Open AccessCharacteristics and Outcomes of Clinical Trials on Gene Therapy in Noncongenital Cardiovascular Diseases: Cross-sectional Study of Three Clinical Trial Registries
Author(s) -
Witold Pińczak,
Sylwia Trzcińska,
Mikołaj Kamińśki
Publication year - 2022
Publication title -
jmir formative research
Language(s) - English
Resource type - Journals
ISSN - 2561-326X
DOI - 10.2196/33893
Subject(s) - medicine , clinical trial , coronary artery disease , placebo , disease , randomized controlled trial , clinical research , physical therapy , alternative medicine , pathology
Background Cardiovascular diseases remain the leading cause of morbidity and mortality worldwide. Gene therapies (GTs) may become a novel therapeutic option for cardiovascular diseases. Objective We aimed to characterize all trials involving human subjects utilizing GT to treat noncongenital cardiovascular diseases. Methods In March 2021, we searched for clinical trials on the ClinicalTrials.gov (CT), International Clinical Trials Registry Platform (ICTRP), and International Standard Randomised Controlled Trials Number (ISRCTN) databases. Two authors screened the titles and registry notes of all the searched studies. We collected details of the included studies regarding their design, location funding source, treated conditions, completion, publication statuses, and final outcomes. Results We generated a total of 3508 records, and 50 unique clinical trials met our eligibility criteria. Of these, 20 (40%) concerned peripheral artery disease, and 18 (36%) concerned coronary artery disease. Most studies were randomized (34/50, 68%) and were performed in multiple locations (30/50, 60%), and around half of the trials compared GT with a placebo (27/50, 54%), while one in four were single-arm (14/50, 28%), and the rest concerned dose-finding (22%). More than half of the trials (29/50, 58%) were funded by industry. Of the 50 clinical trials, 28 (56%) published their results by the data collection date (March 2021), and 22 of 31 (71%) were slated to be completed before 2021. Overall, 12 of 28 (42.9%) clinical trials showed favorable outcomes of the intervention. Conclusions Among noncongenital cardiovascular diseases, GTs are mostly investigated in peripheral artery disease and coronary artery disease. Many clinical trials on GT use in noncongenital cardiovascular diseases did not disclose their results. Regardless of the trial phase, less than half of published studies on GT in noncongenital cardiovascular diseases showed promising results.