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New treatment option for Sickle Cell Disease
Official title Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
ClinicalTrials.gov ID: NCT01245179
What this study is testing
What is panobinostat?
panobinostat is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for sickle cell disease.
Also referred to as LBH589, LBH589 lactate.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join 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...
- 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
You likely can't join 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...
- 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...
- 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
See the full eligibility criteria
Who can join
- 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.
What rules you out
- 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...
- 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)
- 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)
- 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...
- Patients who are currently receiving treatment with any study drug or have been on any study medications within the past 60 days.
- Patients who have undergone major surgery 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.
- 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...
- 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...
- Known diagnosis of HIV infection, Hepatitis B; or acute/chronic, active Hepatitis C
- 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)
- 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Augusta, Georgia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Augusta, Georgia, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.