Phase 2 Study of LJPC-401 for the Treatment of Myocardial Iron Overload in Adults with Transfusion-Dependent Beta Thalassemia Has Now Begun

January 11, 2018 – La Jolla Pharmaceutical Company has initiated a Phase 2 study to evaluate the effect of LJPC-401 (synthetic human hepcidin) on myocardial iron overload in adult patients with transfusion-dependent beta thalassemia. Hepcidin is a hormone that regulates iron absorption and distribution in the body. Hepcidin prevents excessive iron accumulation in vital organs such as the liver and the heart. La Jolla Pharmaceuticals is developing LJPC-401 for the potential treatment of iron overload.

100 participants will be enrolled in this 1 year study. To be eligible for this study, patients must meet the following criteria:

  • 18 years of age or older with transfusion-dependent beta thalassemia
  • Increased iron blood levels as measured by transferrin saturation (TSAT)
  • Increased iron levels in the heart as measured by MRI
  • Received iron chelation therapy for a minimum of 1 year and be on a stable dose prior to enrollment in the study

The primary outcome measure in this study is the effect of LJPC-401 on cardiac iron. Secondary outcome measures include the effect of LJPC-401 on blood iron levels, on hematology laboratory parameters, and on iron laboratory parameters.

In a phase I study of LJPC-401 in 15 patients with iron overload, no dose-limiting toxicities were observed at any dose level. LJPC-401 demonstrated a dose-dependent, statistically significant reduction in serum iron with maximum iron reduction at 8 hours and durable effect observed through the last day on Day 7.

The first clinical trial site for this Phase 2 study of LJPC-401 has now opened in San Diego, CA. Additional trial sites will open in the U.S. in the near future.

Further information about this trial is available at:


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