NCT07429266

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
250

participants targeted

Target at P50-P75 for phase_3

Timeline
55mo left

Started May 2026

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress1%
May 2026Jan 2031

First Submitted

Initial submission to the registry

February 18, 2026

Completed
6 days until next milestone

First Posted

Study publicly available on registry

February 24, 2026

Completed
3 months until next milestone

Study Start

First participant enrolled

May 25, 2026

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2028

Expected
2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2031

Last Updated

June 5, 2026

Status Verified

June 1, 2026

Enrollment Period

2.3 years

First QC Date

February 18, 2026

Last Update Submit

June 3, 2026

Conditions

Polycythemia Vera

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants with Absence of Phlebotomy Eligibility

    Response is defined as absence of phlebotomy eligibility.

    Week 20 through Week 32

Secondary Outcomes (7)

  • Number of Phlebotomies

    Week 0 through Week 32

  • Percentage of Participants with Hct Control

    Week 0 to Week 32

  • 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

  • +2 more secondary outcomes

Study Arms (2)

Sapablursen

EXPERIMENTAL

Sapablursen (32 Weeks) - Sapablursen (124 Weeks Open-label)

Drug: Sapablursen

Placebo

EXPERIMENTAL

Placebo (32 Weeks) - Sapablursen (124 Weeks Open-label)

Drug: SapablursenDrug: Placebo

Interventions

SapablursenDRUG

Administered subcutaneously (SC)

Also known as: ONO-0530, ISIS 702843
PlaceboSapablursen
PlaceboDRUG

Administered SC

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Regis Clinical Research LLC.

Miami, Florida, 33126, United States

RECRUITING

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative Disorders

Study Officials

  • Clinical Team

    Deciphera Pharmaceuticals, LLC

    STUDY DIRECTOR

Central Study Contacts

Clinical Team

CONTACT

888-724-3274clinicaltrials@deciphera.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 18, 2026

First Posted

February 24, 2026

Study Start

May 25, 2026

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

January 1, 2031

Last Updated

June 5, 2026

Record last verified: 2026-06

Data Sharing

IPD Sharing
Will not share

Locations

US(1)