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PTG-300 in Subjects With Hereditary Hemochromatosis

Primary Purpose

Hereditary Hemochromatosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
PTG-300
Sponsored by
Protagonist Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Hemochromatosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site
  • Protagonist Investigational Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PTG-300

Arm Description

PTG-300 Subcutaneous

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

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.

Full Information

First Posted
December 16, 2019
Last Updated
May 22, 2023
Sponsor
Protagonist Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04202965
Brief Title
PTG-300 in Subjects With Hereditary Hemochromatosis
Official Title
Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
March 19, 2020 (Actual)
Primary Completion Date
October 6, 2021 (Actual)
Study Completion Date
February 15, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Protagonist Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Hemochromatosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Single Arm Study
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PTG-300
Arm Type
Experimental
Arm Description
PTG-300 Subcutaneous
Intervention Type
Drug
Intervention Name(s)
PTG-300
Other Intervention Name(s)
Rusfertide
Intervention Description
Active treatment with PTG-300 injected subcutaneously.
Primary Outcome Measure Information:
Title
Effect of PTG-300 on Transferrin Saturation
Description
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Time Frame
Week 24 (or End of Treatment if treated for less than 24 weeks)
Title
Effect of PTG-300 on Serum Iron
Description
Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.
Time Frame
Week 24 (or End of Treatment if treated for less than 24 weeks)
Secondary Outcome Measure Information:
Title
Effect of PTG-300 on Phlebotomy Frequency
Description
Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300.
Time Frame
Over 24 weeks
Other Pre-specified Outcome Measures:
Title
Mental Component Summary of SF-36
Description
Change from Baseline to Week 24 (or End of Treatment) in Mental Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Mental Component Summary (MCS), for mental health can be calculated. The MCS ranges from 0 (low functioning) to 100 (high functioning).
Time Frame
Week 24 (or End of Treatment if treated for less than 24 weeks)
Title
Physical Component Summary of SF-36
Description
Change from Baseline to Week 24 (or End of Treatment) in Physical Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Physical Component Summary (PCS), for mental health can be calculated. The PCS ranges from 0 (low functioning) to 100 (high functioning).
Time Frame
Week 24 (or End of Treatment if treated for less than 24 weeks)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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 prostate cancer, cervical cancer, or ductal carcinoma in situ Receipt of an investigational agent within 30 days of screening
Facility Information:
Facility Name
Protagonist Investigational Site
City
Fayetteville
State/Province
Georgia
ZIP/Postal Code
30214
Country
United States
Facility Name
Protagonist Investigational Site
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Facility Name
Protagonist Investigational Site
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28210
Country
United States
Facility Name
Protagonist Investigational Site
City
Conroe
State/Province
Texas
ZIP/Postal Code
77384
Country
United States
Facility Name
Protagonist Investigational Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75246
Country
United States
Facility Name
Protagonist Investigational Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Protagonist Investigational Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77058
Country
United States
Facility Name
Protagonist Investigational Site
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23226
Country
United States
Facility Name
Protagonist Investigational Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Protagonist Investigational Site
City
Halifax
State/Province
Nova Scotia
Country
Canada
Facility Name
Protagonist Investigational Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M6H 3M1
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

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PTG-300 in Subjects With Hereditary Hemochromatosis

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