Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Status:
Active, not recruiting
Trial end date:
2025-03-01
Target enrollment:
Participant gender:
Summary
The goal of this clinical research study is to find out about the safety and effects of a
drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan
histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly
increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell
disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which
is a primary contributor to the debilitating effects of sickle cell disease. Given the
relevance of these mechanisms of action in SCD, panobinostat may prove to contribute
significantly to the management of SCD patients, a population in critical need of further
effective treatment options.