A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Condition: Hereditary Hemochromatosis
MeSH terms: Hemochromatosis; Iron Overload; Hemosiderosis
Intervention: LJPC-401; Placebo
Intervention description: LJPC-401 subcutaneous injection, up to 20 mg weekly for 16 weeks. The minimum weekly dose will be 5 mg and the maximum weekly dose of LJPC-401 will be 20 mg.; 0.9% Sodium Chloride Injection, USP, or equivalent
Intervention MeSH terms: Hepcidins
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Enrollment: 70.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This study is a Phase 2 multicenter, randomized, placebo controlled, single-blind study. The
      primary objective of the study is to compare the effect of weekly dosing of LJPC-401
      (synthetic human hepcidin) versus placebo on transferrin saturation (TSAT) in an adult
      hereditary hemochromatosis patient population.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patients with clinical diagnosis of hereditary hemochromatosis

          2. Patients who are prescribed therapeutic phlebotomy for treatment of hereditary
             hemochromatosis

          3. Patients with serum ferritin and TSAT levels above treatment guidelines

          4. Female patients of child bearing potential must have a negative pregnancy test and
             must be using a highly effective method of contraception during participation in the
             study, and for 30 days after the last dose of study drug

          5. Males must be surgically sterile (vasectomy), or using a highly effective method of
             contraception during participation in the study, and for 30 days after the last dose
             of study drug

          6. Patient must be willing and able to provide written informed consent

        Exclusion Criteria:

          1. Patients receiving iron chelation therapy within 7 days prior to the first dose of
             study drug

          2. Patients initiating phlebotomy treatments less than 3 months prior to the first dose
             of study drug

          3. Pregnant or lactating women

          4. Patients taking an immunosuppressive agent without prior Sponsor approval

          5. Patients participating in an unapproved investigational drug or investigational
             therapeutic device within 30 days of study drug

          6. Patients who are unwilling or unable to comply with the study protocol requirements

          7. Patients with type 1 or poorly controlled type 2 diabetes

          8. Patients with a concomitant disease, disability or condition, including laboratory
             abnormality and ECG findings, which may interfere with the conduct of the study, or
             which would, in the opinion of the Investigator, pose an unacceptable risk to the
             patient in this study, including, but not limited to, clinically significant
             arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric
             disease