Phase 2
N=16
PTG-300 in Subjects With Hereditary Hemochromatosis
Hereditary Hemochromatosis
Bottom Line
View on ClinicalTrials.gov: NCT04202965 ↗Enrolled (actual)
16
Serious AEs
6.3%
Results posted
Jun 2023
Primary outcome: Primary: Effect of PTG-300 on Transferrin Saturation — -6.07 percent saturation
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PTG-300 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Protagonist Therapeutics, Inc.
- Primary completion
- Oct 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Effect of PTG-300 on Transferrin Saturation |
-6.07 | — |
| PRIMARY Effect of PTG-300 on Serum Iron |
-2.34 | — |
| SECONDARY Effect of PTG-300 on Phlebotomy Frequency |
0.00 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT04202965). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.