Sickle-cell and Other Hemoglobinopathies

SMART (Sickle-cell Multicenter Aspirin Response Trial)
The Hemoglobinopathy Program is preparing for participation in the National Institutes of Health Sickle Cell Multicenter Aspirin Response Trial (SMART).  This national trial evaluates the use of aspirin to prevent central nervous system strokes in patients with sickle cell disease.

ASSERT - A Stratified Sickle Event Randomized Trial
The goal of this clinical trial is to reduce the number of acute sickle-related painful episodes in those patients with sickle cell disease. The investigational drug ICA-17043 will be compared to placebo in patients with sickle cell disease who have had two or more sickle-related painful crises within the preceding 12 months. This study is being conducted by Icagen, Inc., and is not yet open to accrual.

Enzyme Replacement Therapy for Individuals with MPS II (Hunter Syndrome)
We are collaborating with the Pediatric Genetics Department to conduct a study involving individuals with MPS II receiving idursulfase enzyme replacement therapy. Transkaryotic Therapies, Inc. is sponsoring this trial which will only be open to subjects who completed the double-blind portion of the trial.