PTG-300 in Subjects With Hereditary Hemochromatosis

• ClinicalTrials.gov Identifier: NCT04202965
• Recruitment Status: Completed
• First Posted: December 18, 2019
• Last Update Posted: March 2, 2022
• Sponsor: Protagonist Therapeutics, Inc.
• Information provided by (Responsible Party): Protagonist Therapeutics, Inc.

Tracking Information

• First Submitted Date: December 16, 2019
• First Posted Date: December 18, 2019
• Last Update Posted Date: March 2, 2022
• Actual Study Start Date: March 19, 2020
• Actual Primary Completion Date: October 6, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 16, 2019)

• Effect of PTG-300 on Transferrin Saturation [ Time Frame: Week 24 ]
  Change in transferrin saturation (TSAT) as measured by blood laboratory tests.
• Effect of PTG-300 on Serum Iron [ Time Frame: Week 24 ]
  Change in serum iron as measured by blood laboratory tests.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 16, 2019)

• 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.
• 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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: PTG-300 in Subjects With Hereditary Hemochromatosis
• Official Title: Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
• 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.
• 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 Type: Interventional
• Study Phase: Phase 2

Study Design

Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:

Single Arm Study

Masking: None (Open Label)
Primary Purpose: Treatment

• Condition: Hereditary Hemochromatosis

Intervention

Drug: PTG-300

Active treatment with PTG-300 subcutaneously

Study Arms

Experimental: PTG-300

PTG-300 Subcutaneous

Intervention: Drug: PTG-300

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 1, 2022): 16
• Original Estimated Enrollment (submitted: December 16, 2019): 28
• Actual Study Completion Date: February 15, 2022
• Actual Primary Completion Date: October 6, 2021 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT04202965
• Other Study ID Numbers: PTG-300-06
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Protagonist Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Protagonist Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Protagonist Therapeutics, Inc.
• Verification Date: September 2021