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

La Jolla Pharmaceutical

Status and phase: Completed; Phase 2

Conditions: Hereditary Hemochromatosis

Treatments: Drug: Placebo; Drug: LJPC-401

Study type: Interventional

Funder types: Industry

Identifiers

NCT03395704

LJ401-HH01

Details and patient eligibility

About

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.

Enrollment: 70 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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70 participants in 2 patient groups, including a placebo group

LJPC-401

Active Comparator group

Description:

LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial

Treatment:

Drug: LJPC-401

Placebo

Placebo Comparator group

Description:

0.9% Sodium Chloride Injection, USP, or equivalent

Treatment:

Drug: Placebo