Research OpportunityTitle: A Randomized Placebo-Controlled Study of Lovastatin in Children with Neurofibromatosis Type 1
This study is for patients diagnosed with neurofibromatosis type 1 (NF1), a gene disorder that affects the nervous system. The most common complications associated with NF1 in children are cognitive and learning difficulties. Despite the negative impact of this disorder on cognitive functioning, no studies have examined the effects of interventions (such as medications) on the cognitive functioning of children with NF1.
This study will determine whether lovastatin can improve cognitive function in children with NF1. Participants in this study will be randomized into one of two groups: lovastatin or a placebo control group (inactive substance). We will compare cognitive function in the two groups, to see whether treatment with lovastatin has resulted in an improvement in cognitive function relative to the placebo group.
The study will last for 24 weeks, which will include 16 weeks of treatment with lovastatin (taken orally every day) or a placebo control. A screening assessment will be performed to determine your child's eligibility for our study. This will include a medical history, blood work, and a physical examination to confirm the diagnosis of NF1. A psychologist or research assistant will also administer a brief measure of intelligence, a test of sustained attention and a test of visual memory. If your child satisfies the inclusion criteria you will be invited to participate in the study.
The timetable below indicates how often we will need to see your child. Please note that the first medical history and side-effects monitoring after commencing treatment (scheduled for week 2) will occur over the phone.
|Beginning of trial||2 weeks||4 weeks||8 weeks||12 weeks||16 weeks (end of treatment)||Follow-up (8 weeks after treatment)|
|Medical history/interim history||X||X||X||X||X||X||X|
|Drug compliance (drug diary check)||X||X||X||X||X|
- Must have a diagnosis of neurofibromatosis type 1 (NF1)*
- Must be between 10 and 16 years old
- Must have difficulty on a measure of sustained attention or spatial memory
The following consent form provides more details. If you think you may be eligible for this study and you would like to talk to someone in person about your interest in qualifying for or participating in this research, please contact us by phone or e-mail.
University of Alabama at Birmingham
Contact: Christina Barger, M.S., CGC (Clinic Coordinator)
Consent Form (pdf)
*This trial is specific to patients with neurofibromatosis type 1. There has been no evidence that individuals with neurofibromatosis type 2 or schwannomatosis would benefit from this clinical trial.