NCT02964884

Interventions for Reading Disabilities in NF1

Neurobiology and Treatment of Reading Disability in NF1

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Sponsor: Vanderbilt University · Academic / Other
• Sex: All
• Age: 8 Years – 20 Years
• Healthy volunteers: Accepted

Summary

Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of neurofibromin - a protein important in a signaling pathway that regulates learning and memory. Our previous work (NS49096) demonstrated that school-age children with NF+RD can respond to standard phonologically-based reading tutoring originally developed to treat reading disability in the general population. Combining our work with that by other researchers suggesting that a medication (Lovastatin) may counteract the effects of the deficient neurofibromin, and possibly ameliorate learning disabilities in NF1, the investigator propose to examine the synergistic effects of medication plus reading tutoring.

Detailed description

Neurofibromitosis Type 1 (NF1) is a common autosomal dominant neurocutaneous syndrome. The most common concern of NF1 is learning disabilities (LDs). Approximately half of children with NF1 have LDs, the most common of which are reading disabilities (RDs).The purpose of this research is to gain a deeper understanding of the characteristics and treatment of RDs in NF1. The Investigator will evaluate four different groups (see list below) to determine if there are differential outcomes in these groups of children with NF+RD. The first two groups will consist of NF1 patients, and the second two groups will consist of participants with RDs but without NF1. 1. Reading tutoring program and a medication called Lovastatin (NF1 patients) 2. Reading tutoring program and no Lovastatin (placebo) (NF1 patients) 3. Reading tutoring program (RD participants) 4. Other Academic "Sham" tutoring (eligible to receive reading tutoring after study participation is complete) (RD participants)

Conditions

• Neurofibromatosis Type 1
• Learning Disability
• Reading Disability
• NF1

Interventions

Type	Name	Description
DRUG	Lovastatin	Those in the Lovastatin group will receive 20mg of Lovastatin per day for the first two weeks and 40mg of Lovastatin per day starting at week 3 through week 11.
BEHAVIORAL	reading tutoring intervention	After the initial 12 weeks of Lovastatin or placebo, children will return for one week of intensive, one-on-one tutoring intervention (either reading or sham; Visit 2)
DRUG	Placebo Oral Tablet	Those in the placebo group will receive a tablet daily
BEHAVIORAL	Other Academic "sham" tutoring	No reading (sham) tutoring. Children will receive intensive tutoring on a subject other than reading.

Timeline

• Start date: 2016-11-01
• Primary completion: 2027-01-01
• Completion: 2027-01-01
• First posted: 2016-11-16
• Last updated: 2025-06-24

Locations

1 site across 1 country: United States