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

NCT ID: NCT05143957

Last Updated: 2026-01-16

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-30
• Study Completion Date: 2026-06-30

Brief 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.

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.

Conditions

Phlebotomy Dependent Polycythemia Vera

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Sapablursen Dose Level 1

Sapablursen will be administered by SC injection every 4 weeks.

Group Type: EXPERIMENTAL

sapablursen

Intervention Type: DRUG

Sapablursen will be administered by SC injection.

Sapablursen Dose Level 2

Sapablursen will be administered by SC injection every 4 weeks

Group Type: EXPERIMENTAL

sapablursen

Intervention Type: DRUG

Sapablursen will be administered by SC injection.

Interventions

sapablursen

Sapablursen will be administered by SC injection.

Intervention Type: DRUG

Other Intervention Names

ISIS 702843; IONIS-TMPRSS6-LRx

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

O'Neal Comprehensive Cancer Center University of Alabama at Birmingham

Birmingham, Alabama, United States

Mayo Clinic Hospital

Phoenix, Arizona, United States

John Theurer Cancer Center

Hackensack, New Jersey, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Calvary Mater Newcastle Hospital

Waratah, New South Wales, Australia

Border Medical Oncology Research Unit

Albury, , Australia

McGill University Health Centre

Montreal, Quebec, Canada

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

Lublin, , Poland

Wojewódzki Szpital Specjalistyczny Sp. z o.o.

Słupsk, , Poland

MICS Centrum Medyczne Toruń

Torun, , Poland

Oxford University Hospitals NHS Foundation Trust

Oxford, England, United Kingdom

University College London Hospitals NHS Foundation Trust

London, , United Kingdom

Countries

United States; Australia; Canada; Poland; United Kingdom

Other Identifiers

2021-003704-40

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ISIS 702843-CS4

Identifier Type: -

Identifier Source: org_study_id