JMIR Publications

ABSTRACT

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 non-congenital cardiovascular diseases.

Methods:

In March 2021, we search for clinical trials in ClinicalTrials.gov, International Clinical Trials Registry Platform, and International Standard Randomised Controlled Trials Number databases. Two authors screened titles and registry notes of all searched studies. We collected from the included study details about the design, location funding source, treated conditions, completion, publication statuses, and final outcomes.

Results:

We generated a total of 3,508 records, and 50 unique clinical trials met our eligibility criteria. Of them, 20 (40.0%) concerned peripheral artery disease and 18 (36.0%) coronary artery disease. Most studies were randomized (68.0%) and were performed in multiple places (61.8%), and around half of the trials compared GT with placebo (54.0%), while one in four were single-arm (28.0%) and the rest concerned dose-finding (22.00%). More than half of the trials (58.0%) were funded by industry. 28 of 50 (56.0%) clinical trials published their results to the data collection date (March 2021), and 22 of 31 (71.0%) that planned to be completed before 2021. Overall, 12/28 (42.9%) clinical trials showed favorable outcomes of the intervention.

Conclusions:

Among non-congenital cardiovascular diseases, GTs are mostly investigated in PAD and CAD. The significant number of clinical trials on GT use in non-congenital cardiovascular diseases are not published. Regardless of the trial phase, less than half of published studies on GT in non-congenital cardiovascular diseases showed promising results.