Clinical trials: Genetic Mutation
FLT3 Gene Mutation Negative, FLT3 Internal Tandem Duplication Positive
Quizartinib With Azacitidine or Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome
This phase I/II trial studies the side effects and best dose of quizartinib when given in combination with azacitidine or cytarabine in treating patients with acute myeloid leukemia or myelodysplastic syndrome that have come back…
- Conditions
- FLT3 Gene Mutation Negative, FLT3 Internal Tandem Duplication Positive, Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelomonocytic Leukemia
- Trial name
- NCT01892371
- Testing
- Azacitidine, Cytarabine, Quizartinib
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- M.D. Anderson Cancer Center
BRCA1 Gene Mutation, BRCA2 Gene Mutation
Prospective Research of Outcomes After Salpingo-oophorectomy
PROSper is a prospective cohort study of 100 women 35-50 years of age with BRCA 1/2 mutations who have elected to either undergo risk reducing salpingo-oophorectomy (RRSO) or nonsurgical management.
- Trial name
- PROSper (NCT01948609)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of California, San Francisco
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the…
- Trial name
- NCT02709109
- Testing
- VX-371 + HS, Hypertonic saline, Orkambi
- Where
- 40 sites in United States, France and 2 more
- Volunteers
- 147 people
- Sponsor
- Parion Sciences
Hemophilia A
Study Evaluating ReFacto in Hemophilia A
To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two established hemophilia mutation testing…
- Trial name
- NCT00038909
- Testing
- BDDrFVII
- Volunteers
- 103 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Polyostotic Fibrous Dysplasia
Studies on Abnormal Bone From Patients With Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
This study will investigate how a gene mutation (change in DNA) causes the abnormal bone in fibrous dysplasia-a condition in which areas of normal bone are replaced with a fibrous growth similar to a scar.
- Trial name
- NCT00001973
- Where
- United States
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
EGFR Gene Mutation
EGFR-TKIs Combine With Anlotinib as First-line Treatment for Patients With Advanced EGFR Mutation-positive NSCLC
A single arm study: EGFR-TKIs Combine With Anlotinib as First-line Treatment in patients with EGFR-mutant non-small-cell lung cancer (NSCLC).
- Trial name
- NCT03720873
- Testing
- erlotinib or gefitinib or icotinib, Anlotinib
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Fujian Cancer Hospital
Non Small Cell Lung Cancer
Study of Anlotinib Combined With Icotinib as the First-line Treatment in Patients With EGFR Mutation-positive NSCLC
Evaluate the efficacy and safety of Anlotinib plus Icotinib as the first-line treatment in patients with sensitive EGFR mutations advanced non-small cell lung cancer.
- Trial name
- ALTER-L004 (NCT03736837)
- Testing
- Anlotinib Plus Icotinib
- Where
- 5 sites in China
- Volunteers
- 60 people
- Sponsor
- Tianjin Medical University Cancer Institute and Hospital
Melanoma, Malignant Melanoma
T3011 in Combination With Cobimetinib in Patients With Advanced Melanoma
This study will evaluate the efficacy and safety of T3011 in combination with Cobimetinib in patients with advanced melanoma.
- Trial name
- NCT05756556
- Testing
- T3011 + Cobimetinib
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- ImmVira Pharma Co. Ltd
Prostate Cancer
A Study to Learn About the Safety of TALZENNA in People With Prostate Cancer.
The purpose of this study is to learn about the safety of TALZENNA in patients with BRCA mutation-positive metastatic castration-resistant prostate cancer. BRCA mutation positive means any changes to the BRCA gene.
- Trial name
- NCT06733337
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Pfizer
Melanoma
MEK Inhibitor FCN-159 To Treat Advanced Melanoma With NRAS-aberrant (Ia) and NRAS-mutant (Ib)or NF1-mutant(1b)
Melanoma is one of the most common cutaneous cancers worldwide. Activating mutations in RAS oncogenes are found in a third of all human cancers and NRAS mutations are found in 15%-20% of melanomas.
