Clinical trials: Gene Mutations
Non-Small Cell Lung Cancer (NSCLC)
A Study to Investigate Safety and Efficacy of Osimertinib and Amivantamab in Participants With Non-small Cell Lung Cancer With Common Epidermal Growth Factor Receptor Mutations
This study will assess the safety and efficacy of Osimertinib with Amivantamab as First-line Treatment in Participants with Epidermal Growth Factor Receptor Mutation-Positive, Locally Advanced or Metastatic Non-small Cell Lung…
- Trial name
- OSTARA (NCT05801029)
- Testing
- Osimertinib, Amivantamab
- Where
- 28 sites in Hong Kong, Malaysia and 4 more
- Volunteers
- 60 people
- Sponsor
- AstraZeneca
Carcinoma, Non-Small-Cell Lung
Study Evaluating Efficacy and Safety of Capmatinib in Combination With Osimertinib in Adult Subjects With Non-small Cell Lung Cancers as Second Line Therapy
This study aimed to evaluate the anticancer activity of capmatinib in combination with osimertinib compared to platinum-pemetrexed based doublet chemotherapy as second line treatment in patients with advanced or metastatic…
- Trial name
- GEOMETRY-E (NCT04816214)
- Testing
- Capmatinib, Osimertinib, Pemetrexed
- Where
- 4 sites in Japan, Singapore and 1 more
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Carcinoma, Non-Small-Cell Lung
A Study of ZN-e4 in Subjects With Epidermal Growth Factor Receptor Mutated Non-Small Cell Lung Cancer
This is a Phase 1/2, open-label, multicenter, sequential dose-escalation study to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of ZN-e4 administered orally in subjects with advanced non-small…
- Trial name
- NCT03446417
- Testing
- ZN-e4
- Where
- 7 sites in United States, Bosnia and Herzegovina
- Volunteers
- 34 people
- Sponsor
- Zeno Pharmaceuticals, Inc.
Non Small Cell Lung Cancer
A Study to Evaluate Safety and Efficacy of HS-10296 as First-Line Treatment in Patients
This is a randomized, controlled, double-blind, multicenter, phase III clinical study.
- Trial name
- NCT03849768
- Testing
- HS-10296, Gefitinib
- Where
- China
- Volunteers
- 429 people
- Sponsor
- Jiangsu Hansoh Pharmaceutical Co., Ltd.
Non-Squamous Non-Small Cell Lung Cancer
A Study of Tarceva (Erlotinib) in First Line in Patients With Locally Advanced or Metastatic Lung Adenocarcinoma With EGFR Mutations
This open-label, non-randomized, one-arm study will evaluate the safety and efficacy of Tarceva (erlotinib) as single-agent first-line treatment in patients with locally advanced or metastatic non-small cell lung cancer who show…
- Trial name
- NCT01609543
- Testing
- erlotinib [Tarceva]
- Where
- 23 sites in Hungary, Latvia and 1 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
This study is a multicenter, single-arm, open-label Phase I/II clinical trial, which is designed to evaluate the safety, tolerability and efficacy of EHT102 injection in treating congenital hearing loss secondary to biallelic…
- Trial name
- NCT07288580
- Testing
- EHT102 Injection
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shanghai Euhearing Therapeutics Co., Ltd
X-Linked Hypophosphatemia
Registry for Patients With X-Linked Hypophosphatemia
This is an international, multicentre, prospective, non-interventional, observational Registry of patients with X-Linked hypophosphatemia (XLH).
- Trial name
- NCT03193476
- Where
- 118 sites in Belgium, Bulgaria and 18 more
- Volunteers
- 1,489 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Parkinson Disease
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
Study J3Z-MC-OJAA is a Phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal LY3884961 administration in patients with moderate to severe Parkinson's disease…
- Trial name
- NCT04127578
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 15 sites in United States, Israel
- Volunteers
- 32 people
- Sponsor
- Prevail Therapeutics
Arrhythmogenic Right Ventricular Cardiomyopathy
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.
- Trial name
- RIDGE-1 (NCT06228924)
- Testing
- TN-401
- Where
- 7 sites in United States
- Volunteers
- 15 people
- Sponsor
- Tenaya Therapeutics
Locally Advanced or Metastatic Non-Small Cell Lung Cancer
A Study to Investigate the Efficacy and Safety of Trastuzumab Deruxtecan as the First Treatment Option for Unresectable, Locally Advanced/Metastatic Non-Small Cell Lung Cancer With HER2 Mutations
DESTINY-Lung04 will investigate the efficacy and safety of Trastuzumab Deruxtecan (T-DXd) versus Standard of Care (SoC) as first-line treatment of Non-Small Cell Lung Cancer (NSCLC) with HER2 Exon 19 or 20 mutations
- Trial name
- NCT05048797
- Testing
- Trastuzumab Deruxtecan, Cisplatin, Carboplatin
- Where
- 130 sites in United States, Austria and 18 more
- Volunteers
- 454 people
- Sponsor
- AstraZeneca
Non-Small-Cell Lung Cancer
Efficacy and Safety Evaluation of PLB1004 in Patients With Non-squamous NSCLC Harboring EGFR Exon 20 Insertion.
