Ionis Pharmaceuticals is the sponsor.
Sapablursen, formerly known as IONIS-TMPRSS6-LRx in phase 2 clinical trial.
The main purpose of this study is to evaluate the safety and efficacy of IONIS-TMPRSS6-LRx in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.
This is a Phase 2a, multi-center, randomized, open-label study of IONIS-TMPRSS6-LRx in up to 40 participants with phlebotomy-dependent polycythemia vera. The study consists of 3 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 33 weeks + a 4-week post-last dose visit; 3) Post-treatment Period: 13 weeks.
In the Treatment Period, study drug is given by subcutaneous (under the skin) injection(s). There will be a total of 10 doses given over about 8 months.
Participants will be assigned to receive one of 2 Dosing Levels - a higher or a lower level, with an equal chance of being assigned to either Dosing Level. All participants will receive study drug; there is no placebo.
Click here to learn more about this clinical trial for Polycythemia Vera (PV).