PTG-300 in Subjects With Hereditary Hemochromatosis

NCT ID: NCT04202965

Last Updated: 2025-08-20

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-03-19

Study Completion Date

2022-02-15

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.

Conditions

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Hereditary Hemochromatosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Single Arm Study
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PTG-300

PTG-300 Subcutaneous

Group Type EXPERIMENTAL

PTG-300

Intervention Type DRUG

Active treatment with PTG-300 injected subcutaneously.

Interventions

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PTG-300

Active treatment with PTG-300 injected subcutaneously.

Intervention Type DRUG

Other Intervention Names

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Rusfertide

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Protagonist Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Protagonist Investigational Site

Fayetteville, Georgia, United States

Site Status

Protagonist Investigational Site

Bethesda, Maryland, United States

Site Status

Protagonist Investigational Site

Charlotte, North Carolina, United States

Site Status

Protagonist Investigational Site

Conroe, Texas, United States

Site Status

Protagonist Investigational Site

Dallas, Texas, United States

Site Status

Protagonist Investigational Site

Houston, Texas, United States

Site Status

Protagonist Investigational Site

Houston, Texas, United States

Site Status

Protagonist Investigational Site

Richmond, Virginia, United States

Site Status

Protagonist Investigational Site

Seattle, Washington, United States

Site Status

Protagonist Investigational Site

Halifax, Nova Scotia, Canada

Site Status

Protagonist Investigational Site

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

References

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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.

Reference Type DERIVED
PMID: 37863080 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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PTG-300-06

Identifier Type: -

Identifier Source: org_study_id

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