PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit (PACIFIC)

A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit (PACIFIC)

This is an open label, single arm trial of PTG-300 in subjects with PV who are newly diagnosed or for whom current therapy is not sufficient to control their hematocrit and have hematocrit >48% prior to dosing. The PTG-300 dose and schedule may be adjusted every 2 to 4 weeks to maintain hematocrit <45% with a target of <43%. Subjects may receive PTG-300 treatment for up to 52 weeks.

Study Overview

• Status: Completed
• Conditions: Polycythemia Vera
• Intervention / Treatment: Drug: PTG-300

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Incheon, Korea, Republic of
    • Gachon University Medical Center
  • Seoul, Korea, Republic of
    • Seoul National University Hospital
  • Seoul, Korea, Republic of
    • Seoul St. Mary's Catholic University Hospital
• Malaysia
  • Johor
    • Johor Bahru, Johor, Malaysia
      • Hospital Sultanah Aminah
  • Kelantan
    • Kota Bharu, Kelantan, Malaysia
      • Hospital Raja Perempuan Zainab
  • Perak
    • Ipoh, Perak, Malaysia
      • Hospital Raja Permaisuki Bainun
  • Sarawak
    • Kuching, Sarawak, Malaysia
      • Hospital Umum Sarawak
  • Selangor
    • Ampang, Selangor, Malaysia
      • Hospital Ampang

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Known diagnosis of polycythemia vera.
2. Hematocrit >48% before dosing.
3. Evidence of hematocrit >48% three or more times in the 28 weeks before dosing or five or more times in 52 weeks before dosing (except for newly diagnosed patients).
4. Clinically reasonable alternative causes for erythrocytosis have been evaluated and excluded.
5. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.

Exclusion Criteria:

1. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 3 months of Screening.
2. Active or chronic bleeding within 4 weeks of Screening.
3. Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
4. Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infection requiring systemic antimicrobial therapy within 4 weeks of dosing. Prophylactic antibiotics are allowed.
5. Any serious or unstable medical or psychiatric condition that would prevent, (as judged by the Investigator) the subject from properly providing informed consent or any condition which would jeopardize compliance with the study.
6. Known primary or secondary immunodeficiency.
7. Known positive for active hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection.
8. Any surgical procedure requiring general anesthesia within 1 month prior to Screening or planned elective surgery during the study.
9. History of invasive malignancies within the last 2 years, except non-melanoma skin cancer and localized curatively treated prostate cancer or cervical cancer.
10. Current or recent history of alcohol dependence or illicit drug use within 1 year prior to Screening.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PTG-300; Evaluate PTG-300's efficacy and safety in subjects with PV and baseline elevated hematocrit.	Drug: PTG-300; Hepcidin mimetic; Other Names: Rusfertide

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate PTG-300's efficacy in subjects with PV and baseline elevated hematocrit.	Proportion of subjects with hematocrit <45%	Estimate the proportion of subject with a hematocrit less than 45% at Week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of PTG-300	Proportion of Subjects Treatment-Emergent Adverse Events	Week 52

Collaborators and Investigators

• Sponsor: Protagonist Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2021
• Primary Completion (Actual): March 15, 2023
• Study Completion (Actual): March 15, 2023

Study Registration Dates

• First Submitted: February 18, 2021
• First Submitted That Met QC Criteria: February 22, 2021
• First Posted (Actual): February 23, 2021

Study Record Updates

• Last Update Posted (Actual): March 18, 2024
• Last Update Submitted That Met QC Criteria: March 14, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: PV
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Polycythemia Vera; Polycythemia
• Other Study ID Numbers: PTG-300-08

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes