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Completed

NCT Number: NCT04202965

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.

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Key information

Conditions

Hereditary Hemochromatosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemochromatosis Iron Metabolism Disorders Iron Overload Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Protagonist Investigational Site, Halifax, Nova Scotia, Canada

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PTG-300

Drug

Active treatment with PTG-300 injected subcutaneously.

Other names: Rusfertide

Primary outcomes

  1. Effect of PTG-300 on Transferrin Saturation

    Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)

    Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.

  2. Effect of PTG-300 on Serum Iron

    Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)

    Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.

Secondary outcomes

  1. Effect of PTG-300 on Phlebotomy Frequency

    Time frame: Over 24 weeks

    Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300.

Other outcomes

  1. Mental Component Summary of SF-36

    Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)

    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).

  2. Physical Component Summary of SF-36

    Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)

    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).

Sponsors and collaborators

Lead sponsor

Protagonist Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2021
Study completion
2022
First posted
Dec 18, 2019
Registry last updated
Aug 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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