A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

Study Overview
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Brief Summary
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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.

Official Title
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A Phase 2, Multi-Center, Randomized, Placebo Controlled, Single-Blind Study With LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

Conditions
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Hereditary Hemochromatosis

Intervention / Treatment
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* Drug: LJPC-401
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with clinical diagnosis of hereditary hemochromatosis Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis Patients with serum ferritin and TSAT levels above treatment guidelines 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 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 Patient must be willing and able to provide written informed consent Exclusion Criteria: Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug Pregnant or lactating women Patients taking an immunosuppressive agent without prior Sponsor approval Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug Patients who are unwilling or unable to comply with the study protocol requirements Patients with type 1 or poorly controlled type 2 diabetes 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: LJPC-401<br>LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial | Drug: LJPC-401<br>* 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.<br>* Other names: synthetic human hepcidin;|
| Placebo Comparator: Placebo<br>0.9% Sodium Chloride Injection, USP, or equivalent | Drug: Placebo<br>* 0.9% Sodium Chloride Injection, USP, or equivalent<br>* Other names: Normal saline;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests. | 16 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect of LJPC-401 Versus Placebo on Number of Phlebotomies |  | 16 Weeks | 
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Change in serum ferritin as measured by blood laboratory tests | 16 Weeks | 
| Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events |  | 20 Weeks |