Summary

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

Description

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.

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)

Details

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 10 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.

Keywords

Phlebotomy Dependent Polycythemia Vera, Polycythemia Vera, Polycythemia, sapablursen

Eligibility

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

Locations

  • 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

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT05143957
Phase
Phase 2 Polycythemia Vera Research Study
Study Type
Interventional
Participants
Expecting 40 study participants
Last Updated