Terminated early for lack of efficacy as determined by interim endpoint analyses

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.

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.