NCT05143957CompletedPhase 2
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
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)
Source: ClinicalTrials.gov · Last updated 9 Jul 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 50 (actual)
- First submitted
- 20 Nov 2021
- Start date
- 30 Dec 2021
- Primary completion date
- 26 Jun 2025
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Diseases
- Myeloproliferative neoplasms
- NCT number
- NCT05143957
- EU CT number
- 2024-512482-14-00
- EudraCT number
- 2021-003704-40
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 50
- From 30 Dec 2021 to the primary completion date, 26 Jun 2025
- 14 sites in 5 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)
- Surgery requiring general anesthesia within 1 month prior to Screening
Other illness
- 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
Other
- 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
- Known primary or secondary immunodeficiency
Who the study is looking for
- 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.
Age in the registry: 18 years to 90 years · Sex: All · Does not accept healthy volunteers
Sites (14)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Border Medical Oncology Research Unit Coordinator of this site? Confirm its status | Albury | Australia | Unknown status |
| Calvary Mater Newcastle Hospital Coordinator of this site? Confirm its status | Waratah | Australia | Unknown status |
| McGill University Health Centre Coordinator of this site? Confirm its status | Montreal | Canada | Unknown status |
| University College London Hospitals NHS Foundation Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Oxford University Hospitals NHS Foundation Trust Coordinator of this site? Confirm its status | Oxford | United Kingdom | Unknown status |
| Uniwersytecki Szpital Kliniczny Nr 1 w Lublinie Coordinator of this site? Confirm its status | Lublin | Poland | Completed |
| Szpital Wojewodzki W Opolu Sp. z o.o. Coordinator of this site? Confirm its status | Opole | Poland | Completed |
| Wojewodzki Szpital Specjalistyczny Im. Janusza Korczaka W Slupsku Sp. z o.o. Coordinator of this site? Confirm its status | Slupsk | Poland | Completed |
| Medicover Integrated Clinical Services Sp. z o.o. Coordinator of this site? Confirm its status | Torun | Poland | Completed |
| O'Neal Comprehensive Cancer Center University of Alabama at Birmingham Coordinator of this site? Confirm its status | Birmingham | United States | Unknown status |
| Cleveland Clinic Foundation Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| John Theurer Cancer Center Coordinator of this site? Confirm its status | Hackensack | United States | Unknown status |
| University of Texas - MD Anderson Cancer Center Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Mayo Clinic Hospital Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
Related publications
- Sapablursen for Erythrocytosis Control in Patients with Polycythemia Vera.Blood · 2026 · PMID 42611791
This page is information, not medical advice. Discuss any trial with your doctor.