known to play an important role in breast development and differentiation. Thus proliferative breast diseases are good models to unravel PRl / PRLR function in proliferative processes.
- Trial name
- Prolacsein (NCT00842465)
- Testing
- blood collection for hormonal status analysis, breast Biopsy or surgery, blood collection
- Where
- France
- Volunteers
- 735 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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.
TP53-mutated MDS and AML, Allogeneic Hematopoietic Stem Cell Transplantation
Maintenance Therapy With Selinexor and Azacitidine in TP53 Mutant AML/MDS After Transplantation
This study aims to explore the safety and efficacy of selinexor combined with azacitidine for maintenance therapy in TP53 mutant AML/MDS patients following transplantation.
- Trial name
- NCT07094464
- Testing
- Maintenance Therapy with Selinexor and Azacitidine
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Daihong Liu
Single Patient Protocol: A Phase II Study Using the Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Mutated Neoantigens in a Patient With Metastatic Cancer Plus the Administration of Pembrolizumab
Single Patient Expanded Access
- Trial name
- NCT04135092
- Testing
- Individual Patient TCR Transduced PBL, Cyclophosphamide, Fludarabine
- Sponsor
- National Institutes of Health Clinical Center (CC)
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Acute Leukemia, KMT2A Rearrangements or NPM1 Mutations Acute Leukemia
BY002 IIT Study in R/R Acute Leukemia
This is a single-center, open-label, investigator-initiated phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of the menin inhibitor BY002 in patients with relapsed or refractory acute…
- Trial name
- NCT07270770
- Testing
- BY002
- Where
- China
- Volunteers
- 18 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Cystic Fibrosis
Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects
The purpose of this research study is to determine the effects of clinically prescribed ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping energy expenditure, growth status and gut health and…
- Trial name
- NCT03783286
- Testing
- Ivacaftor
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance…
- Trial name
- NCT01705145
- Testing
- Ivacaftor
- Where
- 20 sites in United States, Canada and 1 more
- Volunteers
- 35 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non Small Cell Lung Cancer
Tipifarnib in Advanced Squamous NSCLC With Oncogen HRAS MutAtionS
This Phase II study consists of 2 parts: 1) pre-screening phase and 2) treatment phase. The pre-screening phase will investigate the presence of HRAS mutations in subjects with a histologically or cytologically confirmed…
- Trial name
- NCT03496766
- Testing
- Tipifarnib
- Where
- 30 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Spanish Lung Cancer Group
Haematological Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 01: Alectinib in Adult, Paediatric and Teenage/Young Adult Patients With ALK Positive Cancers
This clinical trial is looking at a drug called alectinib. Alectinib is approved as standard of care treatment for adult patients with certain types of lung cancer.
- Conditions
- Haematological Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT05770037)
- Testing
- Alectinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Vascular Dysfunction, Genetic Mutations
Vascular Disease Discovery Protocol
Background: Some genetic diseases put increase the risk of heart and blood diseases, which are the number one cause of death and disability in the U.S. Researchers want to study diseases of the heart and/or blood vessels.
- Conditions
- Vascular Dysfunction, Genetic Mutations, Genetic Predisposition
- Trial name
- NCT03538639
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Non-Small Cell Lung Cancer
KOREA Study (Korea Osimertinib Real World Evidence Study to Assess Safety and Efficacy)
The objectives of this study are to assess the safety and efficacy of single agent Tagrisso (Osimertinib, hereinafter "the study drug") in a real world setting in adult patients with advanced or metastatic, epidermal growth…
- Trial name
- KOREA (NCT02777567)
- Where
- South Korea
- Volunteers
- 315 people
- Sponsor
- AstraZeneca
Hereditary Multiple Melanoma, Melanoma (Skin)
Identifying Gene Mutations in Patients With Melanoma and in Families With a History of Hereditary Melanoma
RATIONALE: Identifying gene mutations and other risk factors in patients with melanoma and in families with a history of hereditary melanoma may help doctors identify persons at risk for melanoma and other types of cancer.
- Trial name
- NCT00450593
- Testing
- gene expression analysis, microarray analysis, molecular genetic technique
- Where
- United Kingdom
- Volunteers
- 5,000 people
- Sponsor
- Leeds Cancer Centre at St. James's University Hospital
Acute Leukemia of Ambiguous Lineage, B Acute Lymphoblastic Leukemia
Testing Blinatumomab With or Without Revumenib in Patients With B-cell Acute Lymphoblastic Leukemia With a Genetic Change Requiring More Treatment
This phase II trial tests how well adding revumenib to usual treatment (blinatumomab) compared to usual treatment alone works in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) or acute leukemia with ambiguous…
- Conditions
- Acute Leukemia of Ambiguous Lineage, B Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative, T Acute Lymphoblastic Leukemia
- Trial name
- NCT07636564
- Testing
- Biospecimen Collection, Blinatumomab, Bone Marrow Aspiration
- Volunteers
- 90 people
- Sponsor
- SWOG Cancer Research Network