PTG-300 in Subjects With Hereditary Hemochromatosis

Protagonist Therapeutics

Status and phase: Completed; Phase 2

Conditions: Hereditary Hemochromatosis

Treatments: Drug: PTG-300

Study type: Interventional

Funder types: Industry

Identifiers

NCT04202965

PTG-300-06

Details and patient eligibility

About

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.

Full 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.

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

PTG-300

Experimental group

Description:

PTG-300 Subcutaneous

Treatment:

Drug: PTG-300