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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_2

Timeline

Completed

Started Mar 2020

Geographic Reach

2 countries

11 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1.9 years study duration

First Submitted

Initial submission to the registry

December 16, 2019

Completed

2 days until next milestone

First Posted

Study publicly available on registry

December 18, 2019

Completed

3 months until next milestone

Study Start

First participant enrolled

March 19, 2020

Completed

1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 6, 2021

Completed

4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 15, 2022

Completed

1.3 years until next milestone

Results Posted

Study results publicly available

June 15, 2023

Completed

Last Updated

August 20, 2025

Status Verified

May 1, 2023

Enrollment Period

1.6 years

First QC Date

December 16, 2019

Results QC Date

April 18, 2023

Last Update Submit

August 4, 2025

Conditions

Hereditary Hemochromatosis

Outcome Measures

Primary Outcomes (2)

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 Outcomes (1)

Effect of PTG-300 on Phlebotomy Frequency
Over 24 weeks

Other Outcomes (2)

Mental Component Summary of SF-36
Week 24 (or End of Treatment if treated for less than 24 weeks)
Physical Component Summary of SF-36
Week 24 (or End of Treatment if treated for less than 24 weeks)

Study Arms (1)

PTG-300

EXPERIMENTAL

PTG-300 Subcutaneous

Drug: PTG-300

Interventions

PTG-300DRUG

Active treatment with PTG-300 injected subcutaneously.

Also known as: Rusfertide

PTG-300

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.