A Randomized-Placebo Controlled Study of Lovastatin in Children with Neurofibromatosis Type 1

Goal

The STARS trial is designed to evaluate the effectiveness of Lovastatin in the treatment of learning disabilities in children with NF1.

Drug

Lovastatin is a drug commonly used to lower cholesterol levels in the blood. It is being tested because results in an animal study suggest that it may improve cognitive function, perhaps through altered regulation of the cell signaling pathway that is involved in NF1.

Eligibility

The STARS trial is open to children between ages 8 years and 15 years, 11 months. They must fulfill diagnostic criteria for NF1, have a full scale IQ greater than 70, and have cognitive impairment that can be demonstrated with standardized tests used in the study. They must be willing to cooperate with the testing and treatment in the study, and cannot have medical problems that prevent their participation. A full list of criteria can be obtained by contacting one of the study sites.

Protocol

The STARS study is a placebo controlled, double-blinded study. This means that participants will receive either the medication or a placebo and neither the patient/family nor their doctor will know which they are receiving. Assignment to receive either medication or placebo will be random. Treatment will continue for 16 weeks, during which time a number of tests of cognitive function will be done on a regular schedule, as well as monitoring for side effects of the medication. There will be continued monitoring of cognitive function for 8 weeks following treatment. Further details about the protocol can be obtained by contacting one of the study sites.

Current Status

The STARS trial is currently sending publication (Fall 2016).

Further Information

For further information, please contact one of the study sites or the Operations Center.