PTG-300 in Subjects With Hereditary Hemochromatosis

Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis

This study will be conducted at multiple sites and every patient will get treated with PTG-300. The objective of the study is to assess the effect of PTG-300 in treating adult hereditary hemochromatosis patients.

Study Overview

• Status: Completed
• Conditions: Hereditary Hemochromatosis
• Intervention / Treatment: Drug: PTG-300

Detailed Description

This study is a multicenter, open-label study. The objective of the study is to assess the effect of PTG-300 on transferrin saturation (TSAT) and serum iron in adult hereditary hemochromatosis patients.

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada
      • Protagonist Investigational Site
  • Ontario
    • Toronto, Ontario, Canada, M6H 3M1
      • Protagonist Investigational Site
• United States
  • Georgia
    • Fayetteville, Georgia, United States, 30214
      • Protagonist Investigational Site
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Protagonist Investigational Site
  • North Carolina
    • Charlotte, North Carolina, United States, 28210
      • Protagonist Investigational Site
  • Texas
    • Conroe, Texas, United States, 77384
      • Protagonist Investigational Site
    • Dallas, Texas, United States, 75246
      • Protagonist Investigational Site
    • Houston, Texas, United States, 77030
      • Protagonist Investigational Site
    • Houston, Texas, United States, 77058
      • Protagonist Investigational Site
  • Virginia
    • Richmond, Virginia, United States, 23226
      • Protagonist Investigational Site
  • Washington
    • Seattle, Washington, United States, 98105
      • Protagonist Investigational Site

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Men and women aged 18 and older
• Confirmed diagnosis of HFE-related hereditary hemochromatosis with prior genotype testing
• Documented stable phlebotomy for ≥ 6 months
• Screening hemoglobin >11.5 g/dL
• Documented evidence of prior serum ferritin ≥500 ng/mL
• Serum ferritin >50 ng/mL and <300 ng/mL at screening

Exclusion Criteria:

• Clinically meaningful laboratory abnormality
• Receiving iron chelation therapy
• Receiving erythrocytapheresis
• Pregnant or lactating females
• Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infections requiring antimicrobial therapy within 4 weeks of dosing.
• Serious or unstable medical or psychiatric condition that would prevent subject providing informed consent
• Organ damage from iron overload
• Primary or secondary immunodeficiency
• Positive hepatitis B or hepatitis C or known human immunodeficiency virus infection
• Known history of autoimmune/inflammatory diseases
• Any surgical procedures requiring general anesthesia within 1 month prior to screening or planned surgery during study
• History of invasive malignancies within last 2 years, except non-malignant skin cancer and local cured prostate cancer, cervical cancer, or ductal carcinoma in situ
• Receipt of an investigational agent within 30 days of 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; PTG-300 Subcutaneous	Drug: PTG-300; Active treatment with PTG-300 injected subcutaneously.; Other Names: Rusfertide

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of PTG-300 on Transferrin Saturation	Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.	Week 24 (or End of Treatment if treated for less than 24 weeks)
Effect of PTG-300 on Serum Iron	Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.	Week 24 (or End of Treatment if treated for less than 24 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of PTG-300 on Phlebotomy Frequency	Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300.	Over 24 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mental Component Summary of SF-36	Change from Baseline to Week 24 (or End of Treatment) in Mental Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Mental Component Summary (MCS), for mental health can be calculated. The MCS ranges from 0 (low functioning) to 100 (high functioning).	Week 24 (or End of Treatment if treated for less than 24 weeks)
Physical Component Summary of SF-36	Change from Baseline to Week 24 (or End of Treatment) in Physical Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Physical Component Summary (PCS), for mental health can be calculated. The PCS ranges from 0 (low functioning) to 100 (high functioning).	Week 24 (or End of Treatment if treated for less than 24 weeks)

Collaborators and Investigators

• Sponsor: Protagonist Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 19, 2020
• Primary Completion (Actual): October 6, 2021
• Study Completion (Actual): February 15, 2022

Study Registration Dates

• First Submitted: December 16, 2019
• First Submitted That Met QC Criteria: December 16, 2019
• First Posted (Actual): December 18, 2019

Study Record Updates

• Last Update Posted (Actual): June 15, 2023
• Last Update Submitted That Met QC Criteria: May 22, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Iron Metabolism Disorders; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Iron Overload; Hemochromatosis; Hemosiderosis
• Other Study ID Numbers: PTG-300-06

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