Clinical trials: Gene Mutation
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms
A Study to Learn About the Study Medicine PF-07985045 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Change in a Gene.
The purpose of this study is to learn about the safety and effects of the study medicine when given alone or together with other anti-cancer therapies. Anti-cancer therapy is a type of treatment to stop the growth of cancer.
- Conditions
- Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
- Trial name
- NCT06704724
- Testing
- PF-07985045, Gemcitabine, Nab-paclitaxel
- Where
- 13 sites in United States, Puerto Rico
- Volunteers
- 30 people
- Sponsor
- Pfizer
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms
A Study to Learn About the Study Medicine PF-07934040 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Genetic Mutation.
The purpose of this study is to learn about the safety and effects of the study medicine alone or when given together with other anti-cancer therapies. This study also aims to find the best dose.
- Conditions
- Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
- Trial name
- NCT06447662
- Testing
- PF-07934040, Gemcitabine, Nab-paclitaxel
- Where
- 27 sites in United States, Puerto Rico
- Volunteers
- 64 people
- Sponsor
- Pfizer
EGFR-TKI-naïve Patients With NSCLC Harboring EGFR Activating Mutations
A Study of ASP8273 in Epidermal Growth Factor Receptor (EGFR) Tyrosine Kinase Inhibitor-Naïve Patients With Non-Small Cell Lung Cancer Harboring EGFR Mutations
The purpose of this study is to determine the safety, the antitumor activity and the pharmacokinetics of ASP8273 in EGFR tyrosine kinase inhibitor (EGFR-TKI)-naïve patients with non-small cell lung cancer (NSCLC) harboring EGFR…
- Trial name
- NCT02500927
- Testing
- ASP8273 Capsules, ASP8273 Capsules A
- Where
- 11 sites in Japan
- Volunteers
- 31 people
- Sponsor
- Astellas Pharma Inc
Achromatopsia
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene.
- Trial name
- NCT02599922
- Testing
- rAAV2tYF-PR1.7-hCNGB3
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Beacon Therapeutics
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Trial name
- NCT06196827
- Testing
- LX101
- Where
- 2 sites in China
- Volunteers
- 9 people
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Advanced or Metastatic Solid Tumors With Germline PTEN Inactivating Mutations
TAS-117 in Patients With Advanced Solid Tumors Harboring Germline PTEN Mutations
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of TAS-117 in patients with advanced or metastatic solid tumors (excluding primary brain tumors)…
- Trial name
- NCT04770246
- Testing
- TAS-117
- Where
- 8 sites in United States, Austria and 2 more
- Volunteers
- 17 people
- Sponsor
- Taiho Oncology, Inc.
Single Gene Disorders
Clinical Use of Parental Support To Detect Single Gene Mutations
Gene Security Network has developed a novel technology called Parental Support (PS) which is used for Preimplantation Genetic Screening/Diagnosis (PGS/D) during in vitro fertilization (IVF).
- Trial name
- NCT01197872
- Testing
- Preimplantation Genetic Diagnosis
- Where
- United States
- Volunteers
- 240 people
- Sponsor
- Natera, Inc.
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.
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Measles, Mumps
Safety, Tolerability, and Immunogenicity of Measles, Mumps, Rubella, and Varicella (MMRV) Vaccine Made With an Alternative Manufacturing Process (AMP)(V221-027)
This study will compare the safety, tolerability, and immunogenicity of measles, mumps, rubella, and varicella (MMRV) vaccine made with an alternative manufacturing process with those of the 2006 process
- Conditions
- Measles, Mumps, Rubella, Varicella
- Trial name
- NCT01536405
- Testing
- MMRV (AMP), MMRV (2006 process)
- Volunteers
- 1,412 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Melanoma
Ph I Ipilimumab Vemurafenib Combo in Patients With v-Raf Murine Sarcoma Viral Oncogene Homolog B1 (BRAF)
Treatment of subjects who have metastatic melanoma that expresses an activated mutant form of the BRAF oncogene (V600E) with a combination of the specific BRAF inhibitor, Vemurafenib, and the Cytotoxic T Lymphocyte Antigen 4…
- Trial name
- NCT01400451
- Testing
- Ipilimumab (BMS-734016), Vemurafenib
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Bristol-Myers Squibb
Non-small Cell Lung Cancer
Phase II of Neoadjuvant and Adjuvant Capmatinib in NSCLC
This study was planned to determine if neoadjuvant capmatinib could improve the major pathological response (MPR) in patients with Stage IB-IIIA, N2 and selected IIIB (T3N2 or T4N2) lung cancers with Mesenchymal Epithelial…
- Trial name
- Geometry-N (NCT04926831)
- Testing
- capmatinib
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Non Small Cell Lung Cancer
XZP-5809-TT1 Tablets in Patients With T790M Mutation-positive Locally Advanced or Metastatic Non-small Cell Lung Cancer
The trial is divided into two parts, one is dose escalation phase, the second one is dose expansion phase. For dose escalation phase, the main purpose is to evaluate safety and tolerability of XZP-5809-TT1 tablets after treatment…
- Trial name
- NCT04622072
- Testing
- XZP-5809-TT1 Tablet
- Where
- 5 sites in China
- Volunteers
- 21 people
- Sponsor
- Sihuan Pharmaceutical Holdings Group Ltd.
