Polycythemia Vera
Conditions
Brief summary
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
DRUGSapablursen
Administered subcutaneously (SC)
DRUGPlacebo
Administered SC
Sponsors
Ono Pharmaceutical Co., Ltd.
Deciphera Pharmaceuticals, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. 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
1. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants with Absence of Phlebotomy Eligibility | Week 20 through Week 32 | Response is defined as absence of phlebotomy eligibility. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Phlebotomies | Week 0 through Week 32 | — |
| Percentage of Participants with Hct Control | Week 0 to Week 32 | Defined as all local laboratory Hct \<45% without phlebotomy. |
| Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score | Baseline, Week 32 | — |
| Change from Baseline in Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score (TSS) | Baseline, Week 32 | — |
| Percentage of Participants with Absence of Phlebotomy Eligibility | Week 20 through Week 52 | Response is defined as absence of phlebotomy eligibility. |
Countries
Australia, Japan, United States
Contacts
CONTACTClinical Team
STUDY_DIRECTORClinical Team
Deciphera Pharmaceuticals, LLC
Outcome results
None listed