Clinical trials: Genetic Mutation
Solid Tumor
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of QLC1101 in Combination With Other Therapies in the Treatment of Patients With Advanced Solid Tumors Harboring a KRAS G12D Mutation
A Phase Ib/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of QLC1101 in Combination with Other Therapies in the Treatment of Patients With Advanced Solid Tumors Harboring a KRAS G12D Mutation.
- Trial name
- NCT06949761
- Testing
- QLC1101+QL1203, QLC1101+QL2107, QLC1101+QL1706
- Volunteers
- 240 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Melanoma, Solid Tumor
Tovorafenib (DAY101) or in Combination With Pimasertib for Participants With Melanoma and Other Solid Tumors
This is a subprotocol of Master Protocol DAY101-102 and is a Phase 1b/2, multi-center, open label subprotocol of participants ≥12 years of age, with recurrent or progressive solid tumors with alterations in the key proteins of…
- Conditions
- Melanoma, Solid Tumor, Pilocytic Astrocytoma, Non Small Cell Lung Cancer
- Trial name
- NCT07121829
- Testing
- Tovorafenib, Tovorafenib Drug: Pimasertib
- Where
- 10 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Day One Biopharmaceuticals, Inc.
Advanced Solid Tumours
A Study Investigating DNA-damage Response Agents in Molecularly Altered Advanced Cancer
The study is investigating efficacy, safety and tolerability of DNA-damage Response Agents (or Combinations), in participants with advanced/metastatic solid malignancies whose tumours contain molecular alterations
- Trial name
- NCT04564027
- Testing
- Ceralasertib
- Where
- 18 sites in United States, France and 1 more
- Volunteers
- 54 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following…
- Trial name
- KONNECTION (NCT01614470)
- Testing
- Ivacaftor
- Where
- 12 sites in United States, Belgium and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
- Trial name
- NCT07523581
- Testing
- Exaluren
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Non Small Cell Lung Cancer
Safety, Pharmacokinetic and Preliminary Efficacy Study of AC0010MA in Advanced Non Small Cell Lung Cancer
AC0010MA is a new, irreversible, Epidermal Growth Factor Receptor (EGFR) mutation selective Tyrosine Kinase Inhibitor. Aim at local advanced or metastatic non-small cell lung cancer patients with EGFR mutation or T790M…
- Trial name
- NCT02448251
- Testing
- AC0010MA
- Where
- 8 sites in United States, France and 1 more
- Volunteers
- 28 people
- Sponsor
- ACEA Therapeutics, Inc.
Retinitis Pigmentosa
Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene
The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B.
- Trial name
- NCT03328130
- Testing
- AAV2/5-hPDE6B
- Where
- France
- Volunteers
- 19 people
- Sponsor
- eyeDNA Therapeutics
HER2-negative Breast Cancer
Efficacy and Safety of BGB-290 in the Treatment of Metastatic HER2-Negative Breast Cancer Patients With BRCA Mutation in China
This is a Phase 2, open-label, multi-center study of BGB-290 administered orally (PO) twice daily (BID) in adult Chinese patients with advanced HER2(-) breast cancer harboring germline BRCA mutation, which have progressed despite…
- Trial name
- NCT03575065
- Testing
- BGB-290
- Where
- 20 sites in China
- Volunteers
- 88 people
- Sponsor
- BeiGene
Anaplastic Thyroid Cancer, ATC
The Efficacy and Safety of HLX208 in Advanced Anaplastic Thyroid Cancer (ATC) With BRAF V600 Mutation
The purpose of this study was to assess efficacy, safety and PK in anaplastic thyroid cancer (ATC) given HLX208 (BRAF V600E inhibitor).
- Trial name
- NCT05102292
- Testing
- HLX208
- Where
- China
- Volunteers
- 25 people
- Sponsor
- Shanghai Henlius Biotech
Dementia, Frontotemporal
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants
This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a…
- Trial name
- SORT-IN-1 (NCT06226064)
- Testing
- VES001
- Where
- Netherlands
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Vesper Biotechnologies ApS
Advanced Solid Tumor, Metastatic Solid Tumor
A Study of PRT7732, an Oral SMARCA2 Degrader, in Patients With Advanced or Metastatic Solid Tumors With a SMARCA4 Mutation
This is a Phase 1 study to determine the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of PRT7732 in patients with select advanced or metastatic solid tumors with a SMARCA4 mutation.