Myeloproliferative Neoplasms
A Study to Evaluate INCA033989 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasms
This study is being conducted to evaluate the safety, tolerability, dose-limiting toxicity (DLT) and determine the maximum tolerated dose (MTD) and/or recommended dose(s) for expansion (RDE) of INCA033989 administered as a…
- Trial name
- NCT06034002
- Testing
- INCA033989, Ruxolitinib
- Where
- 13 sites in United States
- Volunteers
- 290 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis
Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation)
32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting of three consecutive treatment periods: two 1-week periods followed by one 2-week period, evaluating one dose of GLPG1837 each.
- Trial name
- SAPHIRA1 (NCT02707562)
- Testing
- GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
- Where
- 16 sites in Australia, Czechia and 3 more
- Volunteers
- 26 people
- Sponsor
- Lakefront Biotherapeutics NV
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
Metastatic Castration-resistant Prostate Cancer (mCRPC), Prostatic Neoplasms
A Study of Opevesostat (MK-5684) Versus Alternative Next-generation Hormonal Agent (NHA) in Metastatic Castration-resistant Prostate Cancer (mCRPC) Post One NHA (MK-5684-004)
The purpose of this study is to assess the efficacy and safety of opevesostat plus daily corticosteroids compared to alternative abiraterone acetate or enzalutamide in participants with Metastatic Castration-resistant Prostate…
- Trial name
- NCT06136650
- Testing
- Opevesostat, Dexamethasone, Fludrocortisone acetate
- Where
- 330 sites in United States, Australia and 37 more
- Volunteers
- 1,314 people
- Sponsor
- Merck Sharp & Dohme LLC
Colorectal Neoplasms, Advanced Metastatic Colorectal Cancer
Biomarker Analysis for Patients With Metastatic Colorectal Cancer (MCC)
Patients are being asked to participate in this study who have colorectal cancer that has come back after initial treatment. The investigators want to improve treatment in patients with this disease.
- Trial name
- NCT01909362
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- US Oncology Research
Metastatic Melanoma
HD IL-2 + Vemurafenib in Patients With BRAF Mutation Positive Metastatic Melanoma
This is a research study to evaluate treatment of metastatic melanoma patients with a combination of drugs. The combination being studied is vemurafenib (also known as Zelboraf®) and High Dose Interleukin-2 (abbreviated as HD…
- Trial name
- PROCLIVITY01 (NCT01683188)
- Testing
- vemurafenib + HD IL-2
- Where
- 21 sites in United States
- Volunteers
- 53 people
- Sponsor
- Clinigen, Inc.
FLT3-ITD Mutation, FLT3/TKD Mutation
Individual Patient Compassionate Use of Crenolanib
Compassionate use of crenolanib for patients with serious life-threatening illness that have exhausted all available therapies used to treat the disease, with no other viable therapy options, who is not eligible for clinical…
- Conditions
- FLT3-ITD Mutation, FLT3/TKD Mutation, PDGFR-Alpha D842V, PDGFRA Gene Amplification
- Trial name
- NCT03620318
- Testing
- Crenolanib besylate
- Where
- Italy
- Sponsor
- Arog Pharmaceuticals, Inc.