Clinical trial NCT04202965: PTG-300 in Subjects With Hereditary Hemochromatosis
Official title: Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Hemochromatosis
Lead sponsor: Protagonist Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-12-18
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single Arm Study
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Effect of PTG-300 on Transferrin Saturation
timeFrame: Week 24 (or End of Treatment if treated for less than 24 weeks)
measure: Effect of PTG-300 on Serum Iron
timeFrame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Secondary outcomes:
measure: Effect of PTG-300 on Phlebotomy Frequency
timeFrame: Over 24 weeks
Other outcomes:
measure: Mental Component Summary of SF-36
timeFrame: Week 24 (or End of Treatment if treated for less than 24 weeks)
measure: Physical Component Summary of SF-36
timeFrame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Locations:
city: Fayetteville
country: United States
facility: Protagonist Investigational Site
state: Georgia
city: Bethesda
country: United States
facility: Protagonist Investigational Site
state: Maryland
city: Charlotte
country: United States
facility: Protagonist Investigational Site
state: North Carolina
city: Conroe
country: United States
facility: Protagonist Investigational Site
state: Texas
city: Dallas
country: United States
facility: Protagonist Investigational Site
state: Texas
city: Houston
country: United States
facility: Protagonist Investigational Site
state: Texas
city: Houston
country: United States
facility: Protagonist Investigational Site
state: Texas
city: Richmond
country: United States
facility: Protagonist Investigational Site
state: Virginia
city: Seattle
country: United States
facility: Protagonist Investigational Site
state: Washington
city: Halifax
country: Canada
facility: Protagonist Investigational Site
state: Nova Scotia
city: Toronto
country: Canada
facility: Protagonist Investigational Site
state: Ontario
Study officials:
References: