PTN hydroxyurea trial opens enrollment to younger participants

The PTN hydroxyurea trial has extended enrollment into Arm 1 of the study, which will target participants ages 2 to ≤5 years.

Enrollment into this trial is proceeding in two stages: the first stage (Arm 2) is focusing on children 5 to 17 years of age. Fourteen of 24 participants have been enrolled into this arm to date. Once the 8th patient was enrolled into this older cohort, the study team submitted an interim analysis of pharmacokinetic bioavailability and safety information to the Data and Safety Monitoring Board, which reviewed the data and recommended that the NICHD open enrollment of younger participants into Arm 1. The recommendation was approved on July 23, 2012.

Hydroxyurea represents the only major medical breakthrough in sickle cell disease in the past 20 years, and it is the only drug approved by the U.S. Food and Drug Administration (FDA) for use in adults with sickle cell. Treatment with hydroxyurea has been shown to significantly decrease the incidence of painful episodes and also reduces overall mortality in adult patients. In spite of the fact that the drug is not labeled for use in children, it is often used to treat children who exhibit signs of severe sickle cell disease. Recent data suggest that hydroxyurea therapy in infants with sickle cell is feasible, well-tolerated, and efficacious; however, only limited pharmacokinetic and pharmacodynamic studies of hydroxyurea use in children exist. The PTN is seeking to fill this knowledge gap.


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