La Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta thalassemia | oneSCDvoice
  • Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials

La Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta thalassemia


La Jolla Pharmaceutical company today announced the initiation of a pivotal clinical study of LJPC‑401 (synthetic human hepcidin) in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. A high level of cardiac iron puts patients at risk of cardiac complications such as heart failure and sudden death.

LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study that is designed to enroll approximately 100 patients across 9 countries, including the United States. Patients will be randomized 1:1 to receive either: (i) weekly subcutaneous injections of LJPC‑401, while continuing standard-of-care chelation therapy (LJPC‑401 treatment arm); or (ii) a continuation of standard-of-care chelation therapy only (observation arm).

https://www.onescdvoice.com/wp-content/uploads/2017/12/LJPC.jpg