Interventions for Reading Disabilities in NF1

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.

Conditions

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

Interventions

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.

Sponsors & Collaborators

• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

  collaborator NIH

• Vanderbilt University

  lead OTHER

Principal Investigators

• Laurie Cutting, PhD · Vanderbilt University Medical Center

• Sheryl L. Rimrodt-Frierson, MD · Vanderbilt University Medical Center

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

8 Years

Max Age

20 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2016-11-30

Primary Completion

2027-01-31

Completion

2027-01-31

Countries

• United States

Study Locations