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

Phase 2

Completed

• Conditions: Hereditary Hemochromatosis
• Interventions: Drug: Placebo; Drug: LJPC-401

• Registration Number: NCT03395704
• Lead Sponsor: La Jolla Pharmaceutical Company

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-11-29
• Study First Submitted: 2017-12-12
• Study First Submitted QC: 2018-01-03
• Study First Posted: 2018-01-10
• Primary Completion: 2019-10-28
• Study Completion: 2019-10-28
• Results First Submitted: 2021-12-15
• Status Verified: 2022-05
• Results First Submitted QC: 2022-05-18
• Last Update Submitted: 2022-05-18
• Results First Posted: 2022-06-09
• Last Update Posted: 2022-06-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	0.9% Sodium Chloride Injection, USP, or equivalent
LJPC-401	LJPC-401	LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (2)

Investigational Site; 🇺🇸 San Diego, California, United States

Investigative Site; 🇬🇧 Portsmouth, England, United Kingdom