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

NCT05143957 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: ISIS 702843-CS42021-003704-40
• Study Type: interventional
• Target: 50
• Locations: 5 countries
• Sites: 13

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.

Conditions

Phlebotomy Dependent Polycythemia Vera

Keywords

Polycythemia Vera

Outcome Measures

Primary Outcomes (1)

• Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period

  Week 17 to Week 37

Secondary Outcomes (2)

• 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

  Week 17 to Week 37

• Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37

  Baseline up to Week 37

Study Arms (2)

Sapablursen Dose Level 1

EXPERIMENTAL

Sapablursen will be administered by SC injection every 4 weeks.

Drug: sapablursen

Sapablursen Dose Level 2

EXPERIMENTAL

Sapablursen will be administered by SC injection every 4 weeks

Drug: sapablursen

Interventions

sapablursen DRUG

Sapablursen will be administered by SC injection.

Also known as: ISIS 702843, IONIS-TMPRSS6-LRx

Sapablursen Dose Level 1; Sapablursen Dose Level 2

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis
• Participant must be phlebotomy dependent.
• 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 may not qualify if:

• Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)
• Moderate to severe splenic pain or spleen-related organ obstruction
• Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)
• Known primary or secondary immunodeficiency
• Active infection with human immunodeficiency virus (HIV), hepatitis C, or hepatitis B.
• Active infection requiring systemic antiviral or antimicrobial therapy or active novel coronavirus disease (Covid-19) infection
• 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

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (13)

O'Neal Comprehensive Cancer Center University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States

Location

Mayo Clinic Hospital

Phoenix, Arizona, 85054, United States

Location

John Theurer Cancer Center

Hackensack, New Jersey, 07601, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Calvary Mater Newcastle Hospital

Waratah, New South Wales, 2298, Australia

Location

Border Medical Oncology Research Unit

Albury, 2640, Australia

Location

McGill University Health Centre

Montreal, Quebec, H4A 3J1, Canada

Location

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

Lublin, 20-081, Poland

Location

Wojewódzki Szpital Specjalistyczny Sp. z o.o.

Słupsk, 76-200, Poland

Location

MICS Centrum Medyczne Toruń

Torun, 87-100, Poland

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, England, OX37LE, United Kingdom

Location

University College London Hospitals NHS Foundation Trust

London, NW1 2PG, United Kingdom

Location

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2021
• First Posted: December 3, 2021
• Study Start: December 30, 2021
• Primary Completion: June 26, 2025
• Study Completion (Estimated): June 1, 2026
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

CA (1); GB (2); AU (2); PL (3); US (5)