Phase I Study of Lovastatin for Neurocognitive Dysfunction in Children with Neurofibromatosis Type 1

We are currently looking for children with NF 1 and learning disabilities to participate in a research study in our Institution. We are trying to learn if Lovastatin, a medication that has been used in children and adults for the treatment of high cholesterol, can be helpful to improve learning disabilities in patients with NF1. Lovastatin has proven to be useful to improve cognitive deficits in a mice model of NF1. We would like to know if Lovastatin is also safe and useful in patients with NF1.

Who: Children with NF1 and history of learning disabilities within an age range of 10 -17 years will be eligible.

What: Participants in this study will undergo a full medical evaluation, blood tests and neuropsychological testing at the beginning of the treatment. Medication will be provided. Participants will come for a re-evaluation every 2 weeks for a total of 3 months. A second neuropsychological testing will be done at the end of the study.

Where: Children’s National Medical Center, 111 Michigan Av, N.W., Washington DC

Benefits: If medication is effective we will be able to develop a multi-center clinical trial. There is no cost for the research study. A summary of the neuropsychological testing will be provided at the end of the study. Parking is free. Patients will receive a gift certificate for each visit to the hospital.

Contact: Leah Seldin-Sommer, Study Coordinator at 202-884-4236, lseldins@cnmc.org or Dr. Maria Acosta, the Principal Investigator at 202-884-2120, macosta@cnmc.org.