PTG-300 in Subjects With Hereditary Hemochromatosis

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Hereditary Hemochromatosis

Intervention / Treatment
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* Drug: PTG-300


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Single Arm Study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PTG-300<br>PTG-300 Subcutaneous | Drug: PTG-300<br>* Active treatment with PTG-300 injected subcutaneously.<br>* Other names: Rusfertide;|

What is the study measuring?
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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 |