A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
Status:
Completed
Trial end date:
2016-12-01
Target enrollment:
Participant gender:
Summary
The specific aim of this study is to determine whether Lovastatin ™ significantly improves
visual spatial learning and/or sustained attention in children with NF1.
Secondary Aims:
To evaluate the effect of Lovastatin ™ on measures of executive function, behavior and
quality of life in children with NF1 and cognitive deficits.
To further evaluate the toxicity and tolerability of Lovastatin ™ in children with NF1 and
cognitive deficits.
Hypotheses
It is hypothesized that Lovastatin ™ will improve the visual spatial memory and/or attention
deficits in children with NF1. This is based on studies demonstrating that Lovastatin ™ has
significantly improved impairments in visual spatial memory and attention in the NF1 murine
model.
It is further expected that Lovastatin ™ will be safe and well tolerated over a 16-week
period.
Phase:
Phase 2
Details
Lead Sponsor:
University of Alabama at Birmingham
Collaborators:
Boston Children's Hospital Boston Children’s Hospital Children's Hospital Medical Center, Cincinnati Children's Hospital of Philadelphia Children's National Research Institute Children's Research Institute National Cancer Institute (NCI) Sydney Children's Hospitals Network Sydney Children's Network University of Chicago University of Texas Southwestern Medical Center University of Utah Washington University School of Medicine