La Jolla Pharmaceutical Company has initiated a phase II clinical study of LJPC-401 in patients with transfusion-dependent beta thalassaemia [ClinicalTrials.gov Identifier: NCT03381833]. LJPC-401 is La Jolla’s proprietary formulation of synthetic human hepcidin, the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in the heart and other vital organs, where it can cause potentially fatal complications. La Jolla is developing LJPC-401 for the potential treatment of iron overload, with promising results reported in preclinical and phase I clinical studies. The phase II clinical study is a multinational, multicentre, randomized, controlled study designed to involve 100 patients from 40 sites across 9 countries. The primary outcome measure in this study is the effect of LJPC-401 on myocardial iron overload in patients receiving chelation therapy. Secondary outcome measures include the effect of LJPC-401 on blood iron levels, on hematology laboratory parameters, and on iron laboratory parameters.

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

Eligibility criteria include:

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

More information: La Jolla announcement, CAF News, LJPC , Clinical trial