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PTG-300 in Subjects With Hereditary Hemochromatosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04202965
Recruitment Status : Completed
First Posted : December 18, 2019
Last Update Posted : March 2, 2022
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Hereditary Hemochromatosis Drug: PTG-300 Phase 2

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Single Arm Study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
Actual Study Start Date : March 19, 2020
Actual Primary Completion Date : October 6, 2021
Actual Study Completion Date : February 15, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemochromatosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: PTG-300
PTG-300 Subcutaneous
 Drug: PTG-300
Active treatment with PTG-300 subcutaneously


Outcome Measures
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Primary Outcome Measures :
  1. Effect of PTG-300 on Transferrin Saturation [ Time Frame: Week 24 ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests.

  2. Effect of PTG-300 on Serum Iron [ Time Frame: Week 24 ]
    Change in serum iron as measured by blood laboratory tests.


Secondary Outcome Measures :
  1. Effect of PTG-300 on Phlebotomy Frequency [ Time Frame: Week 24 ]
    Change in phlebotomy frequency as measured by number of phlebotomies over a 24 week period.

  2. SF-36 [ Time Frame: Week 24 ]
    The SF-36 is a participant-reported outcome measure. The instrument includes 36 items in a Likert-type format to measure the following health dimensions over the past week: limitations in physical activities; limitations in social activities; limitations in usual role activities; bodily pain; general mental health; vitality; and general health perceptions.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 prostrate cancer, cervical cancer, or ductal carcinoma in situ
  • Receipt of an investigational agent within 30 days of screening
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04202965


Locations
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United States, Georgia
Protagonist Investigational Site
Fayetteville, Georgia, United States, 30214
United States, Maryland
Protagonist Investigational Site
Bethesda, Maryland, United States, 20817
United States, North Carolina
Protagonist Investigational Site
Charlotte, North Carolina, United States, 28210
United States, Texas
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
United States, Virginia
Protagonist Investigational Site
Richmond, Virginia, United States, 23226
United States, Washington
Protagonist Investigational Site
Seattle, Washington, United States, 98105
Canada, Nova Scotia
Protagonist Investigational Site
Halifax, Nova Scotia, Canada
Canada, Ontario
Protagonist Investigational Site
Toronto, Ontario, Canada, M6H 3M1
Sponsors and Collaborators
Protagonist Therapeutics, Inc.
More Information
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Responsible Party: Protagonist Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04202965    
Other Study ID Numbers: PTG-300-06
First Posted: December 18, 2019    Key Record Dates
Last Update Posted: March 2, 2022
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemochromatosis
Hemosiderosis
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases