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

Part of paid clinical trials in Augusta, Georgia.

Sponsor
Abdullah Kutlar
Study ID
NCT01245179
Phase
PHASE1
Status
Recruiting

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • panobinostat — DRUG
    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

Study Details

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.

Key Dates

Start date
Nov 30, 2010
Status verified
Jan 2026
Primary completion
Dec 31, 2026
Completion
Jan 31, 2027

Study Design

Enrollment
18 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Panobinostat
    All patients will receive Panobinostat at specified dose levels and dosing schedules.

Primary Outcome Measure

Primary Outcome Measure [ Time Frame: Days 1, 8, 15, 22, 29, 43, 57, 85, 113 ]

Locations (1)

FacilityCityStateZIPSite coordinators
Augusta UniversityAugustaGeorgia30912
Leigh Wells, MSN, FNP
(706) 721-2171
Latanya Bowman, RN, BSN
706-721-2171

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