A Phase 2 interventional study of Lovastatin and reading tutoring intervention in Neurofibromatosis Type 1, Learning Disability and Reading Disability, sponsored by Vanderbilt University. Active, not recruiting at 1 site in United States. Open to participants aged 8 Years to 20 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-07-01.
Sponsored by Vanderbilt University · Phase 2, Interventional, and Treatment
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.
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.
176 studies on the registry are indexed under Neurofibromatosis 1; 43 are open to participants now.
This study's planned enrollment of 120 is above the median of 35 across 114 interventional studies indexed under Neurofibromatosis 1.
Browse Neurofibromatosis 1 studies →Vanderbilt University is the lead sponsor of 522 studies on the registry; 20 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 5 (63%) have results posted.
Counted across the registry records on this site, refreshed daily.
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:
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:
Known conditions which are contraindicated to Lovastatin
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.
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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: Lovastatin · Behavioral: reading tutoring intervention
Participants (NF-1 patients) will receive placebo daily And one week of intensive, one-on-one reading tutoring intervention
Behavioral: reading tutoring intervention · Drug: Placebo Oral Tablet
Participants (RD-only participants) will receive one week of intensive, one-on-one reading tutoring intervention
Behavioral: 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.
Behavioral: Other Academic "sham" tutoring
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.
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)
Those in the placebo group will receive a tablet daily
No reading (sham) tutoring. Children will receive intensive tutoring on a subject other than reading.
Woodcock Johnson Tests of Achievement IV
WJ-IV (Forms A and B) Letter Word Identification, Word Attack, and Passage Comprehension subtests will be administered in order to assess word recognition accuracy, decoding accuracy, and comprehension.
Time frame: 24 weeks
Woodcock Reading Mastery Test-III
(WRMT-III; Forms A and B); Word Identification, Word Attack, and Passage Comprehension will be administered to assess word recognition accuracy, decoding accuracy, and comprehension. The WRMT-III and WJ-III correlate at .87, and at the item level show similar characteristics (e.g., containing 31% and 34% multisyllabic words, respectively).
Time frame: 24 weeks
Test of Word Reading Efficiency-2
(TOWRE-2; Forms A, B, C, D) Sight Word Efficiency and Pseudoword Decoding will be used to assess real and pseudoword reading speed.
Time frame: 24 weeks
Experimental Word and Pseudoword Lists
At each visit, 2 word and 2 pseudoword lists (20 words each) will be administered. Word lists are matched on relevant characteristics (e.g., frequency, bigram frequency). Real word lists have already been developed and tested, and show high inter-correlations (r =.87-.97).
Time frame: 24 weeks
Test of Silent Contextual Reading Fluency-2
(TOSCRF-2; 91; Forms A, B, C, D). This test requires identifying as many words as possible in 3 min within a sentential context.
Time frame: 24 weeks
Comprehensive Test of Phonological Processing-2
CTOPP-2 core subtests will be used to assess phonological processing, a known predictor of decoding and word recognition.
Time frame: 24 weeks
Delis Kaplan Executive Function System
Sorting from the D-KEFS will be used to assess higher-level planning and shifting abilities.
Time frame: 24 weeks
Visuospatial Learning and Memory, Morris Water Maze
The child "hops" from square to square in the grid, repeated from various starting points. After landing on the target square, a fun goal pops up. The number of "hops" to reach the goal is measured. A control probe trial is given in which there is no goal.
Time frame: 24 weeks
Visuospatial Learning and Memory, Judgment of Line Orientation
(JLO, Forms J and H)98 requires the judgment of spatial relationships between two lines compared to a reference of a protractor-like half-circle of 11 lines. The Investigator will create 4 forms from the two already existing by splitting each test in half by selecting every other item, a method used by others (Leach et al., 2003). This will allow us to generate 4 different versions of the JLO.
Time frame: 24 weeks
Clinical Evaluation of Language Fundamentals-Fifth Edition
CELF-5 will be used to assess aspects of oral language (e.g., syntax, semantics, \& working memory).
Time frame: 24 weeks
Plan to share: No
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This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Lovastatin