First patient enrolled in hydroxyurea study

Breaking news announcement

On January 27, 2012, the first patient was successfully enrolled into the PTN’s hydroxyurea (HU) study.

Hydroxyurea is the only major drug breakthrough for the treatment of sickle cell disease within the past 20 years. It is the only drug approved by the FDA to treat adults with sickle cell anemia, and so it is a part of the standard of care for those with severe sickle cell disease in the United States.

Few studies have been done with HU in children to tell us how it is absorbed and expelled. The PTN study will measure how much HU gets into the bloodstream at different time points in children, and how fast it is removed. The study will enroll eligible children ages 2–17 with a confirmed diagnosis of sickle cell anemia. For the children ages 5–17, the study will also explore whether the body handles HU differently in liquid versus pill form.

Study enrollment for all ages will take place during regularly scheduled clinic visits. Children will take their usual dose of HU, and small blood samples will be obtained over the course of a day. Study results will provide information to help the FDA in considering approval of HU for use in infants and children with sickle cell anemia.

The first enrolled patient provided the required blood draws over a full day in the clinic at Children’s Memorial Hospital in Chicago, IL, under the direction of Dr. Robert Liem, the study site principal investigator. The HU study plans to enroll 40 patients total over the course of several months.

Related: Hydroxyurea in pediatric patients with sickle cell anemia