Actively Recruiting
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Led by Abdullah Kutlar · Updated on 2026-01-09
18
Participants Needed
1
Research Sites
843 weeks
Total Duration
On this page
Sponsors
A
Abdullah Kutlar
Lead Sponsor
S
Secura Bio, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients aged 18 years or older
- Confirmed diagnosis of homozygous SS or S-β0Thalassemia
- Intolerance to, refusal of, or failure to respond to hydroxyurea therapy
- Clinically significant sickle cell disease defined by at least two hospitalizations, three or more pain crises requiring medical treatment, history of leg ulcers, Acute Chest Syndrome in past five years, priapism requiring intervention in past two years, or history of stroke (not on chronic transfusion)
- Ability to provide written informed consent
- Clinically euthyroid, including those on thyroid hormone supplements for hypothyroidism
You will not qualify if you...
- Use of agents inducing Hb F within 60 days before Day 1 (hydroxyurea, butyrates, decitabine, 5-azacytidine, IMiDs®, erythropoietin); prior HDACi use allowed if discontinued over 60 days
- Vaso-occlusive crisis requiring parenteral medication within past 2 weeks
- Gastrointestinal disease affecting panobinostat absorption
- On chronic transfusion regimen or Hb A% over 20% from recent transfusion
- Certain lab abnormalities at screening (low neutrophils, hemoglobin, platelets; high creatinine, liver enzymes, bilirubin; low albumin, potassium, calcium, magnesium, phosphorus)
- Known impaired cardiac function or significant cardiac diseases
- Other severe or uncontrolled medical conditions increasing safety risks
- Current or recent treatment with study drugs within 60 days
- Recent major surgery or not recovered from surgery side effects
- Women pregnant, breastfeeding, or unwilling to use two birth control methods; men with partners of childbearing potential not using two methods
- Known HIV, Hepatitis B, or active Hepatitis C infection
- Prior malignancy within 5 years except certain skin or cervical cancers
- History of non-compliance with medical regimens or inability to follow study instructions
- Current treatment with prohibited medications without feasible discontinuation or switch
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Augusta University
Augusta, Georgia, United States, 30912
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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