Clinical trials: Genetic Mutation
HIV-1-infection, HIV Infections
Study to Evaluate Safety, Tolerance and Efficacy of ASC22 Combined With ART in Subjects With HIV
This study is a phase II, multicenter, randomized, blind, placebo-controlled to evaluate the safety, tolerance, efficacy of ASC22 injection in combination with anti-retroviral therapy to treat subjects living with human…
- Conditions
- HIV-1-infection, HIV Infections, PD-L1 Gene Mutation
- Trial name
- NCT05330143
- Testing
- ASC22 1mg/kg, ASC22 2.5mg/kg, Antiretroviral Therapy
- Where
- China
- Volunteers
- 19 people
- Sponsor
- Ascletis Pharmaceuticals Co., Ltd.
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Lymphoma, Follicular
A Study of Tazemetostat on Safety in Participants With Relapsed or Refractory Follicular Lymphoma With Enhancer of Zeste Homolog 2 (EZH2) Gene Mutation in Japan
The primary purpose of the study is to investigate the safety of tazemetostat in participants with relapsed or refractory follicular lymphoma with EZH2 gene mutation under daily clinical practice.
- Trial name
- NCT05228158
- Testing
- Tazemetostat
- Where
- 135 sites in Japan
- Volunteers
- 145 people
- Sponsor
- Eisai Co., Ltd.
Lung Sarcomatoid Carcinoma
A Phase II Study of HMPL-504 in Lung Sarcomatoid Carcinoma and Other Non-small Cell Lung Cancer
This is a Phase II, open-label, multicenter study of Savolitinib administered orally once per day(QD) to locally advanced/metastatic PSC patients and other NSCLC patients with MET Exon 14 mutation.
- Trial name
- NCT02897479
- Testing
- Savolitinib
- Where
- 2 sites in China
- Volunteers
- 76 people
- Sponsor
- Hutchison Medipharma Limited
Neoplasms
A Study of The Impact of Severe Hepatic Impairment on the Pharmacokinetics and Safety of Vemurafenib in BRAF V600 Mutation-Positive Cancer Participants
This open-label, Phase I study will evaluate the impact of severe hepatic impairment on the pharmacokinetics and safety of vemurafenib in participants with BRAF V600 mutation positive cancer.
- Trial name
- NCT01767623
- Testing
- Vemurafenib
- Where
- 8 sites in United States, Australia and 5 more
- Volunteers
- 8 people
- Sponsor
- Hoffmann-La Roche
Non Small Cell Lung Cancer
AZD6244 in Combination With Docetaxel Versus Docetaxel Alone in KRAS Mutation Positive NSCLC Patients
The purpose of this study is to compare the efficacy of AZD6244 in combination with docetaxel versus docetaxel alone in patients with KRAS mutation positive locally advanced or metastatic non small cell lung cancer.
- Trial name
- NCT00890825
- Testing
- AZD6244, docetaxel
- Where
- 63 sites in United States, Belgium and 10 more
- Volunteers
- 88 people
- Sponsor
- AstraZeneca
β-thalassemia Major
β-globin Restored Autologous HSC in β-thalassemia Major Patients
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
- Trial name
- NCT04205435
- Testing
- β-globin restored autologous HSC
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Bioray Laboratories
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
NSCLC
A Study of TY-9591 With Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor NSCLC
This study was a parallel, randomized, multicenter phase II clinical trial to evaluate the efficacy and safety of TY-9591 combined with platinum-based chemotherapy as first-line treatment in patients with locally advanced or…
- Trial name
- NCT06672068
- Testing
- TY-9591 80mg QD+pemetrexed+Cisplatin/Carboplatin, TY-9591 160mg QD+pemetrexed+Cisplatin/Carboplatin
- Volunteers
- 60 people
- Sponsor
- TYK Medicines, Inc
Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Non-Small Cell Lung Cancer
Phase 1b/2 Study of Futibatinib in Combination With Binimetinib in Patients With Advanced KRAS Mutant Cancer
Phase 1b/2 study to evaluate the FGFRi futibatinib in combination with the MEKi binimetinib in patients with advanced KRASmt tumors.
- Conditions
- Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Non-Small Cell Lung Cancer, KRAS Gene Mutation
- Trial name
- NCT04965818
- Testing
- Futibatinib and Binimetinib
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- Taiho Oncology, Inc.
