Breakthrough Clinical Trial May Provide Relief for Thalassemia

La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients who, despite other therapies, test high for cardiac iron. Keep reading to learn more, or follow the original source here.
Thalassemia is a disorder of the blood. Typically, thalassemia is characterized by a lack of hemoglobin in the blood. In more extreme cases, known as beta thalassemia intermermedia, and beta thalassemia major, patients may develop a surplus of iron in their bodies. In the case of cardiac iron, an overload may result in heart failure, or sudden death. Click here for more information on thalassemia.

La Jola Pharmaceutical tests LJPC-401 (synthetic human hepcidin) in hopes that it will be able to alter the iron content of the heart. The study lasts six months, during which scientists test patients’ cardiac iron through cardiac magnetic resonance imaging.

Ja Jolla describes the study for LJPC-401 as a “pivotal, multinational, multi-center, randomized, controlled study.” Researchers designed the study to include 100 patients spanning nine different countries. Researchers intend to randomize the patients 1:1. Patients groups include those who receive LJPC-401 on a weekly basis alongside standard chelation therapy, and those receiving only chelation. After six months, patients from the second group will join those of the first in receiving the experimental treatment for another six months.

The study represents a significant and potentially historic moment for researchers in the field of blood diseases.

LJPC-401 exists as the first opportunity to explore, in humans, the effects of a “master regulator of body iron.” This refers to the nature of LJPC-401 as a synthetic version of the body’s own hepcidin which naturally controls the amount of iron in the human body.

“We are pleased to initiate this pivotal study at leading research centers in the U.S. and worldwide,” says La Jolla’s President and Chief Executive Officer, George F. Tidmarsh, M.D., Ph.D.

“We look forward to continuing the research and development efforts of LJPC-401, with a goal of helping patients suffering from iron overload disorders.”

Further details on the study can also be found here or by searching with the identifier NCT03381833.

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