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

ClinicalTrials.gov processed this data on May 22, 2023. Link to the current ClinicalTrials.gov record.

Recruitment Status

COMPLETED - HAS RESULTS
 (See Contacts and Locations)
Verified May 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 16, 2019
First Posted: December 18, 2019
Results First Posted: June 15, 2023
Last Update Posted: June 15, 2023
Last Verified: May 2023
History of Changes

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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 Disease Intervention/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 19, 2020
Actual Primary Completion DateOctober 6, 2021
Actual Study Completion DateFebruary 15, 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 [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.
  2. Effect of PTG-300 on Serum Iron [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.

Secondary Outcome Measures

  1. Effect of PTG-300 on Phlebotomy Frequency [Over 24 weeks]
    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 18 Years and Older (Adult, Older Adult)
Sexes Eligible for Study All
Accepts Healthy Volunteers No
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 Collaborators Protagonist Therapeutics, Inc.
Locations
  • Protagonist Investigational Site | Fayetteville, Georgia, United States, 30214
  • Protagonist Investigational Site | Bethesda, Maryland, United States, 20817
  • Protagonist Investigational Site | Charlotte, North Carolina, United States, 28210
  • Protagonist Investigational Site | Conroe, Texas, United States, 77384
  • Protagonist Investigational Site | Dallas, Texas, United States, 75246
  • Protagonist Investigational Site | Houston, Texas, United States, 77030
  • Protagonist Investigational Site | Houston, Texas, United States, 77058
  • Protagonist Investigational Site | Richmond, Virginia, United States, 23226
  • Protagonist Investigational Site | Seattle, Washington, United States, 98105
  • Protagonist Investigational Site | Halifax, Nova Scotia, Canada,
  • Protagonist Investigational Site | Toronto, Ontario, Canada, M6H 3M1

Study Documents (Full Text)

More Information

Additional Relevant MeSH Terms

  • Hemochromatosis
  • Hemosiderosis
  • Metal Metabolism, Inborn Errors
  • Metabolism, Inborn Errors
  • Genetic Diseases, Inborn
  • Iron Overload
  • Iron Metabolism Disorders
  • Metabolic Diseases