INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Sapablursen, Placebo.
- Who it may be relevant to
- Registry conditions: Polycythemia Vera. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3 Randomized, Double-blind, Placebo-controlled Global Study of Sapablursen in Polycythemia Vera
Overview
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.
Interventions
- Drug Sapablursen
Administered subcutaneously (SC) - Drug Placebo
Administered SC
Primary outcome measures
- Percentage of Participants with Absence of Phlebotomy Eligibility [Time frame: Week 20 through Week 32]
Secondary outcome measures (7)
- Number of Phlebotomies [Time frame: Week 0 through Week 32]
- Percentage of Participants with Hct Control [Time frame: Week 0 to Week 32]
- Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score [Time frame: Baseline, Week 32]
- Change from Baseline in Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score (TSS) [Time frame: Baseline, Week 32]
- Percentage of Participants with Absence of Phlebotomy Eligibility [Time frame: Week 20 through Week 52]
- Number of Phlebotomies [Time frame: Week 0 through Week 52]
- Percentage of Participants with Hct Control [Time frame: Week 0 through Week 52]
Eligibility criteria
Inclusion criteria
- Meet revised 2022 World Health Organization (WHO) and 2022 International Consensus Classification criteria for the diagnosis of PV.
- Participants must be phlebotomy-dependent.
- Hct less than (<) 45% at study start.
- Participants receiving Cytoreduction therapy (CRT) must be on a stable regimen at study start.
- Adequate organ function and electrolytes.
Exclusion criteria
- Prior treatment of PV with Transmembrane serine protease 6 (TMPRSS6) inhibitors, including sapablursen, or hepcidin mimetics.
- Clinically significant thrombosis (eg, myocardial infarction, stroke, deep vein thrombosis or splenic vein thrombosis) within 1 month prior to randomization.
- Participants who require phlebotomy at Hct levels <45%.
- Meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment.
- Any serious or unstable medical condition or uncontrolled psychiatric condition that would interfere with their ability to comply with study requirements.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- Regis Clinical Research LLC. — Miami
- Florida Clinical Trials Group — Plantation
- Florida Clinical Trials Group — Tamarac
- The Center for Cancer & Blood Disorders — Bethesda
- Montefiore Medical Center — The Bronx
- Gabrail Cancer Center Research — Canton
Identifiers
NCT: NCT07429266 · ONO-0530-03-001