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

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

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Hemochromatosis
• Intervention / Treatment: Drug: LJPC-401; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 70
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Liverpool, New South Wales, Australia, 2170
      • Investigative Site
    • Westmead, New South Wales, Australia, 2145
      • Investigative Site
  • Queensland
    • Brisbane, Queensland, Australia, 4120
      • Investigative Site
    • Herston, Queensland, Australia, 4029
      • Investigative Site
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • Investigative Site
  • Western Australia
    • Murdoch, Western Australia, Australia, 6150
      • Investigative Site
• France
  • Bondy, France, 93140
    • Investigative Site
  • Orléans, France, 45000
    • Investigative Site
  • Pessac, France, 33604
    • Investigative Site
  • Rennes Cedex 9, France, 35033
    • Investigative Site
• United Kingdom
  • England
    • Bradford, England, United Kingdom, BD9 6RJ
      • Investigative Site
    • Newcastle Upon Tyne, England, United Kingdom, NE7 7DN
      • Investigative Site
    • Portsmouth, England, United Kingdom, PO6 3LY
      • Investigative Site
• United States
  • Arkansas
    • North Little Rock, Arkansas, United States, 72117
      • Investigative Site
  • California
    • Los Angeles, California, United States, 90036
      • Investigative Site
    • Palo Alto, California, United States, 94305
      • Investigative Site
    • Rialto, California, United States, 92377
      • Investigative Site
    • San Diego, California, United States, 91942
      • Investigational Site
    • San Francisco, California, United States, 94115
      • Investigative Site
  • Florida
    • Jacksonville, Florida, United States, 32204
      • Investigative Site
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Investigative Site
  • Michigan
    • Wyoming, Michigan, United States, 49519
      • Investigative Site
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • Investigative Site
  • New York
    • East Setauket, New York, United States, 11733
      • Investigative Site
    • Manhasset, New York, United States, 11030
      • Investigative Site
    • New York, New York, United States, 10029
      • Investigative Site
  • Texas
    • Dallas, Texas, United States, 75246
      • Investigative Site
    • Fort Worth, Texas, United States, 76104
      • Investigative Site
    • Houston, Texas, United States, 77058
      • Investigative Site
    • San Antonio, Texas, United States, 78215
      • Investigative Site
  • Washington
    • Seattle, Washington, United States, 98104
      • Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: La Jolla Pharmaceutical Company
• Collaborators: PRA Health Sciences

Study record dates

Study Major Dates

• Study Start (Actual): November 29, 2017
• Primary Completion (Actual): October 28, 2019
• Study Completion (Actual): October 28, 2019

Study Registration Dates

• First Submitted: December 12, 2017
• First Submitted That Met QC Criteria: January 3, 2018
• First Posted (Actual): January 10, 2018

Study Record Updates

• Last Update Posted (Actual): June 9, 2022
• Last Update Submitted That Met QC Criteria: May 18, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Iron Metabolism Disorders; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Iron Overload; Hemochromatosis; Hemosiderosis; Anti-Infective Agents; Hepcidins
• Other Study ID Numbers: LJ401-HH01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No