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

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

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.

Study Overview

• Status: Terminated
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Drug: LJPC-401; Drug: LJPC-401

• Study Type: Interventional
• Enrollment (Actual): 84
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Investigative Site
    • Liverpool, New South Wales, Australia, 2170
      • Investigative Site
• Greece
  • Attica
    • Goudí, Attica, Greece, 115 27
      • Investigative Site
  • Macedonia
    • Thessaloníki, Macedonia, Greece, 54642
      • Investigative Site
  • Peloponnese
    • Patra, Peloponnese, Greece, 26504
      • Investigative Site
• Italy
  • Cagliari, Italy, 09121
    • Investigative Site
  • Modena, Italy, 41124
    • Investigative Site
  • Napoli, Italy, 80138
    • Investigative Site
  • Orbassano, Italy, 10043
    • Investigative Site
  • Verona, Italy, 37134
    • Investigative Site
• Lebanon
  • Beirut, Lebanon, 1107 2020
    • Investigative Site
• Thailand
  • Bangkok Noi, Thailand, 10700
    • Investigative Site
  • Chiang Mai, Thailand, 50200
    • Investigative Site
• Turkey
  • Adana, Turkey, 01330
    • Investigative Site
  • Ankara, Turkey, 06100
    • Investigative Site
  • Antalya, Turkey, 07059
    • Investigative Site
  • Antalya, Turkey, 07160
    • Investigative Site
  • İzmir, Turkey, 35100
    • Investigative Site
• United Kingdom
  • London, United Kingdom, N19 5NF
    • Investigative Site
  • England
    • London, England, United Kingdom, E1 1BB
      • Investigative Site
• United States
  • California
    • Oakland, California, United States, 94609
      • Investigative Site
  • New York
    • New York, New York, United States, 10021
      • Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Group A - Delayed therapy; standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks	Drug: LJPC-401; subcutaneous injection, up to 20 mg per week from week 26 to 52; Other Names: synthetic human hepcidin; Drug: LJPC-401; subcutaneous injection, up to 20 mg per week from week 1 to 52; Other Names: synthetic human hepcidin
Active Comparator: Group B - Immediate therapy; standard chelation therapy plus LJPC-401 for 52 weeks	Drug: LJPC-401; subcutaneous injection, up to 20 mg per week from week 26 to 52; Other Names: synthetic human hepcidin; Drug: LJPC-401; subcutaneous injection, up to 20 mg per week from week 1 to 52; Other Names: synthetic human hepcidin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of LJPC-401 on cardiac iron	Change in cardiac T2* magnetic resonance imaging (MRI)	26 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of LJPC-401 on cardiac iron	Change in cardiac T2*MRI	52 Weeks
Effect of LJPC-401 on hepatic iron	Change in hepatic T2*MRI	52 Weeks
Effect of LJPC-401 on serum iron	Mean change in serum iron	56 Weeks
Effect of LJPC-401 on hemoglobin	Mean change in hemoglobin	52 Weeks
Effect of LJPC-401 on volume of blood transfused	Change in transfused blood volume	56 Weeks
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
Effect of LJPC-401 on the incidence of treatment-emergent adverse events		56 Weeks
Effect of LJPC-401 on vital signs-heart rate		56 Weeks
Effect of LJPC-401 on vital signs-body temperature		56 Weeks
Effect of LJPC-401 on vital signs-respiratory rate		56 Weeks
Effect of LJPC-401 on vital signs-blood pressure		56 Weeks
Effect of LJPC-401 on body weight	Change in body weight (kilograms)	56 Weeks

Collaborators and Investigators

• Sponsor: La Jolla Pharmaceutical Company

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2017
• Primary Completion (Actual): December 3, 2019
• Study Completion (Actual): January 14, 2020

Study Registration Dates

• First Submitted: December 12, 2017
• First Submitted That Met QC Criteria: December 20, 2017
• First Posted (Actual): December 22, 2017

Study Record Updates

• Last Update Posted (Actual): July 29, 2021
• Last Update Submitted That Met QC Criteria: July 28, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Iron Metabolism Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Iron Overload; Thalassemia; beta-Thalassemia; Anti-Infective Agents; Hepcidins
• Other Study ID Numbers: LJ401-BT01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

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