A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
- Conditions
- Phlebotomy Dependent Polycythemia Vera
- Interventions
- Registration Number
- NCT05143957
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief 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.
- Detailed 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 50
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Sapablursen Dose Level 1 sapablursen Sapablursen will be administered by SC injection every 4 weeks. Sapablursen Dose Level 2 sapablursen Sapablursen will be administered by SC injection every 4 weeks
- Primary Outcome Measures
Name Time Method Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period Week 17 to Week 37
- Secondary Outcome Measures
Name Time Method Proportion of patients achieving a reduction in the frequency of phlebotomy by ≥ 30%, ≥ 50%, ≥ 75% and ≥ 90% comparing Baseline with the last 20 weeks of the 37-week Treatment Period Week 17 to Week 37 Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37 Baseline up to Week 37
Trial Locations
- Locations (18)
Mayo Clinic Hospital
🇺🇸Phoenix, Arizona, United States
Wojewódzki Szpital Specjalistyczny Sp. z o.o.
🇵🇱Słupsk, Poland
MICS Centrum Medyczne Toruń
🇵🇱Toruń, Poland
O'Neal Comprehensive Cancer Center University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Norris Comprehensive Cancer Center
🇺🇸Los Angeles, California, United States
University of California Irvine
🇺🇸Orange, California, United States
John Theurer Cancer Center
🇺🇸Hackensack, New Jersey, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
The Perth Blood Institute
🇦🇺West Perth, Australia
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Calvary Mater Newcastle Hospital
🇦🇺Waratah, New South Wales, Australia
Border Medical Oncology Research Unit
🇦🇺Albury, Australia
McGill University Health Centre
🇨🇦Montreal, Quebec, Canada
Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie, Klinika Hematoonkologii i Transplantacji Szpiku
🇵🇱Lublin, Poland
United Lincolnshire Hospitals NHS Trust
🇬🇧Lincoln, England, United Kingdom
Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, England, United Kingdom
University College London Hospitals NHS Foundation Trust
🇬🇧London, United Kingdom
Sandwell and West Birmingham Hospitals NHS Trust
🇬🇧West Bromwich, United Kingdom