The NF1 Statin Randomized Study (NF1 STARS): A Randomized-Placebo Controlled Study of Lovastatin in Children with Neurofibromatosis Type 1

GOALS

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

DRUGS

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

This is a Phase II study involving children with NF1 (aged 8 years to less than 16 years at time of screening) with evidence of cognitive impairment, defined as having a score of at least one standard deviation or more below the population mean on a measure of visual spatial learning and/or attention.

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.

PROTOCOL CHAIRS

• Kathryn North, MD, Chair
• Belinda Barton, PhD, Co-Chair
• Jonathan Payne, MD, Co-Chair
• Maria Acosta, MD, Co-Chair
• Gerard A. Gioia, PhD, Co-Chair

CURRENT STATUS

The study is complete and closed. Please see trial results via publications or visit clinicaltrials.gov/ct2/show/NCT00853580.