Efficacy and safety evaluation of PLB1004 in patients with locally advanced/metastatic non-squamous NSCLCharboring EGFR exon 20 insertion.
- Trial name
- KANNON-2 (NCT06281964)
- Testing
- PLB1004, Pemetrexed+（carboplatin or Cisplatin）with or without Sintilimab
- Where
- China
- Volunteers
- 327 people
- Sponsor
- Avistone Biotechnology Co., Ltd.
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Attention Deficit Disorder With Hyperactivity
A Noninterventional Genotype/Phenotype Study of mGluR Mutations in Children and Adolescents With ADHD
This noninterventional study will assess genomic changes in the metabotropic glutamate receptor (mGluR) network in children and adolescents with ADHD.
- Trial name
- NCT02811211
- Where
- United States
- Volunteers
- 1,894 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
Non-small Cell Lung Cancer
Clinical Effectiveness and Safety of Trastuzumab Deruxtecan (T-DXd) in Chinese Patients With HER2m NSCLC (RERUN)
Trastuzumab deruxtecan (T-DXd) is an approved therapy for non-small cell lung cancer (NSCLC) in China. Despite the clinical trial evidence, there is no real-world data of T-DXd used in Chinese lung cancer patients with HER2…
- Trial name
- RERUN (NCT06809764)
- Testing
- No drug
- Where
- 36 sites in China
- Volunteers
- 150 people
- Sponsor
- Daiichi Sankyo
ESR1 Gene Mutation, Advanced Breast Cancer
Safety of Elacestrant in ER+/HER2- and ESR1 Mutations MBC
This study is a prospective non-interventional real-world study enrolling patients with advanced breast cancer who are ER+/HER2- and have ESR1- gene mutations, collecting information on patients' complaints, physical examination…
- Conditions
- ESR1 Gene Mutation, Advanced Breast Cancer, Safety
- Trial name
- NCT06544577
- Testing
- ELacestrant
- Where
- China
- Volunteers
- 350 people
- Sponsor
- SciClone Pharmaceuticals
Advanced or Metastatic Ovarian Cancer
A Study of DAT-2645 in Patients With BRCA1/2 Mutations or HRD-Positive Ovarian Cancer
This is a Phase II, multicenter, single-arm dose expansion trial planned to enroll up to 30 subjects with advanced or metastatic ovarian cancer.
- Trial name
- NCT07652073
- Testing
- DAT-2645 tablet
- Where
- 2 sites in China
- Volunteers
- 30 people
- Sponsor
- Danatlas Pharmaceuticals Co., Ltd
Non-Small Cell Lung Cancer
An epidemiOlogy Study to deteRmine the Prevalence of EGFR (Epidermal Growth Factor Receptor) muTations in RUSsian Patients With Advanced NSCLC (Non-Small Cell Lung Cancer).
This is a multicentre, non-interventional, prospective study to be carried out in representative oncology departments / institutions in order to determine the prevalence of EGFR mutations in treatment-naive Russian patients with…
- Trial name
- ORTUS (NCT02321046)
- Where
- 26 sites in Russia
- Volunteers
- 26 people
- Sponsor
- AstraZeneca
Non-small Cell Lung Cancer (NSCLC), Epidermal Growth Factor Receptor (EGFR) Mutations
A Study of ASP8273 in Subjects With Non-small Cell Lung Cancer (NSCLC) Harboring Epidermal Growth Factor Receptor (EGFR) Mutations
The objective of this study is to evaluate the bioequivalence of a tablet formulation versus a capsule formulation of ASP8273 following a single dose under fasted condition in subjects with non-small cell lung cancer (NSCLC)…
- Trial name
- NCT03082300
- Testing
- naquotinib
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Nonsmall-cell Lung Cancer
Assessing an Oral EGFR Inhibitor, DZD6008 in Patients Who Have Advanced NSCLC With EGFR Mutations (TIAN-SHAN2)
This study is designed to evaluate safety and antitumor activity of DZD6008 in patients with advanced NSCLC with EGFR mutations. This is the first time the drug is tested in human.
- Trial name
- NCT06813365
- Testing
- DZD6008
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Dizal Pharmaceuticals
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA).
- Trial name
- REALM-DCM (NCT03439514)
- Testing
- ARRY-371797 (PF-07265803)
- Where
- 107 sites in United States, Argentina and 8 more
- Volunteers
- 77 people
- Sponsor
- Pfizer