Myeloproliferative Neoplasms Clinical Trial
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
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.
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 10 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.
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.
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
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There are 31 Locations for this study
Birmingham Alabama, 35249, United States
Phoenix Arizona, 85054, United States
Arroyo Grande California, 93420, United States
Duarte California, 91010, United States
Los Angeles California, 90095, United States
Los Angeles California, 91011, United States
Orange California, 92868, United States
Whittier California, 90603, United States
Saint Louis Missouri, 63110, United States
Hackensack New Jersey, 07601, United States
New York New York, 10029, United States
New York New York, 10032, United States
Canton Ohio, 44718, United States
Cleveland Ohio, 44195, United States
Houston Texas, 77030, United States
San Antonio Texas, 78229, United States
Milwaukee Wisconsin, 53226, United States
Albury , 2640, Australia
Box Hill , 3128, Australia
West Perth , 6005, Australia
Montreal Quebec, H4A 3, Canada
Budapest , 1088, Hungary
Debrecen , 4032, Hungary
Lublin , 20-08, Poland
Opole , 45-06, Poland
Słupsk , 76-20, Poland
Toruń , 87-10, Poland
Lincoln England, LN2 5, United Kingdom
Lincoln England, LN2 5, United Kingdom
Oxford England, OX37L, United Kingdom
London , NW1 2, United Kingdom
West Bromwich , B71 4, United Kingdom
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