A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Beta-Thalassemia Clinical Trial

Official title:

A Multi-Center, Randomized, Open-Label, Parallel-Group Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03381833
• Other study ID #: LJ401-BT01
• Status: Terminated
• Phase: Phase 2
• Start date: November 30, 2017
• Est. completion date: January 14, 2020

Study information

• Verified date: July 2021
• 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, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with transfusion-dependent beta thalassemia with myocardial iron overload.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 84
• Est. completion date: January 14, 2020
• Est. primary completion date: December 3, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

Inclusion Criteria:

• Patients = 14 years of age with transfusion-dependent beta thalassemia.
• Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2*MRI from 6 to 35 msec; <= 15% difference between the two.
• Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose prior to study and expected to remain stable during study.
• Female patients of childbearing potential must not be pregnant, must have negative pregnancy tests, and must use an effective birth control method during the study.
• Male patients must be either surgically sterile or use an effective birth control method during the study.
• Patient must be willing and able to provide written informed consent. Parent of legal guardian to patients younger than age of majority must be willing and able to provide informed consent.

Exclusion Criteria:

• Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
• Pregnant or lactating women.
• Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
• Patients participating in an unapproved investigational clinical trial within 30 days of this study.
• Patients with a disease, disability or condition 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.
• Patients who are unwilling or unable to comply with the study requirements.
• Patients with known hepatitis B or hepatitis C, or being treated for a positive viral load or are noncompliant with hepatitis medications.
• Known and active human immunodeficiency virus (HIV) infection.
• Patients with Child Pugh class C cirrhosis or liver failure.
• Patients with severe congestive heart failure (NYHA Class 4).
• Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
• History of allergic reaction to hepcidin or excipients.
• Contraindication to MRI scanning.

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Thalassemia

Intervention

Drug:
• LJPC-401
subcutaneous injection, up to 20 mg per week from week 26 to 52
• LJPC-401
subcutaneous injection, up to 20 mg per week from week 1 to 52

Locations

Country	Name	City	State
Australia	Investigative Site	Camperdown	New South Wales
Australia	Investigative Site	Liverpool	New South Wales
Greece	Investigative Site	Goudí	Attica
Greece	Investigative Site	Patra	Peloponnese
Greece	Investigative Site	Thessaloníki	Macedonia
Italy	Investigative Site	Cagliari
Italy	Investigative Site	Modena
Italy	Investigative Site	Napoli
Italy	Investigative Site	Orbassano
Italy	Investigative Site	Verona
Lebanon	Investigative Site	Beirut
Thailand	Investigative Site	Bangkok Noi
Thailand	Investigative Site	Chiang Mai
Turkey	Investigative Site	Adana
Turkey	Investigative Site	Ankara
Turkey	Investigative Site	Antalya
Turkey	Investigative Site	Antalya
Turkey	Investigative Site	Izmir
United Kingdom	Investigative Site	London	England
United Kingdom	Investigative Site	London
United States	Investigative Site	New York	New York
United States	Investigative Site	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
La Jolla Pharmaceutical Company

Countries where clinical trial is conducted

United States,  Australia,  Greece,  Italy,  Lebanon,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effect of LJPC-401 on cardiac iron	Change in cardiac T2* magnetic resonance imaging (MRI)	26 Weeks
Secondary	Effect of LJPC-401 on cardiac iron	Change in cardiac T2*MRI	52 Weeks
Secondary	Effect of LJPC-401 on hepatic iron	Change in hepatic T2*MRI	52 Weeks
Secondary	Effect of LJPC-401 on serum iron	Mean change in serum iron	56 Weeks
Secondary	Effect of LJPC-401 on hemoglobin	Mean change in hemoglobin	52 Weeks
Secondary	Effect of LJPC-401 on volume of blood transfused	Change in transfused blood volume	56 Weeks
Secondary	Effect of LJPC-401 on its potential to elicit an immune response	Measured by blood laboratory tests and the presence of anti-drug antibodies	56 Weeks
Secondary	Effect of LJPC-401 on the incidence of treatment-emergent adverse events		56 Weeks
Secondary	Effect of LJPC-401 on vital signs-heart rate		56 Weeks
Secondary	Effect of LJPC-401 on vital signs-body temperature		56 Weeks
Secondary	Effect of LJPC-401 on vital signs-respiratory rate		56 Weeks
Secondary	Effect of LJPC-401 on vital signs-blood pressure		56 Weeks
Secondary	Effect of LJPC-401 on body weight	Change in body weight (kilograms)	56 Weeks