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.

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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 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.

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.
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

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:

60

Study ID:

NCT05143957

Recruitment Status:

Recruiting

Sponsor:

Ionis Pharmaceuticals, Inc.

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There are 40 Locations for this study

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O'Neal Comprehensive Cancer Center University of Alabama at Birmingham
Birmingham Alabama, 35249, United States
Mayo Clinic Hospital
Phoenix Arizona, 85054, United States
PCR Oncology
Arroyo Grande California, 93420, United States
UCLA Health - Beverly Hills Cancer Care
Beverly Hills California, 90212, United States
UCLA Health - Burbank Cancer Care
Burbank California, 91505, United States
City of Hope National Medical Center
Duarte California, 91010, United States
UCLA
Los Angeles California, 90095, United States
Norris Comprehensive Cancer Center
Los Angeles California, 91011, United States
University of California Irvine
Orange California, 92868, United States
UCLA Health - Pasadena Cancer Care
Pasadena California, 91105, United States
UCLA Health -Westlake Village Cancer Care
Westlake Village California, 91361, United States
Innovative Clinical Research Institute
Whittier California, 90603, United States
Medstar Georgetown University Hospital
Washington District of Columbia, 20007, United States
Washington University School of Medicine
Saint Louis Missouri, 63110, United States
Summit Medical Group
Florham Park New Jersey, 07932, United States
John Theurer Cancer Center
Hackensack New Jersey, 07601, United States
Icahn School of Medicine at Mount Sinai
New York New York, 10029, United States
Herbert Irving Comprehensive Cancer Center
New York New York, 10032, United States
Atrium Health Levine Cancer Institute
Charlotte North Carolina, 28204, United States
Atrium Health Wake Forest Baptist
Winston-Salem North Carolina, 27157, United States
Gabrail Cancer Center Research
Canton Ohio, 44718, United States
Cleveland Clinic Foundation
Cleveland Ohio, 44195, United States
University of Texas MD Anderson Cancer Center
Houston Texas, 77030, United States
Mays Cancer Center
San Antonio Texas, 78229, United States
Medical College of Wisconsin
Milwaukee Wisconsin, 53226, United States
St. George Hospital
Kogarah New South Wales, 2217, Australia
Border Medical Oncology Research Unit
Albury , 2640, Australia
Box Hill Hospital
Box Hill , 3128, Australia
The Perth Blood Institute
West Perth , 6005, Australia
McGill University Health Centre
Montreal Quebec, H4A 3, Canada
Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika
Budapest , 1088, Hungary
Debreceni Egyetem Klinikai Központ
Debrecen , 4032, Hungary
Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie, Klinika Hematoonkologii i Transplantacji Szpiku
Lublin , 20-08, Poland
Szpital Wojewódzki w Opolu
Opole , 45-06, Poland
Wojewódzki Szpital Specjalistyczny Sp. z o.o.
Słupsk , 76-20, Poland
MICS Centrum Medyczne Toruń
Toruń , 87-10, Poland
Lincoln County Hospital
Lincoln England, LN2 5, United Kingdom
United Lincolnshire Hospitals NHS Trust
Lincoln England, LN2 5, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford England, OX37L, United Kingdom
University College London Hospitals NHS Foundation Trust
London , NW1 2, United Kingdom
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:

60

Study ID:

NCT05143957

Recruitment Status:

Recruiting

Sponsor:


Ionis Pharmaceuticals, Inc.

How clear is this clinincal trial information?

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