Lay Description

This is a Phase 2a, multi-center, randomized, open-label study of IONIS-TMPRSS6-LRx 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.


  • Cancers and Other Neoplasms
IRB Number
NCT Number registration not required
Open to Enrollment


Eligible Ages
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

  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

The participant's cytoreductive therapy must either be discontinued at least 3 months prior to Screening, OR participant must be on a stable dose for at least 3 months prior to Screening

Exclusion Criteria

  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

Surgery requiring general anesthesia within 1 month prior to Screening

Study Design

Arm Groups

Study Contact

Regulatory Point of Contact
Frances Crawford
(210) 450-5037

Regulatory Point of Contact
Sonia Creighton
(210) 450-1366

Regulatory Point of Contact
Myrna Montenegro
(210) 450-5954

Regulatory Point of Contact
Mailbox Ctrc Regulatory Affairs

Regulatory Point of Contact
Kathleen Rodriguez
(210) 450-1365

Regulatory Point of Contact
Benjamin Schleif
(210) 450-1366

Regulatory Point of Contact
Morgan Seekatz
(210) 450-1133

Principal Investigator
Ruben Mesa