Trusted Resources: News & Events
Latest announcements and gatherings
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).
+myBinderRelated Content
-
Progress in pursuit of sickle cell cureBao, in collaboration with Baylor Colleg...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Prevention of acute chest syndrome by implementing a standardized process to improve incentive spirometry use in hos...Acute chest syndrome (ACS) is the second...
-
Sickle Cell Disease Hotline | Cayenne Wellness Center and Sickle Cell Disease Education & AwarenessPatients living with Sickle Cell Disease...
-
Hospitals See No Link Between US Opioid Crisis and Patients’ Use of Treatment, Study ReportsThe U.S. opioid epidemic is not linked t...
-
New digital education platform for the SCD communityhttps://www.onescdvoice.com/wp-content/u...
-
Endari reduces pain crises, hospitalizations in sickle cell patients, phase 3 trial showsTreatment with Endari (L-glutamine) le...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.