for people ages 18-90 (full criteria)
at Orange, California and other locations
study started
completion around



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.

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)


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.


Phlebotomy Dependent Polycythemia Vera, Polycythemia Vera, Polycythemia, sapablursen


You can join if…

Open to people ages 18-90

  1. Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis
  2. Participant must be phlebotomy dependent.
  3. Participants do not need to be on cytoreductive therapy and do not need to have been previously treated with cytoreductive therapy. If the patient was previously on cytoreductive therapy it must have been discontinued at least 3 months prior to Screening, with all associated AEs resolved. If the patient is currently on cytoreductive therapy they must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months prior to Screening.

You CAN'T join if...

  1. Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)
  2. Moderate to severe splenic pain or spleen-related organ obstruction
  3. Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)
  4. Known primary or secondary immunodeficiency
  5. Active infection with human immunodeficiency virus (HIV), hepatitis C, or hepatitis B.
  6. Active infection requiring systemic antiviral or antimicrobial therapy or active novel coronavirus disease (Covid-19) infection
  7. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or non-metastatic prostate cancer that has been successfully treated
  8. Surgery requiring general anesthesia within 1 month prior to Screening


  • University of California Irvine accepting new patients
    Orange California 92868 United States
  • Innovative Clinical Research Institute accepting new patients
    Whittier California 90603 United States
  • UCLA accepting new patients
    Los Angeles California 90095 United States


accepting new patients
Start Date
Completion Date
Ionis Pharmaceuticals, Inc.
Phase 2 Polycythemia Vera Research Study
Study Type
Expecting 60 study participants
Last Updated