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Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

NCT01245179 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-01-09

No results posted yet for this study

Summary

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. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.

Conditions

Interventions

DRUG

panobinostat

Panobinostat oral capsules taken THRICE WEEKLY (Monday, Wednesday, and Friday) for 12 weeks, exploring the following dosing regimens: 1. 15 mg MWF 3 weeks on, 1 week off (if needed) 2. 15 mg MWF every week (starting dose) 3. 20 mg MWF 3 weeks on, 1 week off 4. 20 mg MWF every week

Sponsors & Collaborators

  • Secura Bio, Inc.

    collaborator INDUSTRY

  • Abdullah Kutlar

    lead OTHER

Principal Investigators

  • Abdullah Kutlar, MD · Augusta University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-11-30
Primary Completion
2026-12-31
Completion
2027-01-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01245179 on ClinicalTrials.gov