Hereditary HemochromatosisPhase 2NCT04202965

PTG-300 in Subjects With Hereditary Hemochromatosis

A Phase 2 study testing PTG-300 in people with Hereditary Hemochromatosis, run by Protagonist Therapeutics, Inc. at 11 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Hereditary Hemochromatosis
What is being tested?: PTG-300
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: 11 sitesin 2 countries
Study dates: 19 Mar 2020to 15 Feb 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Show all 13Show fewer

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 locations and contacts

11 sites in 2 countries.

Country

City, postcode or hospital

11 sites in 2 countries

United States9 sites

Protagonist Investigational Site
Bethesda, Maryland, 20817
Protagonist Investigational Site
Charlotte, North Carolina, 28210
Protagonist Investigational Site
Conroe, Texas, 77384
Protagonist Investigational Site
Dallas, Texas, 75246
Protagonist Investigational Site
Fayetteville, Georgia, 30214
Protagonist Investigational Site
Houston, Texas, 77030
Protagonist Investigational Site
Houston, Texas, 77058
Protagonist Investigational Site
Richmond, Virginia, 23226
Protagonist Investigational Site
Seattle, Washington, 98105

Other trials in United States

Canada2 sites

Protagonist Investigational Site
Halifax, Nova Scotia
Protagonist Investigational Site
Toronto, Ontario, M6H 3M1

Other trials in Canada

About this study

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

Read the 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.

Conditions: Hereditary Hemochromatosis
Being tested: PTG-300
Medical subject headings: Hemochromatosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PTG-300Drug
Also called Rusfertide
Active treatment with PTG-300 injected subcutaneously.

Study groups in NCT04202965
Study group	Role	What they receive
PTG-300	Experimental	PTG-300 Subcutaneous Receives: Drug: PTG-300

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: Week 24 (or End of Treatment if treated for less than 24 weeks)

Secondary outcomes (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.
Measured: Over 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

19 Mar 202015 Feb 2022

First posted18 Dec 2019
Study start19 Mar 2020
Primary completion6 Oct 2021
Study completion15 Feb 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Hereditary HemochromatosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Single Arm Study
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04202965
Sponsor's protocol ID: PTG-300-06
Status last verified: May 2023
First submitted: 16 Dec 2019
Last updated: 20 Aug 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Kowdley KV, Modi NB, Peltekian K, Vierling JM, Ferris C, Valone FH, Gupta S. Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial. Lancet Gastroenterol Hepatol. 2023 Dec;8(12):1118-1128. doi: 10.1016/S2468-1253(23)00250-9. Epub 2023 Oct 17.
PubMed 37863080

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 2
Treatment of Hemochromatosis
Hemochromatosis
Active, not recruitingPhase 2
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Active, not recruiting
Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
Haemochromatosis
RecruitingPhase 1/2
A Study to Evaluate BBI-001 in Healthy Volunteers and in Patients With Hereditary Hemochromatosis
Hereditary Hemochromatosis
RecruitingObservational study
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Chronic Liver Disease

All Hereditary Hemochromatosis trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
ClinicalTrials.gov ID: NCT04202965
Acronym / sponsor ID: PTG-300-06
Conditions: Hereditary Hemochromatosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: Protagonist Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 19 Mar 2020 (actual)
Primary completion: 6 Oct 2021 (actual)
Study completion: 15 Feb 2022 (actual)
First posted: 18 Dec 2019 (actual)
Results: Posted 15 Jun 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Single Arm Study

Arm	Type	Receives
PTG-300	Experimental	Drug: PTG-300PTG-300 Subcutaneous

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

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

Key exclusion criteria (13 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PTG-300Also: RusfertideActive treatment with PTG-300 injected subcutaneously.	Drug	PTG-300

Primary outcome

Effect of PTG-300 on Transferrin Saturation
Time frame: 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
Time frame: 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.

1 secondary outcome

Effect of PTG-300 on Phlebotomy Frequency
Time frame: Over 24 weeks

Results and safety

Results posted to the registry on 15 Jun 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
PTG-300	16	12

Primary outcome: Effect of PTG-300 on Transferrin Saturation

Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks). Mean (Standard Deviation). Unit: percent saturation

Measure	PTG-300 n = 14
Value	-6.07 (19.4)

Population analysed: Subjects who had paired values to allow determination of change from baseline

Primary outcome: Effect of PTG-300 on Serum Iron

Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks). Mean (Standard Deviation). Unit: micromol/L

Measure	PTG-300 n = 14
Value	-2.34 (9.76)

Population analysed: Subjects who had paired values to allow determination of change from baseline

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 6 months. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
PTG-300	1 of 16 (6.3%)	12 of 16 (75%)	0 of 16

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 37863080Kowdley KV, Modi NB, Peltekian K, Vierling JM, Ferris C, Valone FH, Gupta S. Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial. Lancet Gastroenterol Hepatol. 2023 Dec;8(12):1118-1128. doi: 10.1016/S2468-1253(23)00250-9. Epub 2023 Oct 17.
Individual participant dataSharing planned: No

Related trials for Hereditary Hemochromatosis

Active, not recruitingPhase 2
Treatment of Hemochromatosis
Hemochromatosis
Active, not recruitingPhase 2
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Active, not recruiting
Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
Haemochromatosis
RecruitingPhase 1/2
A Study to Evaluate BBI-001 in Healthy Volunteers and in Patients With Hereditary Hemochromatosis
Hereditary Hemochromatosis
RecruitingObservational study
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Chronic Liver Disease

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

11 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PTG-300 in people with Hereditary Hemochromatosis, run by Protagonist Therapeutics, Inc. at 11 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Effect of PTG-300 on Transferrin Saturation (time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04202965, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2025.