PTG-300 in Subjects With Hereditary Hemochromatosis

Recruitment Status
COMPLETED - HAS RESULTS
(See Contacts and Locations)Verified April 2023 by Protagonist Therapeutics, Inc.
Sponsor
Protagonist Therapeutics, Inc.
Information Provided by (Responsible Party)
Protagonist Therapeutics, Inc.
Clinicaltrials.gov Identifier
NCT04202965
Other Study ID Numbers:
PTG-300-06
First Submitted
December 15, 2019
First Posted
December 17, 2019
Results First Posted
April 17, 2023
Last Update Posted
August 19, 2025
Last Verified
April 2023

ClinicalTrials.gov processed this data on August 2025. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study 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.

Condition or DiseaseIntervention/Treatment
Hereditary Hemochromatosis
Drug: PTG-300

Study Design

Study TypeInterventional
Actual Enrollment16 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleOpen-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
Study Start DateMarch 18, 2020
Actual Primary Completion DateOctober 5, 2021
Actual Study Completion DateFebruary 14, 2022

Groups and Cohorts

Group/CohortIntervention/Treatment
PTG-300
PTG-300 Subcutaneous
Drug: PTG-300
Active treatment with PTG-300 injected subcutaneously.

Outcome Measures

Primary Outcome Measures
  1. 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.
  2. 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.
Secondary Outcome Measures
  1. 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.

Eligibility Criteria

Ages Eligible for Study(Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsProtagonist Therapeutics, Inc.
Locations
Protagonist Investigational Site | Fayetteville Georgia, United States, 30214Protagonist Investigational Site | Bethesda Maryland, United States, 20817Protagonist Investigational Site | Charlotte North Carolina, United States, 28210Protagonist Investigational Site | Conroe Texas, United States, 77384Protagonist Investigational Site | Dallas Texas, United States, 75246Protagonist Investigational Site | Houston Texas, United States, 77030Protagonist Investigational Site | Houston Texas, United States, 77058Protagonist Investigational Site | Richmond Virginia, United States, 23226Protagonist Investigational Site | Seattle Washington, United States, 98105Protagonist Investigational Site | Halifax Nova Scotia, Canada, Protagonist Investigational Site | Toronto Ontario, Canada, M6H 3M1
Study Documents (Full Text)
Documents provided by Protagonist Therapeutics, Inc.: Study Protocol and Statistical Analysis Plan  September 13, 2020