PTG-300 in Subjects With Hereditary Hemochromatosis

Phase 2

Completed

• Conditions: Hereditary Hemochromatosis
• Interventions: Drug: PTG-300

• Registration Number: NCT04202965
• Lead Sponsor: Protagonist Therapeutics, Inc.

Brief Summary

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.

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 Timeline

• Study First Submitted: 2019-12-16
• Study First Submitted QC: 2019-12-16
• Study First Posted: 2019-12-18
• Study Start: 2020-03-19
• Primary Completion: 2021-10-06
• Study Completion: 2022-02-15
• Results First Submitted: 2023-04-18
• Status Verified: 2023-05
• Results First Submitted QC: 2023-05-22
• Last Update Submitted: 2023-05-22
• Results First Posted: 2023-06-15
• Last Update Posted: 2023-06-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PTG-300	PTG-300	PTG-300 Subcutaneous

Primary Outcome Measures

Name	Time	Method
Effect of PTG-300 on Transferrin Saturation	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.
Effect of PTG-300 on Serum Iron	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 Outcome Measures

Name	Time	Method
Effect of PTG-300 on Phlebotomy Frequency	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.

Trial Locations

Locations (1)

Protagonist Investigational Site; 🇨🇦 Toronto, Ontario, Canada