LaJolla Pharmaceuticals initiates a Phase 2 clinical trial assessing LJPC-401 (synthetic human hepcidin) in patients with hereditary hemochromatosis, a deficiency of the hormone hepcidin which leads to the accumulation of iron in the body. If left untreated, the disorder can lead to liver cirrhosis and/or cancer, heart disease/failure and diabetes.
The 60-subject study will evaluate weekly subcutaneous injections of LJPC-401 or placebo for 12 weeks. The primary endpoint is the change from baseline in transferrin saturation.
Transferrins are proteins that control the level of free iron in the blood and other body fluids.