Clinical Trial 42920

Philadelphia, PA 19107


This study is designed to assess the safety and tolerability of PTG-300 in HH subjects. Also to assess the effect of PTG-300 therapy on transferrin saturation (TSAT) and serum ferritin. Also to assess PTG-300’s efficacy for treating HH subjects as defined by incidence of phlebotomy and assess the effect of PTG-300 on quality of life (QoL)


1.Male and female subjects aged 18 years or older.

2.Have a confirmed diagnosis of HFE-related hereditary hemochromatosis with prior genotype testing.

3.Documented stable phlebotomy for ≥6 months prior to screening; with a phlebotomy frequency of at least 0.25 per month (e.g., received at least three phlebotomies over the previous 12 months, or at least four phlebotomies over the previous 15 months) and a phlebotomy frequency of less than 1.0 per month.

4.Screening hemoglobin >11.5 g/dL.

5.Documented evidence of prior serum ferritin ≥500 ng/mL.

6.Serum ferritin >50 ng/mL and <300 ng/mL at screening (before screening phlebotomy).

7.Women of childbearing potential (WOCBP) and men with partners of childbearing potential agree to use a highly effective contraceptive measure (based on the Clinical Trials Facilitation Group [CTFG]) during the duration of the study and for 28 days after the last dose of study drug in the case of women and 90 days after the last dose of study drug in the case of men.

8.Subject understands the study procedures, is willing and able to adhere to study requirements and agrees to participate in the study by giving written informed consent.

Qualified Participants May Receive:

Compensation for time and travel.

Clinical trials are medical research studies designed to test the safety and/or effectiveness of new investigational drugs, devices, or treatments in humans. These studies are conducted worldwide for a range of conditions and illnesses. Learn more about clinical research and participating in a study at About Clinical Trials.