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

February 23, 2024 updated by: Ionis Pharmaceuticals, Inc.

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
      • Albury, Australia, 2640
        • Recruiting
        • Border Medical Oncology Research Unit
      • Box Hill, Australia, 3128
        • Recruiting
        • Box Hill Hospital
      • West Perth, Australia, 6005
        • Recruiting
        • The Perth Blood Institute
    • New South Wales
      • Kogarah, New South Wales, Australia, 2217
        • Recruiting
        • St. George Hospital
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H4A 3J1
        • Recruiting
        • Mcgill University Health Centre
  • Hungary
      • Budapest, Hungary, 1088
        • Recruiting
        • Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika
      • Debrecen, Hungary, 4032
        • Recruiting
        • Debreceni Egyetem Klinikai Kozpont
  • Poland
      • Lublin, Poland, 20-081
        • Recruiting
        • Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie, Klinika Hematoonkologii i Transplantacji Szpiku
      • Opole, Poland, 45-064
        • Recruiting
        • Szpital Wojewodzki w Opolu
      • Słupsk, Poland, 76-200
        • Recruiting
        • Wojewódzki Szpital Specjalistyczny Sp. z o.o.
      • Toruń, Poland, 87-100
        • Recruiting
        • MICS Centrum Medyczne Torun
  • United Kingdom
      • London, United Kingdom, NW1 2PG
        • Recruiting
        • University College London Hospitals NHS Foundation Trust
      • West Bromwich, United Kingdom, B71 4HJ
        • Recruiting
        • Sandwell and West Birmingham Hospitals NHS Trust
    • England
      • Lincoln, England, United Kingdom, LN2 5QY
        • Recruiting
        • Lincoln County Hospital
      • Lincoln, England, United Kingdom, LN2 5QY
        • Recruiting
        • United Lincolnshire Hospitals NHS Trust
      • Oxford, England, United Kingdom, OX37LE
        • Recruiting
        • Oxford University Hospitals NHS Foundation Trust
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35249
        • Recruiting
        • O'Neal Comprehensive Cancer Center University of Alabama at Birmingham
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Recruiting
        • Mayo Clinic Hospital
    • California
      • Arroyo Grande, California, United States, 93420
        • Recruiting
        • PCR Oncology
      • Beverly Hills, California, United States, 90212
        • Recruiting
        • UCLA Health - Beverly Hills Cancer Care
      • Burbank, California, United States, 91505
        • Recruiting
        • UCLA Health - Burbank Cancer Care
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope National Medical Center
      • Los Angeles, California, United States, 90095
        • Recruiting
        • UCLA
      • Los Angeles, California, United States, 91011
        • Recruiting
        • Norris Comprehensive Cancer Center
      • Orange, California, United States, 92868
        • Recruiting
        • University of California Irvine
      • Pasadena, California, United States, 91105
        • Recruiting
        • UCLA Health - Pasadena Cancer Care
      • Westlake Village, California, United States, 91361
        • Recruiting
        • UCLA Health -Westlake Village Cancer Care
      • Whittier, California, United States, 90603
        • Recruiting
        • Innovative Clinical Research Institute
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Recruiting
        • MedStar Georgetown University Hospital
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
    • New Jersey
      • Florham Park, New Jersey, United States, 07932
        • Recruiting
        • Summit Medical Group
      • Hackensack, New Jersey, United States, 07601
        • Recruiting
        • John Theurer Cancer Center
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Icahn School of Medicine at Mount Sinai
      • New York, New York, United States, 10032
        • Recruiting
        • Herbert Irving Comprehensive Cancer Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Atrium Health Levine Cancer Institute
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Atrium Health Wake Forest Baptist
    • Ohio
      • Canton, Ohio, United States, 44718
        • Recruiting
        • Gabrail Cancer Center Research
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas MD Anderson Cancer Center
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • Mays Cancer Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 78 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Sapablursen Dose Level 1
Sapablursen will be administered by SC injection every 4 weeks.
Drug: sapablursen
Sapablursen will be administered by SC injection.
Other Names:
  • ISIS 702843
  • IONIS-TMPRSS6-LRx
Experimental: Sapablursen Dose Level 2
Sapablursen will be administered by SC injection every 4 weeks
Drug: sapablursen
Sapablursen will be administered by SC injection.
Other Names:
  • ISIS 702843
  • IONIS-TMPRSS6-LRx

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period
Time Frame: Week 17 to Week 37
Week 17 to Week 37

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of patients achieving a reduction in the frequency of phlebotomy by ≥ 30%, ≥ 50%, ≥ 75% and ≥ 90% comparing Baseline with the last 20 weeks of the 37-week Treatment Period
Time Frame: Week 17 to Week 37
Week 17 to Week 37
Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37
Time Frame: Baseline up to Week 37
Baseline up to Week 37

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ionis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 30, 2021

Primary Completion (Estimated)

January 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

November 20, 2021

First Submitted That Met QC Criteria

November 20, 2021

First Posted (Actual)

December 3, 2021

Study Record Updates

Last Update Posted (Actual)

February 28, 2024

Last Update Submitted That Met QC Criteria

February 23, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISIS 702843-CS4
  • 2021-003704-40 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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