PTG-300 in Subjects With Hereditary Hemochromatosis

Hereditary Hemochromatosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04202965
• Other study ID #: PTG-300-06
• Status: Completed
• Phase: Phase 2
• Start date: March 19, 2020
• Est. completion date: February 15, 2022

Study information

• Verified date: May 2023
• Source: Protagonist Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: February 15, 2022
• Est. primary completion date: October 6, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemochromatosis
• Hemosiderosis
• Hereditary Hemochromatosis

Intervention

Drug:
• PTG-300
Active treatment with PTG-300 injected subcutaneously.

Locations

Country	Name	City	State
Canada	Protagonist Investigational Site	Halifax	Nova Scotia
Canada	Protagonist Investigational Site	Toronto	Ontario
United States	Protagonist Investigational Site	Bethesda	Maryland
United States	Protagonist Investigational Site	Charlotte	North Carolina
United States	Protagonist Investigational Site	Conroe	Texas
United States	Protagonist Investigational Site	Dallas	Texas
United States	Protagonist Investigational Site	Fayetteville	Georgia
United States	Protagonist Investigational Site	Houston	Texas
United States	Protagonist Investigational Site	Houston	Texas
United States	Protagonist Investigational Site	Richmond	Virginia
United States	Protagonist Investigational Site	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Protagonist Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Mental Component Summary of SF-36	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).	Week 24 (or End of Treatment if treated for less than 24 weeks)
Other	Physical Component Summary of SF-36	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).	Week 24 (or End of Treatment if treated for less than 24 weeks)
Primary	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.	Week 24 (or End of Treatment if treated for less than 24 weeks)
Primary	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.	Week 24 (or End of Treatment if treated for less than 24 weeks)
Secondary	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.	Over 24 weeks