A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
a study on Polycythemia Vera
Summary
- Eligibility
- for people ages 18-90 (full criteria)
- Location
- at UC Irvine UCLA
- Dates
- study startedcompletion around
Description
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.
Official Title
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)
Details
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.
Keywords
Phlebotomy Dependent Polycythemia Vera, Polycythemia Vera, Polycythemia, sapablursen
Eligibility
You can join if…
Open to people ages 18-90
- 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 CAN'T join 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
- 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
Locations
- UCLA Health - Beverly Hills Cancer Care
accepting new patients
Beverly Hills California 90212 United States - UCLA Health - Burbank Cancer Care
accepting new patients
Burbank California 91505 United States - UCLA
accepting new patients
Los Angeles California 90095 United States - University of California Irvine
accepting new patients
Orange California 92868 United States - UCLA Health - Pasadena Cancer Care
accepting new patients
Pasadena California 91105 United States - UCLA Health -Westlake Village Cancer Care
accepting new patients
Westlake Village California 91361 United States - Norris Comprehensive Cancer Center
accepting new patients
Los Angeles California 91011 United States - City of Hope National Medical Center
accepting new patients
Duarte California 91010 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Ionis Pharmaceuticals, Inc.
- ID
- NCT05143957
- Phase
- Phase 2 Polycythemia Vera Research Study
- Study Type
- Interventional
- Participants
- Expecting 60 study participants
- Last Updated