INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)

Phase 3RecruitingNCT07429266

Ono Pharmaceutical Co., Ltd.250 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

250

Conditions

Polycythemia Vera

Interventions

SapablursenDRUG

Administered subcutaneously (SC)

PlaceboDRUG

Administered SC

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (10)

Regis Clinical Research LLC.

Miami, Florida, United States

RECRUITING

Florida Clinical Trials Group

Plantation, Florida, United States

RECRUITING

Outcomes

Primary Outcomes

Percentage of Participants with Absence of Phlebotomy Eligibility

Response is defined as absence of phlebotomy eligibility.

Time frame: Week 20 through Week 32

Secondary Outcomes

Number of Phlebotomies

Time frame: Week 0 through Week 32

Percentage of Participants with Hct Control

Defined as all local laboratory Hct \<45% without phlebotomy.

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

Response is defined as 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

Defined as all local laboratory Hct \<45% without phlebotomy.

Time frame: Week 0 through Week 52

Central Contacts

Clinical Team

CONTACT

888-724-3274 clinicaltrials@deciphera.com

Data from ClinicalTrials.gov

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