Clinical trials: Gene
Neoplasms Malignant
Studying An Investigational Drug Crizotinib (PF-02341066) In Non Non-Small Cell Lung Cancer Tumors That Are Positive For Anaplastic Lymphoma Kinase (ALK)
This is a Phase 1 trial evaluating the safety and efficacy of crizotinib in patients with tumors except non-small cell lung cancer that are positive for ALK.
- Trial name
- NCT01121588
- Testing
- Crizotinib
- Where
- 21 sites in United States, China and 5 more
- Volunteers
- 44 people
- Sponsor
- Pfizer
Mucopolysaccharidosis Type II (MPS II)
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system.
- Trial name
- NCT04571970
- Testing
- RGX-121
- Where
- 2 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- REGENXBIO Inc.
Gastric Cancer, Esophagogastric Junction Disorder
Savolitinib for Treating Gastric Cancer and Esophagogastric Junction Adenocarcinoma Patients
Treating Gastric Cancer and Esophagogastric junction adenocarcinoma Patients with MET gene amplifications with Savolitinib
- Trial name
- NCT04923932
- Testing
- Savolitinib
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Hutchison Medipharma Limited
Phenylketonuria (PKU)
To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.
- Trial name
- NCT07318909
- Testing
- GS1168 injection
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Gritgen Therapeutics Co., Ltd.
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Long-Term Follow-up Protocol
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- NCT04917887
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Krystal Biotech, Inc.
RAS Mutation, NRAS Gene Mutation
NAUTILUS: OKI-179 Plus Binimetinib in Patients with Advanced Solid Tumors in the RAS Pathway (Phase 1b) and NRAS-mutated Melanoma (Phase 2)
The NAUTILUS study is a Phase 1b/2, multi-center, open-label study in which patients with activating mutations in the RAS pathway (Phase 1b) and patients with NRAS-mutated Melanoma (Phase 2) will be treated with a combination of…
- Conditions
- RAS Mutation, NRAS Gene Mutation, Melanoma
- Trial name
- NCT05340621
- Testing
- OKI-179 + binimetinib
- Where
- 12 sites in United States
- Volunteers
- 36 people
- Sponsor
- OnKure, Inc.
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
Hereditary Spastic Paraplegia Type 50
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
- Trial name
- SPG50 (NCT06692712)
- Testing
- MELPIDA
- Where
- 2 sites in United States, Spain
- Volunteers
- 24 people
- Sponsor
- Elpida Therapeutics SPC
Usage of Prescription Opioids
Determining Genetic Biomarkers Predictive of the Development of Prescription Opioid Addiction
This is a clinical study on patients who have been prescribed opioids (narcotic pain relievers) for the treatment of pain, to determine what genetic factors influence outcome of treatment.
- Trial name
- NCT01562353
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Analgesic Solutions
Maxillofacial Bone Defects, Alveolar Bone Atrophy
Gene-activated Matrix for Bone Tissue Repair in Maxillofacial Surgery
The purpose of this study is to evaluate safety and efficacy of gene-activated matrix ("Nucleostim") for regeneration of bone tissue in maxillofacial area.
- Conditions
- Maxillofacial Bone Defects, Alveolar Bone Atrophy, Jaw Fractures, Maxillofacial Bone Deformities
- Trial name
- NCT02293031
- Testing
- Gene-activated matrix "Nucleostim"
- Where
- 2 sites in Russia
- Volunteers
- 12 people
- Sponsor
- NextGen Company Limited
Leber Congenital Amaurosis
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
- Trial name
- NCT00749957
- Testing
- rAAV2-CB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Beacon Therapeutics
HER2 Gene Mutation
To Evaluate IAH0968 in Combination With CAPEOX in HER2-positive Metastatic Colorectal Cancer
The Phase IIa of this clinical study, a dose-escalation study of IAH0968 in combination with CAPEOX, is designed for safety and tolerability in subjects with HER2-positive advanced or metastatic solid tumors.
- Trial name
- NCT05673512
- Testing
- Injection of IAH0968 + CAPEOX
- Where
- China
- Volunteers
- 279 people
- Sponsor
- SUNHO（China）BioPharmaceutical CO., Ltd.
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
- Trial name
- NCT05885412
- Testing
- RP-A601
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Retinitis Pigmentosa, Leber Congenital Amaurosis
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101).
- Trial name
- NCT05203939
- Testing
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose
- Where
- 7 sites in United States
- Volunteers
- 22 people
- Sponsor
- Ocugen
Glioma
Study of DS-1001b in Patients With Gene IDH1-Mutated Gliomas
This is a study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of DS-1001b in patients with gliomas that harbor IDH1-R132 mutations.
- Trial name
- NCT03030066
- Testing
- DS-1001b
- Where
- Japan
- Volunteers
- 47 people
- Sponsor
- Daiichi Sankyo
Tumors Harboring NTRK Fusion
Expanded Access to Provide Larotrectinib for the Treatment of Cancers With a NTRK Gene Fusion
Larotrectinib expanded access is for patients with cancer with a NTRK1, NTRK2, or NTRK3 gene fusion, who are ineligible for an ongoing larotrectinib clinical trial or have other considerations that prevent access to larotrectinib…
- Trial name
- NCT03025360
- Testing
- Larotrectinib (Vitrakvi, BAY2757556)
- Sponsor
- Bayer
Bladder Cancer, Urocystitis
A Multicenter Clinical Trial of Urine DNA Testing for Bladder Cancer in China
The primary objective of this study is to assess the safety and effectiveness of Human Multigene Methylation Detection Kit (Fluorescent PCR Method) for help diagnose bladder cancer by comparing with clinical standard method…
- Conditions
- Bladder Cancer, Urocystitis, Urinary Bladder Stone, Urothelial Carcinoma
- Trial name
- NCT05337189
- Testing
- Urine DNA methylation analysis, medical imaging and/or cystoscopy and/or pathological examination
- Where
- China
- Volunteers
- 482 people, healthy volunteers welcome
- Sponsor
- Creative Biosciences (Guangzhou) Co., Ltd.
Critical Limb Ischemia, Vascular Diseases
A Phase IIB Pilot Study of a Modified Dosage Regimen of AMG0001 in Subjects With Critical Limb Ischemia
The purpose of the study is to confirm the feasibility of study procedures and the tolerability of a new dose regimen of AMG0001 in subjects with Critical Limb Ischemia (CLI)
- Conditions
- Critical Limb Ischemia, Vascular Diseases, Peripheral Arterial Disease
- Trial name
- NCT02016755
- Testing
- HGF Plasmid
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- AnGes USA, Inc.
Non-Small Cell Lung Cancer
Study of MCLA-129 in the Treatment of Advanced Non-small Cell Lung Cancer with AGA and MET Amplification.
This is a multi-center, open-label, phase II clinical study of MCLA-129 as monotherapy in patients with advanced non-small cell lung cancer (NSCLC) with actionable gene alterations and MET amplification to evaluate the efficacy…
- Trial name
- NCT06885840
- Testing
- MCLA-129
- Where
- 20 sites in China
- Volunteers
- 100 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
NTRK Family Gene Mutation
Registry for Molecular Testing, Treatment and Outcome of Patients With Solid Tumors Harboring a NTRK Gene Fusion
The objective of this registry is to analyze treatment reality and outcome of patients with locally advanced or metastatic solid tumors harboring a fusion of NTRK1, NTRK2 or NTRK3
- Trial name
- REALTRK (NCT04557813)
- Where
- 26 sites in Germany, Switzerland
- Volunteers
- 88 people
- Sponsor
- iOMEDICO AG