Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

Recruiting now · Phase 1

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT01245179

Run by

Abdullah Kutlar

People needed

18

Starts

2010-11-01

Expected to finish

2027-01-01

Last updated by the study team

2026-01-09

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• History of priapism requiring medical intervention within the past two years; or
• History of stroke (but not currently on a chronic blood transfusion regimen).
• Ability to provide written informed consent obtained prior to participation in the study and any related procedures being performed.
• Clinically euthyroid. Note: Patients are permitted to receive thyroid hormone supplements to treat underlying hypothyroidism.

You may not qualify if…

• 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:
• Absolute neutrophil count (ANC) < 1.5 x 109/L
• Hemoglobin < 6 g/dl
• Platelets < 100x 109/L
• Serum creatinine >1.5 x Upper limits of normal (ULN)
• AST and ALT > 2.5 x ULN
• Serum total bilirubin > 10 mg/dL
• Serum direct bilirubin > 1 mg/dL
• Albumin <3.0 g/dl
• Serum potassium < Lower limits of normal (LLN)
• Total serum calcium [corrected for serum albumin] or ionized calcium <LLN
• Serum magnesium < LLN
• Serum phosphorus < LLN
• Known impaired cardiac function or clinically significant cardiac diseases, including any one of the following:
• Left ventricular ejection fraction (LVEF) < lower limit of the institutional normal as determined by screening echocardiogram
• Complete left bundle branch block
• Obligate use of a cardiac pacemaker
• Congenital long QT syndrome
• History or presence of ventricular tachyarrhythmia
• Presence of unstable atrial fibrillation (ventricular response > 100 bpm). Patients with stable atrial fibrillation are eligible, provided they do not meet any of the other cardiac exclusion criteria.
• Clinically significant resting bradycardia (< 50 bpm)

Where it is running

• Augusta University — Augusta, Georgia, United States (enrolling)

Full record on ClinicalTrials.gov

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