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

NCT03395704 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: LJ401-HH01
• Study Type: interventional
• Target: 70
• Locations: 4 countries
• Sites: 31

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.

Conditions

Hereditary Hemochromatosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Effect of LJPC-401 Versus Placebo on Number of Phlebotomies

  16 Weeks

• Effect of LJPC-401 Versus Placebo on Blood Iron Levels

  16 Weeks

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

  20 Weeks

Study Arms (2)

LJPC-401

ACTIVE COMPARATOR

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

Drug: LJPC-401

Placebo

PLACEBO COMPARATOR

0.9% Sodium Chloride Injection, USP, or equivalent

Drug: Placebo

Interventions

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.

Also known as: synthetic human hepcidin

LJPC-401

Placebo DRUG

0.9% Sodium Chloride Injection, USP, or equivalent

Also known as: Normal saline

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• La Jolla Pharmaceutical Company: lead
• PRA Health Sciences: collaborator

Study Sites (31)

Investigative Site

North Little Rock, Arkansas, 72117, United States

Location

Investigative Site

Los Angeles, California, 90036, United States

Location

Investigative Site

Palo Alto, California, 94305, United States

Location

Investigative Site

Rialto, California, 92377, United States

Location

Investigational Site

San Diego, California, 91942, United States

Location

Investigative Site

San Francisco, California, 94115, United States

Location

Investigative Site

Jacksonville, Florida, 32204, United States

Location

Investigative Site

Indianapolis, Indiana, 46202, United States

Location

Investigative Site

Wyoming, Michigan, 49519, United States

Location

Investigative Site

Jackson, Mississippi, 39216, United States

Location

Investigative Site

East Setauket, New York, 11733, United States

Location

Investigative Site

Manhasset, New York, 11030, United States

Location

Investigative Site

New York, New York, 10029, United States

Location

Investigative Site

Dallas, Texas, 75246, United States

Location

Investigative Site

Fort Worth, Texas, 76104, United States

Location

Investigative Site

Houston, Texas, 77058, United States

Location

Investigative Site

San Antonio, Texas, 78215, United States

Location

Investigative Site

Seattle, Washington, 98104, United States

Location

Investigative Site

Liverpool, New South Wales, 2170, Australia

Location

Investigative Site

Westmead, New South Wales, 2145, Australia

Location

Investigative Site

Brisbane, Queensland, 4120, Australia

Location

Investigative Site

Herston, Queensland, 4029, Australia

Location

Investigative Site

Melbourne, Victoria, 3004, Australia

Location

Investigative Site

Murdoch, Western Australia, 6150, Australia

Location

Investigative Site

Bondy, 93140, France

Location

Investigative Site

Orléans, 45000, France

Location

Investigative Site

Pessac, 33604, France

Location

Investigative Site

Rennes, 35033, France

Location

Investigative Site

Bradford, England, BD9 6RJ, United Kingdom

Location

Investigative Site

Newcastle upon Tyne, England, NE7 7DN, United Kingdom

Location

Investigative Site

Portsmouth, England, PO6 3LY, United Kingdom

Location

MeSH Terms

Conditions

Hemochromatosis

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Stew Kroll, MA
• Organization: La Jolla Pharmaceutical Company

skroll@ljpc.com

650-576-9679

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2017
• First Posted: January 10, 2018
• Study Start: November 29, 2017
• Primary Completion: October 28, 2019
• Study Completion: October 28, 2019
• Last Updated: June 9, 2022
• Results First Posted: June 9, 2022

Record last verified: 2022-05

Locations

GB (3); AU (6); FR (4); US (18)