Clinical trials: Gene
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Clostridium Difficile Infection
Validation of the GenePOC CDiff Assay for the Detection of the Toxin B Gene From Toxigenic Clostridium Difficile Strains
The primary purpose of this clinical investigation is to verify the performance of the GenePOC CDiff test on the GenePOC instrument.
- Trial name
- NCT02968758
- Testing
- Comparison between GenePOC PCR and Reference Method
- Where
- 7 sites in United States, Canada
- Volunteers
- 2,461 people
- Sponsor
- Meridian Bioscience, Inc.
HRAS Gene Mutation, HNSCC
Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy
An international, multicenter, open-label, 2 cohort, non-comparative, pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC (AIM-HN).
- Trial name
- AIM-HN/SEQ-HN (NCT03719690)
- Testing
- Tipifarnib, HRAS Detection Assay
- Where
- 102 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Kura Oncology, Inc.
Merkel Cell Carcinoma
IL-12 Gene and in Vivo Electroporation-Mediated Plasmid DNA Vaccine Therapy in Patients With Merkel Cell Cancer
This phase II trial studies the effectiveness of ImmunoPulse IL-12® in treating patients with Merkel cell cancer. ImmunoPulse IL-12® is the combination of intratumoral interleukin-12 gene (also known as tavokinogene telseplasmid…
- Trial name
- MCC (NCT01440816)
- Testing
- Tavokinogene Telseplasmid (tavo), OncoSec Medical System (OMS)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- OncoSec Medical Incorporated
Pneumonia
Detection of Microorganisms and Antibiotic Resistance Genes Using the Curetis Unyvero LRT55 Application
The Curetis Unyvero LRT55 Application is intended to detect and to identify genes of 21 microorganisms and 19 genes associated with antibiotic resistance in 4 hours.
- Trial name
- LRT55 (NCT01922024)
- Testing
- LRT55 Testing
- Where
- 9 sites in United States
- Volunteers
- 2,124 people
- Sponsor
- Curetis GmbH
Chronic Myeloid Leukemia in Chronic Phase
Efficacy and Safety of LBH589 in Adult Patients With Refractory Chronic Myeloid Leukemia (CML) in Chronic Phase
This study will evaluate the efficacy and safety of LBH589B in adult patients with chronic phase chronic myeloid leukemia with resistant disease following treatment with at least two BCR-ABL tyrosine kinase inhibitors
- Trial name
- NCT00451035
- Testing
- LBH589
- Where
- 10 sites in Belgium, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
FGFR Inhibition, Pharmacokinetics, Biomarkers, ER+ Breast Cancer
Safety and Efficacy of AZD4547 in Combination With Fulvestrant vs. Fulvestrant Alone in ER+ Breast Cancer Patients
The purpose of this study is to assess the safety and effectiveness of AZD4547 in combination with fulvestrant vs. fulvestrant alone in ER+ breast cancer patients with FGFR1 polysomy (FISH4/5) or gene amplification (FISH 6)
- Trial name
- GLOW (NCT01202591)
- Testing
- AZD4547, Exemestane, Fulvestrant
- Where
- 21 sites in Belgium, Czechia and 6 more
- Volunteers
- 127 people
- Sponsor
- AstraZeneca
Progressive Metastatic Malignancies
Safety and Efficacy Study of ALT-801 to Treat Progressive Metastatic Malignancies
This is a Phase 1, open-labeled, non-randomized, multi-center, competitive enrollment and dose-escalation study of ALT-801, the study drug.
- Trial name
- NCT00496860
- Testing
- ALT-801
- Where
- 4 sites in United States
- Volunteers
- 26 people
- Sponsor
- Altor BioScience
AADC Deficiency
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of…
- Trial name
- NCT04903288
- Testing
- Eladocagene Exuparvovec
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Painful Diabetic Neuropathy, Diabetic Neuropathy, Painful
Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy
The purpose of this study is to explore the overall safety profile and durability of efficacy of Engensis (VM202) in painful diabetic peripheral neuropathy.
- Trial name
- NCT04055090
- Testing
- Long-Term Follow-Up of Patients who Received Engensis (VM202)
- Where
- 14 sites in United States
- Volunteers
- 101 people
- Sponsor
- Helixmith Co., Ltd.
Glycogen Storage Disease Type IA
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose…
- Trial name
- NCT05139316
- Testing
- DTX401, Oral prednisolone
- Where
- 20 sites in United States, Brazil and 7 more
- Volunteers
- 52 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia B
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02618915
- Testing
- DTX101
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Actinic Keratosis
Gene Expression in Renal Transplant Patients With Field Actinic Keratosis Undergoing Metvix® Photodynamic Therapy (PDT)
The aim of this study is to determine possible molecular changes on large scale gene expression profiling after treatment with Metvix photodynamic therapy (PDT) of actinic keratoses (AK) and cancerised field in renal transplant…
- Trial name
- NCT01000636
- Testing
- Metvix PDT
- Where
- United Kingdom
- Volunteers
- 9 people
- Sponsor
- Galderma R&D
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
NSCLC (Non-small Cell Lung Cancer)
A Study Of Crizotinib Versus Chemotherapy In Previously Untreated ALK Positive East Asian Non-Small Cell Lung Cancer Patients
This is a Phase III, Randomized, Open-label, Efficacy and Safety Study of Crizotinib single agent versus Chemotherapy Regimens (Pemetrexed/Cisplatin or Pemetrexed/Carboplatin) in First-Line ALK (Anaplastic Lymphoma Kinase)…
- Trial name
- NCT01639001
- Testing
- Crizotinib, Pemetrexed/Cisplatin, Pemetrexed/Carboplatin
- Where
- 47 sites in China, Hong Kong and 3 more
- Volunteers
- 207 people
- Sponsor
- Pfizer
Parkinson's Disease
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of…
- Trial name
- NCT05603312
- Testing
- AAV-GAD Low Dose, AAV-GAD High Dose
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- MeiraGTx, LLC
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
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