Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease (NCT01245179) | Clinical Trial Compass
RecruitingPhase 1
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
United States18 participantsStarted 2010-11
Plain-language 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.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Male or female patients ages ≥ 18 years
✓. Confirmed diagnosis of homozygous SS or S-β0Thalassemia
✓. Intolerance to hydroxyurea therapy, refusal of hydroxyurea therapy, or failure to respond (refractoriness) to hydroxyurea therapy, either clinically or hematologically.
✓. Clinically significant sickle cell disease as defined by:
✓. At least two hospitalizations over the past twelve months for any complication of sickle cell disease; or
✓. At least three pain crises over the past twelve months that last four or more hours and require a visit to a medical facility for treatment with oral or parenteral narcotics; or
✓. History of recurrent leg ulcers; or
✓. History of Acute Chest Syndrome within the past five years; or
Exclusion criteria
✕. Use of agents that can induce Hb F within 60 days of Day 1 (i.e. hydroxyurea, butyrates, decitabine, 5-azacytidine, IMiDs®, or erythropoietin). Prior use of HDACi, including panobinostat, is not an exclusion criterion if discontinued \> 60 days.
✕. Patients who have had a vaso-occlusive crisis within the past 2 weeks that required treatment with parenteral medication.
✕. Impairment of GI function or GI disease that may significantly alter the absorption of panobinostat (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
✕. Patients on a chronic transfusion regimen, or any patient who has Hb A% \> 20% from a recent transfusion
✕. Any of the following laboratory abnormalities derived from the screening visit:
✕. Known impaired cardiac function or clinically significant cardiac diseases, including any one of the following:
✕. Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes, active or uncontrolled infection, chronic obstructive or chronic restrictive pulmonary disease) that could cause unacceptable safety risks or compromise compliance with the protocol
✕. Patients who are currently receiving treatment with any study drug or have been on any study medications within the past 60 days.