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

NCT03381833 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: LJ401-BT01
• Study Type: interventional
• Target: 84
• Locations: 8 countries
• Sites: 22

Brief 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.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (1)

• Effect of LJPC-401 on cardiac iron

  Change in cardiac T2\* magnetic resonance imaging (MRI)

  26 Weeks

Secondary Outcomes (12)

• Effect of LJPC-401 on cardiac iron

  52 Weeks

• Effect of LJPC-401 on hepatic iron

  52 Weeks

• Effect of LJPC-401 on serum iron

  56 Weeks

• Effect of LJPC-401 on hemoglobin

  52 Weeks

• Effect of LJPC-401 on volume of blood transfused

  56 Weeks

Study Arms (2)

Group A - Delayed therapy

ACTIVE COMPARATOR

standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks

Drug: LJPC-401

Group B - Immediate therapy

ACTIVE COMPARATOR

standard chelation therapy plus LJPC-401 for 52 weeks

Drug: LJPC-401

Interventions

LJPC-401 DRUG

subcutaneous injection, up to 20 mg per week from week 26 to 52

Also known as: synthetic human hepcidin

Group A - Delayed therapy

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• La Jolla Pharmaceutical Company: lead

Study Sites (22)

Investigative Site

Oakland, California, 94609, United States

Location

Investigative Site

New York, New York, 10021, United States

Location

Investigative Site

Camperdown, New South Wales, 2050, Australia

Location

Investigative Site

Liverpool, New South Wales, 2170, Australia

Location

Investigative Site

Goudi, Attica, 115 27, Greece

Location

Investigative Site

Thessaloniki, Macedonia, 54642, Greece

Location

Investigative Site

Patra, Peloponnese, 26504, Greece

Location

Investigative Site

Cagliari, 09121, Italy

Location

Investigative Site

Modena, 41124, Italy

Location

Investigative Site

Napoli, 80138, Italy

Location

Investigative Site

Orbassano, 10043, Italy

Location

Investigative Site

Verona, 37134, Italy

Location

Investigative Site

Beirut, 1107 2020, Lebanon

Location

Investigative Site

Bangkok Noi, 10700, Thailand

Location

Investigative Site

Chiang Mai, 50200, Thailand

Location

Investigative Site

Adana, 01330, Turkey (Türkiye)

Location

Investigative Site

Ankara, 06100, Turkey (Türkiye)

Location

Investigative Site

Antalya, 07059, Turkey (Türkiye)

Location

Investigative Site

Antalya, 07160, Turkey (Türkiye)

Location

Investigative Site

Izmir, 35100, Turkey (Türkiye)

Location

Investigative Site

London, England, E1 1BB, United Kingdom

Location

Investigative Site

London, N19 5NF, United Kingdom

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2017
• First Posted: December 22, 2017
• Study Start: November 30, 2017
• Primary Completion: December 3, 2019
• Study Completion: January 14, 2020
• Last Updated: July 29, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

IT (5); TU (5); TH (2); GR (3); LE (1); GB (2); US (2); AU (2)