Non Small Cell Lung Cancer
A Phase Ib/II Clinical Trial to Evaluate the Safety and Efficacy of HLX208+HLX10 in NSCLC With BRAF V600E Mutation
An open-label, multicenter phase Ib/II clinical study to evaluate safety, tolerability, pharmacokinetics, and efficacy of HLX208 (BRAF V600E Inhibitor) combined with HLX10 （anti-PD-1 monoclonal antibody）in advanced NSCLC patients…
- Trial name
- NCT05641493
- Testing
- HLX208+HLX10
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Shanghai Henlius Biotech
Familial Adenomatous Polyposis
Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)
This is a multicenter, two-part trial in participants with FAP.
- Trial name
- TUPELO (NCT05552755)
- Testing
- REC-4881
- Where
- 17 sites in United States
- Volunteers
- 67 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Advanced Solid Tumor
Phase I Clinical Study of YL-15293 in Patients With Advanced Solid Tumor With KRAS Mutation
This is a phase 1 / 2 open label multicenter study to evaluate the maximum tolerance, safety, tolerance and PK of oral YL-15293 in patients with advanced solid tumors with KRAS mutation, so as to confirm the recommended phase 2…
- Trial name
- NCT05173805
- Testing
- YL-15293
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Shanghai YingLi Pharmaceutical Co. Ltd.
Advanced/Metastatic Non-Small Cell Lung Cancer (NSCLC)
PH009-1 in Patients With EGFR Mutation Locally Advanced or Metastatic Non-small Cell Lung Cancer (NSCLC)
The study will contain three stages: Phase I includes dose escalation phase (i.e., phase Ia) and dose expansion phase (i.e., phase Ib).
- Trial name
- NCT06590194
- Testing
- PH009-1 tablet
- Where
- China
- Volunteers
- 96 people
- Sponsor
- Suzhou Puhe Pharmaceutical Technology Co., LTD
EGFR T790M Mutation Positive NSCLC
AZD9291 US Expanded Access Program
To provide access to AZD9291 for adult patients with advanced/metastatic, epidermal growth factor receptor T790M mutation-positive non-small cell lung cancer.
- Trial name
- NCT02451852
- Testing
- AZD9291
- Where
- 32 sites in United States
- Sponsor
- AstraZeneca
RAS Mutation, Non-small Cell Lung Cancer (NSCLC)
This is a Multicenter, Open-label, Phase I/II Study to Explore the Safety, Tolerability, Pharmacokinetics (PK), and Preliminary Efficacy of GFH276 in Patients With Advanced NSCLC Harboring a RAS Mutation.
This is an open-label, multicenter Phase Ib/II clinical study evaluating GFH276 in combination with tislelizumab plus pemetrexed/platinum based regimen as first-line therapy in participants with locally advanced or metastatic…
- Trial name
- GFH276X1203 (NCT07846358)
- Testing
- GFH276, with or without Tislelizumab, Pemetrexed
- Volunteers
- 84 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Non-small Cell Lung Cancer, Colorectal Carcinoma
Phase 1 Study to Investigate TCRTs KRAS Mutation in Unresectable, Advanced, and/or Metastatic Solid Tumors
Phase I Study, a master protocol to investigate TCR-Engineered T cells recognizing KRAS mutations in adult subjects with Unresectable, Advanced, and/or Metastatic Solid Tumors.
- Conditions
- Non-small Cell Lung Cancer, Colorectal Carcinoma, Pancreatic Ductal Adenocarcinoma, Endometrial Cancer
- Trial name
- NCT06218914
- Testing
- NT-112: Autologous, engineered T Cells targeting KRAS G12D, AZD0240: Autologous, engineered T Cells targeting KRAS G12D
- Where
- 18 sites in United States
- Volunteers
- 108 people
- Sponsor
- AstraZeneca
Non-Small-Cell Lung Carcinoma
A Post Approval Commitment Study on Tabrecta® (Capmatinib) in South Korea
This is an open label, prospective, multicenter, non-comparative study to assess the safety and effectiveness of Tabrecta® (Capmatinib) in real world setting.
- Trial name
- NCT05703516
- Testing
- Capmatinib
- Where
- 10 sites in South Korea
- Volunteers
- 44 people
- Sponsor
- Novartis Pharmaceuticals
MSS, RAS-mutant Colorectal Cancer
Study of Binimetinib + Nivolumab Plus or Minus Ipilimumab in Patients With Previously Treated Microsatellite-stable (MSS) Metastatic Colorectal Cancer With RAS Mutation
This is a multicenter, open-label, Phase 1B/2 study to evaluate the safety and assess the preliminary anti-tumor activity of binimetinib administered in combination with nivolumab or nivolumab + ipilimumab in adult patients with…
- Trial name
- NCT03271047
- Testing
- binimetinib, nivolumab, ipilimumab
- Where
- 50 sites in United States, Belgium and 3 more
- Volunteers
- 75 people
- Sponsor
- Pfizer