Phase 1 Trial of Hepcidin Formulation is Enrolling

March 6, 2015 – San Diego-based La Jolla Pharmaceutical Company is currently seeking qualified patients for consideration in their trial in Patients at Risk for Iron Overload using its novel formulation of hepcidin (LJPC-401).

Hepcidin, an endogenous peptide hormone, is a naturally occurring regulator of iron absorption and distribution. Manipulation of hepcidin has the potential to prevent excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of diseases such as beta thalassemia and sickle cell disease (SCD).

LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing involing animals. La Jolla’s Phase 1 clinical trial of LJPC-401 in patients at risk of iron overload is currently ongoing. Phase 1 trials are intended to evaluate safety and potential dosing requirements and are not intended to evaluate efficacy.

La Jolla is currently seeking patients with conditions putting them at risk for iron overload for a Phase 1 clinical trial at study centers in the United States.

Enrollment criteria include the following:

Patients with any of the following disorders:

  1. Refractory or hemolytic anemia of any type. Examples include, but are not limited to, hemoglobinopathies, sideroblastic anemia, myelodysplastic syndrome (MDS), and congential anemias


Who have been transfused with at least 2 units of blood over the past two (2) months, OR 4 units of blood over the past six (6) months, OR received any form of iron chelation therapy over the past six (6) months, OR have a serum ferritin > 1,000 µg/L.

2. Patients with hemochromatosis that require phlebotomy at least once every two months OR have received iron chelation therapy in the past six (6) months.

Patients 18-85 years of age, inclusive.

Anyone wishing more information about this study may contact the following clinical trial sites:

Cincinnati, OH @ Jennifer Palmer – (513) 721-3868

San Diego, CA @ Ruth Garcia – (877) 500-3788

The Cooley’s Anemia Foundation provides information on select clinical trials that may be of interest to the thalassemia population. This information is provided for educational purposes only and does not imply an endorsement of any trial. Patients who are considering participating in a clinical trial and do not know what questions to ask may want to consult with CAF Patient Outreach Director Sandy Gilbert ( who can help them determine what questions they should ask the investigators to determine if a trial is right for them.

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