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.
Arrhythmogenic Cardiomyopathy, PKP2-ACM
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Trial name
- SNAPSHOT- PKP2 (NCT06976606)
- Where
- 6 sites in United States
- Volunteers
- 40 people
- Sponsor
- Lexeo Therapeutics
Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations.
- Conditions
- Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1
- Trial name
- NCT05735184
- Testing
- Ziftomenib, Venetoclax, Azacitidine
- Where
- 44 sites in United States
- Volunteers
- 420 people
- Sponsor
- Kura Oncology, Inc.
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
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.
HEPATITIS B CHRONIC
Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients With Chronic Hepatitis B
This is a Phase 1, open-label, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, and antiviral activity of PBGENE-HBV in adult participants with chronic hepatitis B.
- Trial name
- ELIMINATE-B (NCT06680232)
- Testing
- PBGENE-HBV
- Where
- 7 sites in United States, France and 4 more
- Volunteers
- 45 people
- Sponsor
- Precision BioSciences, 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
Alveolar Bone Loss, Alveolar Ridge Pathology | Gingiva | Dental
Comparative Study of Gene-activated Bone Substitute and Xenogenic Bone Matrix in Alveolar Ridge Augmentation
The study aim is to compare the effectiveness of two bone substitutes, the gene-activated bone substitute based on octacalcium phosphate and plasmid DNA encoding VEGFA gene and xemogenic deproteinized bone matrix, mixed with…
- Trial name
- NCT04511689
- Testing
- gene-activated bone substitute mixed with autobone
- Where
- Russia
- Volunteers
- 24 people
- Sponsor
- Histograft Co., Ltd.
Solid Tumor
A Study of Gene Edited Autologous Neoantigen Targeted TCR T Cells With or Without Anti-PD-1 in Patients With Solid Tumors
This is a first in human, single arm, open label, Phase 1a/1b study to determine the safety, feasibility, and efficacy of a single dose of NeoTCR-P1 T cells in participants with solid tumors.
- Trial name
- NCT03970382
- Testing
- NeoTCR-P1 adoptive cell therapy, nivolumab, IL-2
- Where
- 9 sites in United States
- Volunteers
- 21 people
- Sponsor
- PACT Pharma, Inc.
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
FGFR2 Gene Fusion/Rearrangement, Other Solid Tumors, Adult
A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement
The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma)…
- Trial name
- ReFocus202 (NCT07359820)
- Testing
- Lirafugratinib
- Where
- 19 sites in United States, France and 3 more
- Volunteers
- 30 people
- Sponsor
- Elevar Therapeutics
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
Arrhythmia, Cardiovascular Diseases
Diagnostic Investigation of Sudden Cardiac Event Risk
The overall objective of the DISCERN study is to develop and validate a genomic diagnostic assay to identify patients at increased risk for lethal ventricular arrhythmias and sudden cardiac death (SCD).
- Conditions
- Arrhythmia, Cardiovascular Diseases, Heart Failure, CVD
- Trial name
- DISCERN (NCT00500708)
- Where
- 8 sites in United States
- Volunteers
- 1,564 people
- Sponsor
- CardioDx
Inherited Retinal Dystrophy Due to RPE65 Mutations
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject.
- Trial name
- NCT03602820
- Testing
- AAV2-hRPE65v2
- Volunteers
- 41 people
- Sponsor
- Genentech, Inc.
Parkinson's Disease
Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD
The purpose of this randomized, double-blind, placebo-controlled study is to assess the efficacy of BIA 28-6156 over placebo in delaying clinical meaningful motor progression over 78 weeks in subjects with Parkinson's disease who…
- Trial name
- ACTIVATE (NCT05819359)
- Testing
- BIA 28-6156 10 mg, BIA 28-6156 60 mg
- Where
- 95 sites in United States, Canada and 9 more
- Volunteers
- 237 people
- Sponsor
- Bial R&D Investments, S.A.
HIV Infections
Safety of an HIV Vaccine (AVX101) in HIV Uninfected Volunteers in the United States and South Africa
The purpose of this study is to see if different doses of an experimental HIV vaccine are safe and to study how the immune system responds to the vaccine. The vaccine will be tested in healthy, HIV uninfected volunteers.
- Trial name
- NCT00063778
- Testing
- AVX101
- Where
- 7 sites in United States, South Africa
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- AlphaVax, Inc.
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
HIV Infection
Safety and Efficacy of T Cell Genetic Immunotherapy for HIV
This study uses autologous (one's own) CD4 T cells modified with a viral vector expressing a genetic antisense targeting HIV, this vector is called VRX496.
- Trial name
- NCT00131560
- Testing
- VRX496-Modified Autologous T cells
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- VIRxSYS Corporation
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