Clinical trials: Genetic Mutation
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
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.
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
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
Metastatic Melanoma
A Study Evaluating the Safety and Efficacy of Cobimetinib Plus Atezolizumab in BRAFV600 Wild-type Melanoma With Central Nervous System Metastases and Cobimetinib Plus Atezolizumab and Vemurafenib in BRAFV600 Mutation-positive Melanoma With Central Nervous System Metastases
This study will evaluate the efficacy and safety of cobimetinib plus atezolizumab in participants with BRAFV600 wild-type melanoma with central nervous system (CNS) metastases and of cobimetinib plus atezolizumab and vemurafenib…
- Trial name
- NCT03625141
- Testing
- Cobimetinib, Atezolizumab, Vemurafenib
- Where
- 22 sites in Brazil, France and 6 more
- Volunteers
- 80 people
- Sponsor
- Hoffmann-La Roche
Granulin Mutation
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Trial name
- NCT02149160
- Testing
- FRM-0334
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 30 people
- Sponsor
- FORUM Pharmaceuticals Inc
DFNB9
An Investigator Initiated Study for OTOV101N+OTOV101C Injection
This study is an investigator initiated study to evaluate the safety, tolerability, and efficacy of OTOV101N+OTOV101C injection in treating patients with OTOF mutation-related deafness.
- Trial name
- NCT05901480
- Testing
- OTOV101N+OTOV101C Injections
- Where
- 23 sites in China
- Volunteers
- 25 people
- Sponsor
- Otovia Therapeutics
Melanoma
Dabrafenib With Trametinib in the Adjuvant Treatment of High-risk BRAF V600 Mutation-positive Melanoma (COMBI-AD).
This was a two-arm, randomized, double-blind Phase III study of dabrafenib in combination with trametinib versus 2 placebos in the adjuvant treatment of melanoma after surgical resection.
- Trial name
- COMBI-AD (NCT01682083)
- Testing
- Dabrafenib, Trametinib
- Where
- 168 sites in United States, Argentina and 23 more
- Volunteers
- 870 people
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Carcinoma (NSCLC)
A Phase II, Open-Label, Multicenter Study of Capmatinib in Subjects With MET Exon 14 Skipping Mutation Positive, Advanced, NSCLC With Brain Metastases
The purpose of this study is to establish the intracranial efficacy of single agent capmatinib in the population of treatment-naïve or pretreated with one or two prior lines of systemic therapies for advanced stage Non Small-Cell…
- Trial name
- NCT04460729
- Testing
- Capmatinib
- Sponsor
- Novartis Pharmaceuticals
Metastatic Hepatocellular Carcinoma, Solid Tumors
Safety and Preliminary Anti-Tumor Activity of TYRA-430 in Advanced Hepatocellular Carcinoma and Other Solid Tumors With Activating FGF/FGFR Pathway Aberrations
A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamic (PD), and preliminary antitumor activity of TYRA-430 in cancers with FGF/FGFR pathway aberrations, including locally advanced/metastatic…
- Conditions
- Metastatic Hepatocellular Carcinoma, Solid Tumors, Solid Tumor, Adult, FGFR Gene Amplification
- Trial name
- SURF431 (NCT06915753)
- Testing
- TYRA-430
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 125 people
- Sponsor
- Tyra Biosciences, Inc
Chronic Lymphocytic Leukemia, Waldenstrom Macroglobulinemia
A Study of Oral LOXO-305 in Patients With Previously Treated CLL/SLL or NHL
This is an open-label, multi-center Phase 1/2 study of oral LOXO-305 (pirtobrutinib) in patients with CLL/SLL and NHL who have failed or are intolerant to standard of care.
- Conditions
- Chronic Lymphocytic Leukemia, Waldenstrom Macroglobulinemia, Mantle Cell Lymphoma, Marginal Zone Lymphoma
- Trial name
- NCT03740529
- Testing
- Pirtobrutinib, Venetoclax, Rituximab
- Where
- 56 sites in United States, Australia and 8 more
- Volunteers
- 803 people
- Sponsor
- Loxo Oncology, Inc.
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants.
- Trial name
- NCT03796637
- Testing
- Ataluren
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
VHL Gene Mutation, VHL
MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)
The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL participants treated with MK-3795.
- Conditions
- VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03108066
- Testing
- MK-3795
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome
Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 Mutation
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in advanced hematologic malignancies that harbor an IDH1 and/or IDH2 mutation
- Conditions
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, Hematologic Malignancies
- Trial name
- NCT02492737
- Testing
- AG881
- Where
- 6 sites in United States, France
- Volunteers
- 46 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Epidermal Growth Factor, Epidermal Growth Factor Receptor
A Phase 1 Study of EPI-326 in EGFR-mutant NSCLC and HNSCC
A phase 1 study to determine the safety, tolerability, PK, PD, and preliminary anti-tumor activity of ascending doses of EPI-326 administered to patients with locally advanced or metastatic HNSCC and to patients with any…
- Conditions
- Epidermal Growth Factor, Epidermal Growth Factor Receptor, Epidermal Growth Factor Receptor Gene Mutation, Non Small Cell
- Trial name
- NCT07462377
- Testing
- EPI-326
- Where
- 8 sites in United States, South Korea and 1 more
- Volunteers
- 110 people
- Sponsor
- EpiBiologics
Solid Tumors, ER+ Breast Cancer
JAB-2485 Activity in Adult Patients With Advanced Solid Tumors
This study is to evaluate the safety and tolerability of JAB-2485 monotherapy in adult participants with advanced solid tumors.
- Conditions
- Solid Tumors, ER+ Breast Cancer, Triple Negative Breast Cancer, TNBC, ARID1A Gene Mutation
- Trial name
- NCT05490472
- Testing
- JAB-2485 (Aurora A inhibitor)
- Where
- 8 sites in United States, China
- Volunteers
- 102 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Severe Familial Hypercholesterolemia
Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
- Trial name
- TAUSSIG (NCT01624142)
- Testing
- Evolocumab
- Where
- 44 sites in United States, Australia and 16 more
- Volunteers
- 300 people
- Sponsor
- Amgen
NSCLC
A Phase 3 Study of Sosimerasib in Combination With IN10018 Versus Standard Therapy for First Line Non-squamous Non-small Cell Lung Cancer With KRAS G12C Mutation
This is a randomized, positive controlled, open-Label and multicenter phase III study, to evaluate the efficacy and safety of Sosimerasib in combination with IN10018 versus anti-PD-1 monoclonal antibody in combination with…
- Trial name
- NCT07833202
- Testing
- Sosimerasib in combination with IN10018, Tislelizumab + Carboplatin + Pemetrexed
- Where
- China
- Volunteers
- 416 people
- Sponsor
- Shanghai Jeyou Pharmaceutical Co., Ltd.
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