Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Dana-Farber Cancer Institute
Study ID
NCT01000155
Phase
PHASE2
Status
Terminated

Conditions

Eligibility Criteria

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

Interventions

Study Details

Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape. People who have SCD have a different type of hemoglobin (protein that carries oxygen). This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood and can cause inflammation and injury to important areas of the body. All babies are born with hemoglobin called fetal hemoglobin (HbF). Soon after birth, HbF production slows down and another hemoglobin called adult hemoglobin (HbA) is made. Clinical studies have shown that increasing the amount of HbF in the blood may prevent sickling of the red blood cells. Vorinostat has been used in the treatment of cancers and in other research studies and information from those suggests that it may help treat SCD by increasing the amount of HbF in the blood. The purpose of this research study is to determine the effectiveness and safety of vorinostat when used to treat SCD.

Key Dates

Start date
Oct 31, 2009
Status verified
Jun 2017
Primary completion
Oct 31, 2014
Completion
Oct 31, 2014

Study Design

Enrollment
5 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Vorinostat
    Patients received vorinostat in a pulsed fashion, once a day for 3 consecutive days every week to a maximum dose of 400 mg per dose (1200 mg/wk), for 12 to 16 weeks at the maximum dose. The first 3 patients were enrolled in an intrapatient dose escalation schedule of 100 mg/d, then 200 mg/d, each for 3 consecutive days a week for 4 weeks, and then 400 mg/d, 3 consecutive days per week for 16 weeks. The last 2 patients were enrolled to receive 400 mg/d, 3 consecutive days per week for 12 weeks, without an initial dose escalation.

Primary Outcome Measure

Percent Fetal Hemoglobin (HbF%) Induction Success Rate [ Time Frame: HbF% was measured at baseline and weekly on treatment. Median duration of treatment was 3 months. ]

Locations (3)

FacilityCityStateZIPSite coordinators
Brigham and Women's HospitalBostonMassachusetts02115-
Children's Hospital BostonBostonMassachusetts02115-
Dana-Farber Cancer InstituteBostonMassachusetts02115-

Find similar trials in Boston, MA

By condition
Sickle cell disease
By research site
Brigham and Women's Hospital· Boston, MAChildren's Hospital Boston· Boston, MADana-Farber Cancer Institute· Boston, MA

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