Project Title

A study to evaluate Sapablursen (formerly ISIS 702843, IONIS-TMPRSS6-LRx) in patients with polycythemia vera

Official Title

A phase 2a, randomized, open-label study to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of ISIS 702843 administered to patients with phlebotomy dependent polycythemia vera (PD-PV).

Project Summary

The main purpose of this study is to evaluate the efficacy of sapablursen 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 sapablursen in up to 40 participants with PD-PV. 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.

Blood Disorder

• Phlebotomy Dependent Polycythemia Vera

Patient Recruitment Details

Patient recruitment status: OPEN

Number of patients (Globally)

40