PTG-300 in Subjects With Hereditary Hemochromatosis (NCT04202965) | Clinical Trial Compass
CompletedPhase 2
PTG-300 in Subjects With Hereditary Hemochromatosis
United States16 participantsStarted 2020-03-19
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Effect of PTG-300 on Transferrin Saturation
Timeframe: Week 24 (or End of Treatment if treated for less than 24 weeks)
2
Effect of PTG-300 on Serum Iron
Timeframe: Week 24 (or End of Treatment if treated for less than 24 weeks)