Clinical trials: Genetic Mutation
Cholangiocarcinoma
Ivosidenib in Locally Advanced or Metastatic Cholangiocarcinoma With IDH1 R132 Mutation After at Least One Prior Systemic Treatment - an Observational Study
Cholangiocarcinoma is a rare and aggressive tumor of the bile duct associated with a poor prognosis and very limited treatment options. The IDH1 inhibitor ivosidenib provides a new, targeted treatment option for this disease.
- Trial name
- IDHIRA (NCT06607302)
- Where
- 19 sites in Germany
- Volunteers
- 100 people
- Sponsor
- iOMEDICO AG
Colorectal Cancer, Metastatic Colorectal Cancer
Study of Onvansertib in Combination With FOLFIRI and Bevacizumab Versus FOLFIRI and Bevacizumab for Second Line Treatment of Metastatic Colorectal Cancer in Participants With a Kirsten Rat Sarcoma Virus Gene (KRAS) or Neuroblastoma-RAS (NRAS) Mutation
The primary objective of this study is to assess the efficacy of 2 different doses of onvansertib in combination with a chemotherapy regimen of irinotecan, fluorouracil [5-FU], and leucovorin (FOLFIRI) and bevacizumab for…
- Trial name
- NCT05593328
- Testing
- Onvansertib, FOLFIRI, Bevacizumab
- Where
- 27 sites in United States
- Volunteers
- 23 people
- Sponsor
- Cardiff Oncology
Non-small-cell Lung Cancer
Gefitinib With Chemotherapy or Anti-angiogenesis in NSCLC Patients With Bim Deletion or Low EGFR Mutation Abundance
This is an open-label, multicenter, randomized, phase II clinical trial, which aims to evaluate the effectiveness and safety of gefitinib versus combination of gefitinib and doublet chemotherapy or apatinib in advanced non-small…
- Trial name
- NCT03267654
- Testing
- gefitinib combined with chemotherapy, gefitinib combined with apatinib, gefitinib single agent
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Lung Cancer
Real World Utilization and Outcomes With Dacomitinib First Line Treatment for EGFR Mutation-positive Advanced Non Small Cell Lung Cancer Among Asian Patients - A Multi Center Chart Review
This is a longitudinal, consecutive case-series, multi-center study with mixed prospective and retrospective data collection. Data will be collected from eligible adults with EGFR mutation-positive advanced non-small cell lung…
- Trial name
- NCT04609319
- Testing
- Dacomitinib
- Where
- 14 sites in China, India and 1 more
- Volunteers
- 307 people
- Sponsor
- Pfizer
B-Cell Malignancies
Study to Evaluate the Safety and Tolerability of TT-01488 in Patients With B-Cell Malignancies
This is a first-in-human (FIH), multicenter, open-label Phase I dose escalation study to evaluate the safety and preliminary efficacy of the TT-01488 tablet, a non-covalent reversible BTK inhibitor, for the treatment of adult…
- Trial name
- NCT05275504
- Testing
- TT-01488
- Where
- 2 sites in United States
- Volunteers
- 37 people
- Sponsor
- TransThera Sciences (Nanjing), Inc.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- ACT DMD (NCT01826487)
- Testing
- Ataluren
- Where
- 55 sites in United States, Australia and 16 more
- Volunteers
- 230 people
- Sponsor
- PTC Therapeutics
Carcinoma, Renal Cell
DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other Malignancies
This was a first in human study of DFF332, a small molecule that targets a protein called HIF2α. By acting on HIF2α, DFF332 may be able to stop the growth of certain types of cancer.
- Trial name
- NCT04895748
- Testing
- DFF332, RAD001, PDR001
- Where
- 11 sites in United States, Czechia and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Solid Tumor
A Phase 1/2, Study of YL-15293 in Subjects With Advanced Solid Tumors With a KRAS G12C Mutation
This is a Phase 1/2, open-label, multicenter study designed to evaluate the maximum tolerated, safety, tolerability and PK of oral YY-15293 in patients with advanced solid tumors with a KRAS G12C mutation, to confirm the…
- Trial name
- NCT05119933
- Testing
- YL-15293
- Where
- 3 sites in United States
- Volunteers
- 55 people
- Sponsor
- Shanghai YingLi Pharmaceutical Co. Ltd.
Carcinoma, Non-Small Cell Lung
Quaratusugene Ozeplasmid (Reqorsa) and Osimertinib in Patients With Advanced Lung Cancer Who Progressed on Osimertinib
The purpose of this randomized study is to determine the safety and efficacy of quaratusugene ozeplasmid (Reqorsa) added to osimertinib in NSCLC patients with activating EGFR mutations who have progressed while on treatment with…
- Trial name
- Acclaim-1 (NCT04486833)
- Testing
- quaratusugene ozeplasmid, osimertinib, Platinum-Based Chemotherapy
- Where
- 10 sites in United States
- Volunteers
- 158 people
- Sponsor
- Genprex, Inc.
Indolent Systemic Mastocytosis
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.
