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A drug, panobinostat, to treat sickle cell disease (SCD)

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18 and older

Phase 1

1 Location

NCT01245179

Clinical Trial Goal


To find out:
  • The highest dose of panobinostat that is safe to give
  • If panobinostat is safe and works well to treat SCD

You may be able to join this trial if you:


  • Are 18 years old or older
  • Have SCD that has not gotten better with treatment 
  • Have complications from the SCD but do not need regular blood transfusions
  • Have not been treated with a drug that blocks HDAC, DAC, or HSP90. Your doctor can tell you this. 
  • Agree to have other standard tests done to see if you can be in the clinical trial

Trial Details


Panobinostat is a small molecule inhibitor that blocks HDAC in certain cells. Doctors think that panobinostat can help your body make hemoglobin F, which doesn’t turn into sickle cells.

Everyone in this trial will get standard care for SCD including pain medicines as needed.

You’ll also get panobinostat, a pill that you take 3 times each week for 3 months.

You'll have scans to see how well your heart is working. After you finish treatment, the clinical trial doctors will check your health for about 4 months.

The Food and Drug Administration (FDA) has approved panobinostat to treat multiple myeloma. Using it to treat SCD is new and unproven.

Locations


Augusta UniversityRECRUITING

Augusta, Georgia
Contacts:

ClinicalTrials.gov record


NCT01245179. First posted on 11/22/10

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