Could a cancer drug ease sickle cell pain? early trial launches

NCT ID NCT01245179

Recruiting now Disease control Sponsor: Abdullah Kutlar Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial is testing the safety of a drug called panobinostat in 18 adults with sickle cell disease. The drug aims to boost fetal hemoglobin and reduce inflammation, which may help control the disease. Participants take capsules three times a week for 12 weeks, and researchers will monitor for side effects and any improvements in blood counts or quality of life.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: panobinostat (a drug taken as capsules three times a week)
What this could lead to: If it works, this could point toward a new treatment option for sickle cell disease that reduces pain crises and hospitalizations.
What could go wrong: This is a very early Phase 1 trial with only 18 participants, so safety and effectiveness are not yet proven. The drug may cause side effects or fail to improve symptoms.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

beta thalassemia sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE

Contacts and locations

Locations

Augusta University
RECRUITING

Augusta, Georgia, 30912, United States

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