Interventions for Reading Disabilities in NF1
Neurofibromatosis Type 1, Learning Disability, Reading Disability
About this trial
This is an interventional treatment trial for Neurofibromatosis Type 1 focused on measuring Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
Eligibility Criteria
Inclusion Criteria:
This study will be open to all English speakers who meet eligibility criteria regardless of race, gender, minority or socioeconomic status. Inclusion criteria to be met are listed below:
- Individuals ages 8-20 (all participants)
- Documented NF-1 (NF patients only)
- If female, participant is post-menarche (NF patients only)
- If male, participant has reached Tanner Stage 2 (NF patients only)
- Participant able to swallow capsule (NF patients only, may be confirmed via swallowability test, described below)
- Participant's English is sufficient for school (all participants)
- Participant must either answer "yes" to question A, or answer "yes" to at least 2 items for questions B-D listed below:
A. Has your child ever been diagnosed with a reading disability? B. Did your child have trouble learning how to "sound out" words? C. Have you (parent) ever been concerned about your child's reading ability? D. Did the school or teacher ever express any concerns about your child's reading ability? Swallowability Assessment: We may ask patients to perform a capsule swallowability assessment prior to enrollment. We will ask subjects to attempt to swallow an empty capsule, matching the same size of the Lovastatin 20mg and 40mg capsules to be used in the study, to confirm swallowability. Patients will be provided with up to 2 capsules as needed to successfully perform the swallowability assessment. If the patient is unable to swallow the capsule, enrollment in the study may deferred or declined.
A child will be excluded if he/she meets any of the following criteria, which will be determined by initial telephone screening as well as review of medical/developmental history prior to and during testing:
- Child 7 years of age or less;
- known uncorrectable visual impairment;
- documented hearing impairment greater than or equal to a 25 dB loss;
- medical contraindication to MRI procedures (e.g., metal devices);
- any psychiatric, behavioral, or developmental disorder that would preclude active participation in in-depth tutoring sessions
- Pregnant at time of screening.
Known conditions which are contraindicated to Lovastatin
- Hypersensitivity to the medication
- Uncontrolled Epilepsy
- Metabolic Syndrome X, High Blood Sugar,
- Muscle Damage Due to Autoimmunity
- Stroke caused by Bleeding in the Brain, Loss of Memory,
- Severely Low Blood Pressure
- Liver Problems including Abnormal Liver Function Tests
- Severe Renal Impairment,
- Serious Muscle Damage that may Lead to Kidney Failure,
- Recent Operation or significant Injury
- Muscle Pain or Tenderness with Increase Creatinine Kinase,
- Habit of Drinking Too Much Alcohol
- Pregnant or lactating. - Lovastatin is contraindicated during pregnancy and in nursing mothers because it decreases synthesis of cholesterol and other products of the cholesterol biosynthesis pathway that are essential components for fetal development.
Children who meet criteria for ADHD, Oppositional Defiant Disorder, adjustment disorder, and/or mild depression will be eligible as long as they are not taking psychotropic medications, with the exception of stimulant medication for ADHD.
For pilot participants only:
Criteria for inclusion/exclusion for pilot participants is outlined below. Children who meet criteria for ADHD, Oppositional Defiant Disorder, adjustment disorder, and/or mild depression will be eligible as long as they are not taking psychotropic medications, with the exception of stimulant medication for ADHD.
- Children ages 8-17
- Participant is a native English speaker
- Participant must either answer "yes" to question A, or answer "yes" to at least 2 items for questions 2-4 listed below:
A. Has your child ever been diagnosed with a reading disability? B. Did your child have trouble learning how to "sound out" words? C. Have you (parent) ever been concerned about your child's reading ability? D. Did the school or teacher ever express any concerns about your child's reading ability?
A child will be excluded if he/she meets any of the following criteria, which will be determined by initial telephone screening as well as review of medical/developmental history prior to and during testing:
- Children 7 years of age or younger
- previous diagnosis of Intellectual Disability;
- known uncorrectable visual impairment;
- documented hearing impairment greater than or equal to a 25 dB loss;
- medical contraindication to MRI procedures (e.g., metal devices);
- known IQ below 70;
- a pervasive developmental disorder; and
- any known neurologic pathology, including epilepsy, spina bifida, cerebral palsy, traumatic brain injury, optic gliomas, and brain tumors (other than UBOs).
- Comorbid severe psychiatric disorders will also be excluded.
Sites / Locations
- Vanderbilt UniversityRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Active Comparator
Placebo Comparator
Placebo Comparator
Sham Comparator
NF1: Lovastatin + reading tutoring
NF1: Placebo + reading tutoring
RD: Reading tutoring
RD: Other Academic (sham) tutoring
Participants (NF-1 patients) will receive 20mg of Lovastatin per day for the first two weeks, the dose will be escalated 40 mg for the remaining 11 weeks. And one week of intensive, one-on-one reading tutoring intervention
Participants (NF-1 patients) will receive placebo daily And one week of intensive, one-on-one reading tutoring intervention
Participants (RD-only participants) will receive one week of intensive, one-on-one reading tutoring intervention
Participants (RD-only participants) will receive one week of intensive, one-on-one tutoring intervention with an academic focus other than reading. These participants will have the opportunity to receive reading tutoring upon finishing study participation.