- Trial name
- AB06006 (NCT00814073)
- Testing
- Masitinib, Best Supportive Care
- Where
- 24 sites in United States, France
- Volunteers
- 135 people
- Sponsor
- AB Science
Advanced Breast Cancer, Advanced or Metastatic Breast Cancer
A Study to Learn About the Study Medicine Called PF-08165784 in People With PIK3CA-Mutated Advanced or Metastatic Solid Tumors
The purpose of this study is to learn about the safety and effects of the study medicine (called PF-08165784) when given alone or together with other anti-cancer therapies.
- Conditions
- Advanced Breast Cancer, Advanced or Metastatic Breast Cancer, Metastatic Breast Cancer (HR+/ HER2-), Metastatic Colorectal Adenocarcinoma
- Trial name
- NCT07897721
- Testing
- PF-08165784, Fulvestrant, Atirmociclib
- Volunteers
- 268 people
- Sponsor
- Pfizer
Acute Myeloid Leukemia (AML)
A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
This is the first study to administer ziftomenib to Japanese patients. In this study, the efficacy, safety, and pharmacokinetics of ziftomenib will be evaluated in patients with relapsed or refractory NPM1-mutated acute myeloid…
- Trial name
- NCT07623616
- Testing
- ziftomenib
- Where
- 20 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Kyowa Kirin Co., Ltd.
KRAS G12D Mutation, NSCLC (Non-small Cell Lung Cancer)
A Study to Evaluate GFH375 Versus Docetaxel in Participants With Non-Small Cell Lung Cancer With KRAS G12D Mutation
The purpose of this study is to compare the effectiveness, safety and tolerability of GFH375 versus docetaxel in participants with KRAS G12D-mutant non-small cell lung cancer (NSCLC).
- Trial name
- NCT07668752
- Testing
- GFH375, Docetaxel
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Stage IIIB/IV, Non-Small Cell Lung Cancer
Diagnosis and Treatment Strategies in Patients With NSCLC With or Without EGFR Mutations.
The purpose of this trial is to compare the evolution of patients receiving first line treatment for locally advanced or metastatic lung cancer, taking into account if they were requested or not the EGFR mutation test as part of…
- Trial name
- IDENTIFY (NCT01525199)
- Where
- 17 sites in Argentina
- Volunteers
- 450 people
- Sponsor
- AstraZeneca
Autosomal Dominant Hypocalcemia
ADH1 and ADH2 Disease Monitoring Study (DMS)
A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease…
- Trial name
- CLARIFY (NCT05227287)
- Where
- 27 sites in United States, Australia and 11 more
- Volunteers
- 95 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Non-small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC)
Study of LT-010391 in Participants With KRAS G12D-Mutant Solid Tumors
This study is to evaluate the safety and tolerability of of LT-010391 as monotherapy in participants with KRAS G12D mutant advanced solid tumors
- Conditions
- Non-small Cell Lung Cancer (NSCLC), Colorectal Cancer (CRC), Pancreatic Ductal Adenocarcinoma (PDAC), Advanced Solid Tumors
- Trial name
- NCT07624214
- Testing
- LT-010391
- Where
- China
- Volunteers
- 198 people
- Sponsor
- Leadingtac Pharmaceutical (Shaoxing) Co., Ltd.
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744)…
- Trial name
- NCT02565914
- Testing
- TEZ/IVA, IVA
- Where
- 154 sites in United States, Australia and 14 more
- Volunteers
- 1,131 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Solid Tumors, Ovarian Cancer
Open-label Phase 1b Study of ARQ 092 in Combination With Anastrozole
An Open-label Phase 1b Study of ARQ 092 in Combination with other Antineoplastic Agents in Subjects with Selected Solid Tumors
- Conditions
- Solid Tumors, Ovarian Cancer, Endometrial Cancer
- Trial name
- NCT02476955
- Testing
- ARQ 092 + carboplatin + paclitaxel (Closed), ARQ 092 + paclitaxel (Closed), ARQ 092 + anastrozole
- Where
- 5 sites in United States
- Volunteers
- 41 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Urea Cycle Disorders
Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function
This diagnostic study will be performed to investigate the performance of the urea cycle in healthy subjects, asymptomatic carriers of Urea Cycle Disorders (UCD) mutations and subjects with genetically proven urea cycle…
- Trial name
- NCT01549015
- Testing
- oral administration of Sodium [1,2-13C]-Acetate
- Where
- 4 sites in Germany
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Cytonet GmbH & Co. KG
Multiple Myeloma, Neoplasms
A Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Cancers
This open-label, multi-center study will assess the efficacy and safety of vemurafenib in participants with BRAF V600 mutation-positive cancers (solid tumors and multiple myeloma, except melanoma and papillary thyroid cancer) and…
- Trial name
- NCT01524978
- Testing
- cetuximab, vemurafenib
- Where
- 34 sites in United States, China and 4 more
- Volunteers
- 208 people
- Sponsor
- Hoffmann-La Roche
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