- Trial name
- NCT03932253
- Testing
- FCN-159
- Where
- China
- Volunteers
- 79 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Biliary Tract Cancer, HER2 Gene Mutation
Efficacy and Safety of GemCis Plus Trastuzumab Plus Pembrolizumab in Previously Untreated HER2-positive Biliary Tract Cancer
This is a multicenter, single arm, prospective, open-label phase II trial investigating the clinical activity triplet regimen consisting of a combination of chemotherapy (gemcitabine/cisplatin) + trastuzumab + pembrolizumab as…
- Conditions
- Biliary Tract Cancer, HER2 Gene Mutation, HER2
- Trial name
- TRAP-BTC (NCT06178445)
- Testing
- SOC plus trastuzumab and pembrolizumab
- Where
- 8 sites in Germany
- Volunteers
- 24 people
- Sponsor
- Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest
MDC1A
LAMA2 Genetic Correction
Merosin-deficient congenital muscle dystrophy type 1a (MDC1a), or LAMA2 muscular dystrophy (LAMA2-MD) is a severe autosomal recessive form of muscular dystrophy that is caused by homozygous or compound heterozygous mutations in…
- Trial name
- NCT06582537
- Testing
- Participants sample collection
- Where
- Netherlands
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Maastricht University
Spinal Cord Stimulation, Failed Back Surgery Syndrome
Transcriptomic Profile of Patients Treated With Different Modalities of Spinal Cord Stimulation
Failed Back Surgery Syndrome (FBSS) is a relatively common condition that can cause a severe disability in patients. Spinal cord stimulation (SCS) is used in those patients refractory to conventional therapies.In this project the…
- Conditions
- Spinal Cord Stimulation, Failed Back Surgery Syndrome, Genetic Change
- Trial name
- SCS-OMICS (NCT05712980)
- Testing
- Implanted pulse generator for SCS with different programming modalities
- Where
- Spain
- Volunteers
- 40 people
- Sponsor
- Hospital General Universitario de Valencia
Non Small Cell Lung Cancer, KRAS Gene Mutation
Trial of Trametinib and Ponatinib in Patients With KRAS Mutant Advanced Non-Small Cell Lung Cancer
The purpose of this study is to evaluate the safety of the combination of ponatinib and trametinib as well as the most appropriate dosages of the combination.
- Trial name
- NCT03704688
- Testing
- Trametinib 0.5 mg, Trametinib 1 MG, Trametinib 1.5 MG
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Frontotemporal Lobar Degeneration
Natural History Characterization in Symptomatic and Asymptomatic Progranuline Gene Mutation Carriers
The purpose of this study is to investigate whether cognitive deficits, structural and functional changes can be detected before symptom onset in presymptomatic progranuline mutation carriers.
- Trial name
- Predict-PGRN (NCT04014673)
- Testing
- Behavioral : Characterization
- Where
- 2 sites in France
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Melanoma Stage III, Melanoma Stage IV
Adjuvant Therapy Based on Pathologic Response After Neoadjuvant Encorafenib Binimetinib in Melanoma
The purpose of this study is to assess rate of disease relapse and hazard rate of disease relapse after neoadjuvant therapy based on the statuses of pathologic complete response or non-pathologic complete response, and…
- Conditions
- Melanoma Stage III, Melanoma Stage IV, BRAF V600 Mutation
- Trial name
- NCT04741997
- Testing
- Encorafenib Pill, Binimetinib Pill, Nivolumab
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Diffuse Midline Glioma (DMG), Diffuse Intrinsic Pontine Glioma (DIPG)
Targeted LSAM-Cisplatin Infusion in Gliomas, Evaluation of Response
Open-label, dose-escalating, Phase 1/2a trial of Large Surface Area Microparticle (LSAM)-Cisplatin to treat participants with diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), via stereotactic…
- Conditions
- Diffuse Midline Glioma (DMG), Diffuse Intrinsic Pontine Glioma (DIPG), Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma
- Trial name
- TIGER-1 (NCT07767110)
- Testing
- LSAM-Cisplatin
- Volunteers
- 20 people
- Sponsor
- NanOlogy, LLC
Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma
Sapanisertib in Treating Patients With Locally Advanced or Metastatic Bladder Cancer With TSC1 and/or TSC2 Mutations
This pilot phase II trial studies how well sapanisertib works in treating patients with bladder cancer that has spread from where it started to nearby tissue or lymph nodes (locally advanced) or other places in the body…
- Conditions
- Locally Advanced Bladder Urothelial Carcinoma, Metastatic Transitional Cell Carcinoma, Metastatic Urothelial Carcinoma, Recurrent Bladder Carcinoma
- Trial name
- NCT03047213
- Testing
- Sapanisertib
- Where
- 20 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Solid Tumor, Non-Small Cell Lung Cancer
A First-in-Human Trial of BLU-924 (SAR449336) in Advanced Solid Tumors Harboring KRAS Mutations
A first in human study to evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of BLU-924 / SAR449336, a pan-KRAS inhibitor, in participants with advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or…
- Conditions
- Advanced Solid Tumor, Non-Small Cell Lung Cancer, Colorectal Neoplasms, Pancreatic Ductal Adenocarcinoma
- Trial name
- NCT07629960
- Testing
- BLU-924
- Where
- 16 sites in United States, Spain
- Volunteers
- 265 people
- Sponsor
- Blueprint Medicines Corporation
Advanced Cancer, Metastatic Cancer
Personalized Vaccine Generated by Autologous Dendritic Cells Pulsed With Autologous Whole Tumor Cell Lysate Treat Advanced Solid Tumor Patients With High Tumor Mutation Burden
The study is to investigate the safety and efficacy of dendritic cells vaccines pulsed with autologous whole tumor cell lysate for treating advanced solid tumor patients with high tumor mutation burden.
- Trial name
- NCT03671720
- Testing
- personalized DC vaccine
- Where
- China
- Sponsor
- Capital Medical University
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