The results of a multi-center clinical trial on the safety and efficacy of volanesorsen – an antisense drug designed to treat hypertriglyceridemia – were recently published in the New England Journal of Medicine (NEJM). The study was managed by the Clinical Trial Management Group (CTMG).
Volanesorsen was developed by Isis Pharmaceuticals and its effects were studied in a clinical trial which was managed by CTMG. CTMG is the world’s first site-specific research organization (SSRO) offering protocol-specific processes, internal quality systems and strategies for continued patient participation.
The potential treatment drug volanesorsen, is a selective inhibitor of the synthesis of apolipoprotein C-111 (APO3), and uses RNA-targeted therapy to inhibit production. APO3 is responsible for the regulation of triglyceride levels in blood plasma.
Patients with hypertriglyceridemia display extremely elevated levels of triglycerides and may have an increased risk of pancreatitis and cardiovascular disease if left untreated. The results from the Phase 2 clinical trial conducted by CTMG and Isis Pharmaceuticals showed volanesorsen was successful in significantly lowering plasma triglyceride levels in a range of patients.
Despite a patient’s baseline triglyceride level, most showed a significant decrease after treatment with volanesorsen. The average inhibition level of APO3 was up to 80 percent while the average reduction in triglyceride levels was 71 percent. No safety concerns over the novel drug were identified and the success of this Phase 2 study has enabled Phase 3 trials to commence.
- NEJM Publishes Positive Clinical Data on Treatment for Hypertriglyceridemia – http://www.appliedclinicaltrialsonline.com/nejm-publishes-positive-clinical-data-treatment-hypertriglyceridemia#_ftn1
- Gaudet, D., Alexander, V., Baker, B., Brissen, D., Tremblay, K., Singleton, W., Geary, R., Hughes, S., Viney, N., Graham, M., et al. (2015). Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia. New Eng. J. Med.