La Jolla Pharmaceutical Company has started a pivotal clinical study of synthetic human hepcidin (LJPC-401) to treat patients with transfusion-dependent beta-thalassemia who have cardiac iron levels above normal, despite chelation therapy.
The trial will be a multinational, multicentre, randomised, controlled study that expects to enrol around 100 patients across nine countries, including the US.
The study is designed as part of an agreement signed between La Jolla and European Medicines Agency (EMA).
La Jolla president and CEO George Tidmarsh said: "We look forward to continuing the research and development efforts of LJPC-401, with a goal of helping patients suffering from iron overload disorders."
During the trial, patients will be randomised 1:1 to receive either weekly subcutaneous injections of LJPC-401, while continuing standard-of-care chelation therapy (LJPC-401 treatment arm) or a continuation of standard-of-care chelation therapy only (observation arm).
After completing six months, patients randomised to the observation arm will be allowed to receive LJPC-401 (plus standard-of-care chelation therapy) for six months, and patients randomised to the LJPC-401 treatment arm will continue with LJPC-401 (plus standard-of-care chelation therapy) for another six months.
The primary efficacy endpoint of the trial is to change the iron content in the heart after six months of treatment, as measured by cardiac magnetic resonance imaging (MRI).
In September last year, La Jolla reported positive results from a Phase l study of LJPC-401 carried out in patients at-risk of iron overload and suffering from hereditary hemochromatosis, thalassemia, and SCD.