NCT05143957

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

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)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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 4 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 37 weeks 3) Treatment Extension Period: 36 weeks; 4) 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 9 doses given over about 8 months. In the Treatment Extension Period, there will be a total of 9 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. This study was extended to allow participants to receive sapablursen for an additional 36 weeks following the initial 37-week treatment period.

Conditions

• Phlebotomy Dependent Polycythemia Vera

Interventions

Type	Name	Description
DRUG	sapablursen	Sapablursen will be administered by SC injection.

Timeline

• Start date: 2021-12-30
• Primary completion: 2025-06-26
• Completion: 2026-06-01
• First posted: 2021-12-03
• Last updated: 2026-01-16

Locations

13 sites across 5 countries: United States, Australia, Canada, Poland, United Kingdom

Regulatory

• FDA-regulated drug study