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

Last updated: April 23, 2025
Sponsor: Ionis Pharmaceuticals, Inc.
Overall Status: Active - Not Recruiting

Phase

2

Condition

Leukemia (Pediatric)

Bone Marrow Disorder

Red Blood Cell Disorders

Treatment

sapablursen

Clinical Study ID

NCT05143957
ISIS 702843-CS4
2021-003704-40
  • Ages 18-90
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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.

  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.

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

  8. Surgery requiring general anesthesia within 1 month prior to Screening

Study Design

Total Participants: 50
Treatment Group(s): 1
Primary Treatment: sapablursen
Phase: 2
Study Start date:
December 30, 2021
Estimated Completion Date:
June 30, 2026

Study 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.

Connect with a study center

  • Blacktown Hospital

    Blacktown, New South Wales 2148
    Australia

    Site Not Available

  • St. George Hospital

    Kogarah, New South Wales 2217
    Australia

    Site Not Available

  • Calvary Mater Newcastle Hospital

    Waratah, New South Wales 2298
    Australia

    Site Not Available

  • Border Medical Oncology Research Unit

    Albury, 2640
    Australia

    Site Not Available

  • Box Hill Hospital

    Box Hill, 3128
    Australia

    Site Not Available

  • The Perth Blood Institute

    West Perth, 6005
    Australia

    Site Not Available

  • McGill University Health Centre

    Montreal, Quebec H4A 3J1
    Canada

    Site Not Available

  • Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika

    Budapest, 1088
    Hungary

    Site Not Available

  • Debreceni Egyetem Klinikai Központ

    Debrecen, 4032
    Hungary

    Site Not Available

  • Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie, Klinika Hematoonkologii i Transplantacji Szpiku

    Lublin, 20-081
    Poland

    Site Not Available

  • Szpital Wojewódzki w Opolu

    Opole, 45-064
    Poland

    Site Not Available

  • Wojewódzki Szpital Specjalistyczny Sp. z o.o.

    Słupsk, 76-200
    Poland

    Site Not Available

  • MICS Centrum Medyczne Toruń

    Toruń, 87-100
    Poland

    Site Not Available

  • Lincoln County Hospital

    Lincoln, England LN2 5QY
    United Kingdom

    Site Not Available

  • United Lincolnshire Hospitals NHS Trust

    Lincoln, England LN2 5QY
    United Kingdom

    Site Not Available

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, England OX37LE
    United Kingdom

    Site Not Available

  • University College London Hospitals NHS Foundation Trust

    London, NW1 2PG
    United Kingdom

    Site Not Available

  • Sandwell and West Birmingham Hospitals NHS Trust

    West Bromwich, B71 4HJ
    United Kingdom

    Site Not Available

  • O'Neal Comprehensive Cancer Center University of Alabama at Birmingham

    Birmingham, Alabama 35249
    United States

    Site Not Available

  • Mayo Clinic Hospital

    Phoenix, Arizona 85054
    United States

    Site Not Available

  • PCR Oncology

    Arroyo Grande, California 93420
    United States

    Site Not Available

  • UCLA Health - Beverly Hills Cancer Care

    Beverly Hills, California 90212
    United States

    Site Not Available

  • UCLA Health - Burbank Cancer Care

    Burbank, California 91505
    United States

    Site Not Available

  • City of Hope National Medical Center

    Duarte, California 91010
    United States

    Site Not Available

  • Norris Comprehensive Cancer Center

    Los Angeles, California 91011
    United States

    Site Not Available

  • UCLA

    Los Angeles, California 90095
    United States

    Site Not Available

  • University of California Irvine

    Orange, California 92868
    United States

    Site Not Available

  • UCLA Health - Pasadena Cancer Care

    Pasadena, California 91105
    United States

    Site Not Available

  • UCLA Health -Westlake Village Cancer Care

    Westlake Village, California 91361
    United States

    Site Not Available

  • Innovative Clinical Research Institute

    Whittier, California 90603
    United States

    Site Not Available

  • Medstar Georgetown University Hospital

    Washington, District of Columbia 20007
    United States

    Site Not Available

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Summit Medical Group

    Florham Park, New Jersey 07932
    United States

    Site Not Available

  • John Theurer Cancer Center

    Hackensack, New Jersey 07601
    United States

    Site Not Available

  • Herbert Irving Comprehensive Cancer Center

    New York, New York 10032
    United States

    Active - Recruiting

  • Icahn School of Medicine at Mount Sinai

    New York, New York 10029
    United States

    Site Not Available

  • Atrium Health Levine Cancer Institute

    Charlotte, North Carolina 28204
    United States

    Site Not Available

  • Atrium Health Wake Forest Baptist

    Winston-Salem, North Carolina 27157
    United States

    Site Not Available

  • Gabrail Cancer Center Research

    Canton, Ohio 44718
    United States

    Site Not Available

  • Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • University of Texas MD Anderson Cancer Center

    Houston, Texas 77030
    United States

    Site Not Available

  • Mays Cancer Center

    San Antonio, Texas 78229
    United States

    Site Not Available

  • Medical College of Wisconsin

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

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