This study investigates the safety and effects of panobinostat in adults with sickle cell disease, a condition where red blood cells become misshaped and break down. Panobinostat is a type of medicine called an HDAC inhibitor, which can help increase a helpful type of hemoglobin, called hemoglobin F, and reduce inflammation that worsens sickle cell disease. This study aims to find the safest dose and understand how well patients tolerate the drug.
Participants will go through three main phases: Screening, Treatment, and Follow-Up. The Screening Phase ensures participants are eligible and involves health checks and lab tests. In the Treatment Phase, lasting 12 weeks, participants take the drug three times a week. The Follow-Up Phase occurs four weeks after treatment ends to monitor health changes.
- Study duration: 12 weeks of treatment plus follow-up.
- Eligibility: Adults (18+) with specific types of sickle cell disease not responding to other treatments.
- Risks: Possible side effects include changes in blood tests and mild discomfort.