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Histone Deacetylase Inhibitor

Panobinostat for Sickle Cell Disease (LBH589 Trial)

Phase 1
Recruiting
Led By Abdullah Kutlar, MD
Research Sponsored by Abdullah Kutlar
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of homozygous SS or S-β0Thalassemia
- History of priapism requiring medical intervention within the past two years
Must not have
- Congenital long QT syndrome
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up days 1, 8, 15, 22, 29, 43, 57, 85, 113
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group

Summary

This trial is testing a drug called panobinostat to see if it is safe and effective in treating adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor, which has been shown to increase hemoglobin F induction and inhibit cell-specific inflammation.

Who is the study for?
Adults over 18 with sickle cell disease who haven't responded well to or can't take hydroxyurea. They should have had at least two hospitalizations or three pain crises in the past year, or a history of other severe complications related to sickle cell disease. People with certain blood counts, organ dysfunctions, heart conditions, active infections like HIV/Hepatitis B/C, recent surgeries, and women who are pregnant/breastfeeding or not using contraception are excluded.
What is being tested?
The trial is testing panobinostat's safety and effectiveness for adults with sickle cell disease. Panobinostat is an HDAC inhibitor that may increase hemoglobin F levels and reduce inflammation—both potentially beneficial for managing this condition.
What are the potential side effects?
Panobinostat could cause side effects such as changes in blood counts leading to increased risk of infections or bleeding, nausea, fatigue, diarrhea, liver problems (elevated enzymes), electrolyte imbalances (like low calcium/magnesium/potassium), heart issues including abnormal rhythms and QT prolongation which can be serious.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with SS or S-β0 Thalassemia.
Select...
I have needed treatment for a prolonged erection in the last two years.
Select...
I cannot tolerate or have not responded to hydroxyurea treatment.
Select...
I've been hospitalized at least twice in the last year for sickle cell complications.
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I've had 3 or more painful episodes in the last year needing hospital visits for strong painkillers.
Select...
I have had leg ulcers more than once.
Select...
I have had Acute Chest Syndrome in the last 5 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a condition that affects my heart's rhythm.
Select...
I cannot stop or change my current medication for the study.
Select...
I have a heart condition or significant heart disease.
Select...
I've had a severe pain crisis needing injection medication in the last 2 weeks.
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I do not have severe stomach or bowel problems that could affect medication absorption.
Select...
I regularly receive blood transfusions or had one recently affecting my hemoglobin.
Select...
I have had episodes of very fast heartbeats.
Select...
I had major surgery more than 2 weeks ago and have recovered from it.
Select...
I have been diagnosed with HIV, Hepatitis B, or Hepatitis C.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~days 1, 8, 15, 22, 29, 43, 57, 85, 113
This trial's timeline: 3 weeks for screening, Varies for treatment, and days 1, 8, 15, 22, 29, 43, 57, 85, 113 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Measure
Secondary study objectives
Define mechanisms of effect of panobinostat (Hb F induction and anti-inflammatory effects) and discover biomarkers of treatment response
Measure

Side effects data

From 2013 Phase 1 & 2 trial • 28 Patients • NCT01105312
56%
Platelet count decreased
56%
Anemia
38%
Hypocalcemia
31%
Electrocardiogram QT corrected interval prolonged
31%
Creatinine increased
31%
White blood cell decreased
25%
Neutrophil count decreased
13%
Anorexia
13%
Fatigue
13%
Nausea
13%
Hypermagnesemia
6%
Hyperkalemia
6%
Alanine aminotransferase increased
6%
Edema limbs
6%
Hypophosphatemia
6%
Lung infection
6%
Vomiting
6%
Fracture
6%
Weight loss
6%
Diarrhea
6%
Glucose intolerance
6%
Blood bilirubin increased
6%
Dyspnea
6%
Hypertension
100%
80%
60%
40%
20%
0%
Study treatment Arm
Phase II
Phase I: Dose Level One
Phase I: Dose Level Two

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: PanobinostatExperimental Treatment1 Intervention
All patients will receive Panobinostat at specified dose levels and dosing schedules.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Panobinostat
FDA approved

Find a Location

Who is running the clinical trial?

Secura Bio, Inc.Industry Sponsor
8 Previous Clinical Trials
201 Total Patients Enrolled
Abdullah KutlarLead Sponsor
Abdullah Kutlar, MDPrincipal InvestigatorAugusta University
1 Previous Clinical Trials
26 Total Patients Enrolled

Media Library

Panobinostat (Histone Deacetylase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT01245179 — Phase 1
Sickle Cell Disease Research Study Groups: Panobinostat
Sickle Cell Disease Clinical Trial 2023: Panobinostat Highlights & Side Effects. Trial Name: NCT01245179 — Phase 1
Panobinostat (Histone Deacetylase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01245179 — Phase 1
~1 spots leftby Dec 2025
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