- Conditions
- Advanced Solid Tumor, Metastatic Solid Tumor, Non-small Cell Lung Carcinoma, SMARCA4 Mutation
- Trial name
- NCT06560645
- Testing
- PRT7732
- Where
- 28 sites in United States, Australia and 4 more
- Volunteers
- 42 people
- Sponsor
- Prelude Therapeutics Incorporated
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGYZE Study
Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the OXPHOS.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS Syndrome
- Trial name
- NCT04165239
- Testing
- KH176
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 27 people
- Sponsor
- Khondrion BV
HER2 Mutant Non-small Cell Lung Cancer, HER2-positive Metastatic Breast Cancer
ELVN-002 in HER2 Mutant Non-Small Cell Lung Cancer
The goal of this clinical trial is to test ELVN-002 in people with cancers that have an abnormal HER2 gene. The main question the trial aims to answer is if ELVN-002 is safe and tolerable at different doses.
- Conditions
- HER2 Mutant Non-small Cell Lung Cancer, HER2-positive Metastatic Breast Cancer, HER2 Gene Mutation, HER2 Amplification
- Trial name
- HER2 (NCT05650879)
- Testing
- ELVN-002, Fam-Trastuzumab Deruxtecan-Nxki, Trastuzumab emtansine
- Where
- 39 sites in United States, Australia and 5 more
- Volunteers
- 198 people
- Sponsor
- Enliven Therapeutics
EGFR Mutation-Related Tumors
A Study of NX-019 in Patients with Advanced, Epidermal Growth Factor Receptor (EGFR) Mutant Cancer
This is a 2-part, first-in-human, open-label study to determine the safety and tolerability of NX-019 and preliminary efficacy in patients with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutant cancer.
- Trial name
- NCT05514496
- Testing
- NX-019
- Where
- 12 sites in United States, South Korea and 1 more
- Volunteers
- 258 people
- Sponsor
- Nalo Therapeutics Inc.
Carcinoma, Non-Small-Cell Lung
A Study Evaluating HS-10370 Plus Platinum-based Doublet Chemotherapy With or Without Adebrelimab vs. Tislelizumab Plus Chemotherapy as First-Line Therapy in Patients With Advanced KRAS G12C-Mutant Non-small Cell Lung Cancer
This is a trial to evaluate the efficacy, safety, and tolerability of HS-10370 in combination with Platinum-based Doublet Chemotherapy With or Without Adebrelimab Versus Tislelizumab Plus Platinum-based Doublet Chemotherapy as…
- Trial name
- NCT07822542
- Testing
- HS-10370, Adebrelimab, Cisplatin
- Volunteers
- 448 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
KRAS Activating Mutation, Metastatic Cancer
Study of Avutometinib (VS-6766) +Defactinib With Gemcitabine and Nab-paclitaxel in Patients With Pancreatic Cancer
This study will assess the safety and efficacy of avutometinib (VS-6766) and defactinib in combination with gemcitabine and nab-paclitaxel in patients with Pancreatic Ductal Adenocarcinoma (PDAC) who have been previously…
- Conditions
- KRAS Activating Mutation, Metastatic Cancer, Pancreas Cancer, Neoplasms Pancreatic
- Trial name
- RAMP205 (NCT05669482)
- Testing
- avutometinib (VS-6766) and defactinib in combination with gemcitabine and nab-paclitaxel
- Where
- 12 sites in United States
- Volunteers
- 40 people
- Sponsor
- Verastem, Inc.
Cancer
A Phase I Study to Investigate the Safety, Tolerability and Pharmacokinetic Profile of of GSK2118436 in Japanese Subjects With BRAF Mutation Positive Solid Tumors
BRF116056 is the first clinical experience with GSK2118436, a BRAF inhibitor, in Japan. This study will be designed to assess safety, tolerability, single and repeat dose PK profile and preliminary efficacy of GSK2118436 in…
- Trial name
- NCT01582997
- Testing
- GSK2118436
- Where
- 2 sites in Japan
- Volunteers
- 12 people
- Sponsor
- GlaxoSmithKline
Cystic Fibrosis
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
- Trial name
- NCT04969224
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Belgium and 2 more
- Volunteers
- 82 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Parkinson Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
The main goal of this clinical trial is to learn about the safety and tolerability of GT-02287. The questions it aims to answer are: What medical problems do participants have when taking GT-02287? How is GT-02287 absorbed…
- Trial name
- NCT06732180
- Testing
- GT-02287
- Where
- 7 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Gain Therapeutics, Inc.
Cystic Fibrosis
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).
- Trial name
- NCT03460990
- Testing
- VX-659/TEZ/IVA, TEZ/IVA, IVA
- Where
- 46 sites in United States, Australia and 4 more
- Volunteers
- 116 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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