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

Hereditary Hemochromatosis Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03395704
• Other study ID #: LJ401-HH01
• Status: Completed
• Phase: Phase 2
• Start date: November 29, 2017
• Est. completion date: October 28, 2019

Study information

• Verified date: May 2022
• Source: La Jolla Pharmaceutical Company
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: October 28, 2019
• Est. primary completion date: October 28, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemochromatosis
• Hereditary Hemochromatosis
• Iron Overload

Intervention

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.
• Placebo
0.9% Sodium Chloride Injection, USP, or equivalent

Locations

Country	Name	City	State
Australia	Investigative Site	Brisbane	Queensland
Australia	Investigative Site	Herston	Queensland
Australia	Investigative Site	Liverpool	New South Wales
Australia	Investigative Site	Melbourne	Victoria
Australia	Investigative Site	Murdoch	Western Australia
Australia	Investigative Site	Westmead	New South Wales
France	Investigative Site	Bondy
France	Investigative Site	Orléans
France	Investigative Site	Pessac
France	Investigative Site	Rennes Cedex 9
United Kingdom	Investigative Site	Bradford	England
United Kingdom	Investigative Site	Newcastle Upon Tyne	England
United Kingdom	Investigative Site	Portsmouth	England
United States	Investigative Site	Dallas	Texas
United States	Investigative Site	East Setauket	New York
United States	Investigative Site	Fort Worth	Texas
United States	Investigative Site	Houston	Texas
United States	Investigative Site	Indianapolis	Indiana
United States	Investigative Site	Jackson	Mississippi
United States	Investigative Site	Jacksonville	Florida
United States	Investigative Site	Los Angeles	California
United States	Investigative Site	Manhasset	New York
United States	Investigative Site	New York	New York
United States	Investigative Site	North Little Rock	Arkansas
United States	Investigative Site	Palo Alto	California
United States	Investigative Site	Rialto	California
United States	Investigative Site	San Antonio	Texas
United States	Investigational Site	San Diego	California
United States	Investigative Site	San Francisco	California
United States	Investigative Site	Seattle	Washington
United States	Investigative Site	Wyoming	Michigan

Sponsors (2)

Lead Sponsor	Collaborator
La Jolla Pharmaceutical Company	PRA Health Sciences

Countries where clinical trial is conducted

United States,  Australia,  France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Effect of LJPC-401 Versus Placebo on Number of Phlebotomies		16 Weeks
Secondary	Effect of LJPC-401 Versus Placebo on Blood Iron Levels	Change in serum ferritin as measured by blood laboratory tests	16 Weeks
Secondary	Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events		20 Weeks