Multi-center, randomized, open-label, parallel-group study with LPC-401 for the treatment of myocardial iron overload in adult patients with transfusion-dependent beta-thalassemia

NCT#/Phase: NCT03381833
Phase 2
IRB#: 2018-003
Description: This is a study of synthetic hepcidin in transfusion-dependent adults with beta-thalassemia and cardiac iron overload. This is an open-label study in which half of the participants will receive weekly subcutaneous injections of hepcidin in addition to their regular standard of care for 52 weeks. The other half will receive regular standard of care for the first 26 weeks and then will receive weekly hepcidin injections for the next 26 weeks in addition to their regular standard of care. The primary goal is to evaluate the effect of hepcidin on cardiac iron stores.
Inclusion: Individuals > 18 years of age with transfusion-dependent beta-thalassemia or E/Beta-thalassemia and cardiac iron overload.
Enrollment Status: Open for enrollment
Sponsor: La Jolla Pharmaceutical Company
Principal Investigator(s): Ashutosh Lal, MD
Contact(s): Ricarda Sawatzki (510-428-3885 x5361)
Jennifer Ferguson, RN, MSN, FNP (510-428-3885 x5324)
Study Link: