Study of Panobinostat for Sickle Cell Disease
This study is testing a drug called panobinostat in adults with sickle cell disease. Panobinostat is being explored because it may increase hemoglobin F (a type of hemoglobin that can improve sickle cell disease) and reduce inflammation, which contributes to the disease's effects. The main goal is to understand how safe panobinostat is and what its effects are. You might be able to join if you are 18 or older, have a confirmed diagnosis of sickle cell disease (homozygous SS or S-β0Thalassemia), and have not responded to, cannot tolerate, or refuse hydroxyurea therapy. The study is also looking for people with clinically significant sickle cell disease. The current status of the study is unclear, and it plans to enroll 18 participants.
- Study design
- This is a Phase I, single-arm, open-label study involving 18 participants. It will test different doses and schedules of panobinostat to find the maximum tolerated dose.
- What's involved
- You would take panobinostat capsules three times a week for 12 weeks. Before starting, you will have screening assessments including a physical exam, blood tests, and heart tests.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your progress will be measured at several points over 113 days, which is about 16 weeks.
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NCT01245179
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Recruiting
PHASE1Ages 18+InterventionalTreatmentAbdullah KutlarInvestigator-initiated
~18 participants
Updated 2026-01-09 on ClinicalTrials.gov
What's tested:panobinostat
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Primary Outcome Measure
Measured over Days 1, 8, 15, 22, 29, 43, 57, 85, 113
Conditions
Where it's being run
1 sites across 1 statesGeorgia1
Study leadership
- Abdullah Kutlar, MD · PRINCIPAL_INVESTIGATOR · Augusta University
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Exclusion
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 6. 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
Clinically significant resting bradycardia (\< 50 bpm)
QTc \> 470 msec on screening ECG
Right bundle branch block + left anterior hemiblock (bifasicular block)
Angina pectoris 3 months prior to starting study drug
Acute MI 3 months prior to starting study drug
Other clinically significant heart disease (e.g., CHF, uncontrolled hypertension, history of labile hypertension, or history of poor compliance with an antihypertensive regimen) 7. 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 8. Patients who are currently receiving treatment with any study drug or have been on any study medications within the past 60 days. 9. Patients who have undergone major surgery 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy. 10. Women of child-bearing potential (WCBP) who are pregnant or breast feeding or who do not agree to use two methods of birth control, including a barrier method, if they are sexually active. WCBP, defined as sexually mature women who have not undergone a hysterectomy or who have not been naturally postmenopausal for at least 12 consecutive months (i.e., who has had menses any time in the preceding 12 consecutive months), must have a negative serum pregnancy test at screening and negative urine pregnancy test within 72 hours prior to starting study treatment. In addition, all sexually active WCBP must agree to use double method of contraception (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) during the study and 3 months after the end of treatment. One of these methods of contraception must be a barrier method. 11. Male patients whose sexual partners are WCBP not using a double method of contraception during and 3 months after the end of treatment. Males must agree to use a condom during any sexual contact with WCBP during study drug treatment, during dose interruptions, and for 3 months after the end of treatment. 12. Known diagnosis of HIV infection, Hepatitis B; or acute/chronic, active Hepatitis C 13. Patients with a prior malignancy with in the last 5 years (except for basal or squamous cell carcinoma, or in situ cancer of the cervix) 14. Patients with any significant history of non-compliance to medical regimens or unwilling or unable to comply with the instructions given to him/her by the study staff. 15. Patients who are currently receiving treatment with certain prohibited medications and cannot either discontinue this treatment or switch to a different medication prior to study enrollment.
What this trial measures
- Primary Outcome MeasureDays 1, 8, 15, 22, 29, 43, 57, 85, 113
To determine the safety and dose limiting toxicities of escalating doses of oral panobinostat in sickle cell disease