Myeloproliferative Neoplasms Clinical Trial

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

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.

View Full Description

Full 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 3 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 33 weeks + a 4-week post-last dose visit; 3) 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 10 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.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria

Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis
Participant must be phlebotomy dependent.
If the participant is currently on cytoreductive therapy or has been previously treated with cytoreductive therapy, the participant's cytoreductive therapy must either be discontinued at least 3 months prior to Screening, OR participant must be on a stable dose for at least 3 months prior to Screening.

Exclusion Criteria

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

Study is for people with:

Myeloproliferative Neoplasms

Phase:

Phase 2

Estimated Enrollment:

40

Study ID:

NCT05143957

Recruitment Status:

Recruiting

Sponsor:

Ionis Pharmaceuticals, Inc.

Check Your Eligibility

Let’s see if you might be eligible for this study.

What is your age and gender ?

Submit

There are 10 Locations for this study

See Locations Near You

City of Hope National Medical Center
Duarte California, 91010, United States
Norris Comprehensive Cancer Center
Los Angeles California, 91011, United States
University of California Irvine
Orange California, 92868, United States
Innovative Clinical Research Institute
Whittier California, 90603, United States
Washington University School of Medicine
Saint Louis Missouri, 63110, United States
Herbert Irving Comprehensive Cancer Center
New York New York, 10032, United States
University of Texas MD Anderson Cancer Center
Houston Texas, 77030, United States
Mays Cancer Center
San Antonio Texas, 78229, United States
McGill University Health Centre
Montreal Quebec, H4A 3, Canada
Sandwell and West Birmingham Hospitals NHS Trust
West Bromwich , B71 4, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myeloproliferative Neoplasms

Phase:

Phase 2

Estimated Enrollment:

40

Study ID:

NCT05143957

Recruitment Status:

Recruiting

Sponsor:


Ionis Pharmaceuticals, Inc.

How clear is this clinincal trial information?

×

Introducing, the Journey Bar

Use this bar to access information about the steps in your cancer journey.