INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
Purpose
The purpose of this study is to evaluate the efficacy and safety of sapablursen when added on to current standard of care (SOC) for Polycythemia Vera (PV) therapy. The study will be conducted in three sequential parts (Part 1a blinded treatment, Part 1b open-label treatment, & Part 2 long-term extension). Participants may receive treatment for up to 156 weeks.
Condition
- Polycythemia Vera
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Meet revised 2022 World Health Organization (WHO) and 2022 International Consensus Classification criteria for the diagnosis of PV. 2. Participants must be phlebotomy-dependent. 3. Hct less than (<) 45% at study start. 4. Participants receiving Cytoreduction therapy (CRT) must be on a stable regimen at study start. 5. Adequate organ function and electrolytes.
Exclusion Criteria
- Prior treatment of PV with Transmembrane serine protease 6 (TMPRSS6) inhibitors, including sapablursen, or hepcidin mimetics. 2. Clinically significant thrombosis (eg, myocardial infarction, stroke, deep vein thrombosis or splenic vein thrombosis) within 1 month prior to randomization. 3. Participants who require phlebotomy at Hct levels <45%. 4. Meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment. 5. Any serious or unstable medical condition or uncontrolled psychiatric condition that would interfere with their ability to comply with study requirements.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Sapablursen |
Sapablursen (32 Weeks) - Sapablursen (124 Weeks Open-label) |
|
|
Experimental Placebo |
Placebo (32 Weeks) - Sapablursen (124 Weeks Open-label) |
|
Recruiting Locations
Regis Clinical Research LLC.
Miami, Florida 33126
Miami, Florida 33126
Florida Clinical Trials Group
Plantation, Florida 33322
Plantation, Florida 33322
Florida Clinical Trials Group
Tamarac, Florida 33321
Tamarac, Florida 33321
The Center for Cancer & Blood Disorders
Bethesda, Maryland 20817
Bethesda, Maryland 20817
Montefiore Medical Center
The Bronx, New York 10461
The Bronx, New York 10461
Gabrail Cancer Center Research
Canton, Ohio 44718
Canton, Ohio 44718
More Details
- Status
- Recruiting
- Sponsor
- Ono Pharmaceutical Co., Ltd.