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NCT Number: NCT03395704

A Study of 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.

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Key information

Conditions

Hereditary Hemochromatosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemochromatosis Iron Metabolism Disorders Iron Overload Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigative Site, Liverpool, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

LJPC-401

Drug

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

Drug

0.9% Sodium Chloride Injection, USP, or equivalent

Other names: Normal saline

Primary outcomes

  1. Effect of LJPC-401 Versus Placebo on Blood Iron Levels

    Time frame: 16 Weeks

    Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.

Secondary outcomes

  1. Effect of LJPC-401 Versus Placebo on Number of Phlebotomies

    Time frame: 16 Weeks

  2. Effect of LJPC-401 Versus Placebo on Blood Iron Levels

    Time frame: 16 Weeks

    Change in serum ferritin as measured by blood laboratory tests

  3. Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events

    Time frame: 20 Weeks

Sponsors and collaborators

Lead sponsor

La Jolla Pharmaceutical Company

Industry

Collaborators

  • PRA Health Sciences

Registry information

Official study title

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

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Jan 10, 2018
Registry last updated
Jun 9, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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