PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit

NCT04767802 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: PTG-300-08
• Study Type: interventional
• Target: 20
• Locations: 2 countries
• Sites: 8

Brief Summary

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.

Conditions

Polycythemia Vera

Keywords

PV

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Safety of PTG-300

  Week 52

Study Arms (1)

PTG-300

EXPERIMENTAL

Evaluate PTG-300's efficacy and safety in subjects with PV and baseline elevated hematocrit.

Drug: PTG-300

Interventions

PTG-300 DRUG

Hepcidin mimetic

Also known as: Rusfertide

PTG-300

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Known diagnosis of polycythemia vera.
• Hematocrit \>48% before dosing.
• 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).
• Clinically reasonable alternative causes for erythrocytosis have been evaluated and excluded.
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.

You may not qualify if:

• Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 3 months of Screening.
• Active or chronic bleeding within 4 weeks of Screening.
• Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
• 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.
• 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.
• Known primary or secondary immunodeficiency.
• Known positive for active hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection.
• Any surgical procedure requiring general anesthesia within 1 month prior to Screening or planned elective surgery during the study.
• History of invasive malignancies within the last 2 years, except non-melanoma skin cancer and localized curatively treated prostate cancer or cervical cancer.
• Current or recent history of alcohol dependence or illicit drug use within 1 year prior to Screening.

Sponsors & Collaborators

• Protagonist Therapeutics, Inc.: lead

Study Sites (8)

Hospital Sultanah Aminah

Johor Bahru, Johor, Malaysia

Location

Hospital Raja Perempuan Zainab

Kota Bharu, Kelantan, Malaysia

Location

Hospital Raja Permaisuki Bainun

Ipoh, Perak, Malaysia

Location

Hospital Umum Sarawak

Kuching, Sarawak, Malaysia

Location

Hospital Ampang

Ampang, Selangor, Malaysia

Location

Gachon University Medical Center

Incheon, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Seoul St. Mary's Catholic University Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2021
• First Posted: February 23, 2021
• Study Start: February 2, 2021
• Primary Completion: March 15, 2023
• Study Completion: March 15, 2023
• Last Updated: March 18, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

MY (5); KR (3)