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Neurofibromatosis Type 1Phase 2NCT02964884

Interventions for Reading Disabilities in NF1

A Phase 2 study testing Lovastatin and reading tutoring intervention in people with Neurofibromatosis Type 1 and Learning Disability, run by Vanderbilt University at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 8 to 20 yearsHealthy volunteers can take part
What is being tested?
Lovastatin and reading tutoring intervention
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
120 participants
Where
1 siteUnited States
Study dates
Nov 2016to Jan 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 20 years
  • All sexes
  • Healthy volunteers accepted
  • Child, Adult

Who may be able to join

  • 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)
Show all 47Show fewer
  • 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:
  • 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:
  • 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.

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Vanderbilt University

    Nashville, Tennessee

Other trials in United States

About this study

Official title: Neurobiology and Treatment of Reading Disability in NF1

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.

Read the 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)
Being tested
Lovastatin, reading tutoring intervention, Placebo Oral Tablet, Other Academic "sham" tutoring
Keywords
Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
Medical subject headings
Neurofibromatosis 1, Learning Disabilities, Dyslexia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LovastatinDrug

    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.

  • reading tutoring interventionBehavioural

    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)

  • Placebo Oral TabletDrug

    Those in the placebo group will receive a tablet daily

  • Other Academic "sham" tutoringBehavioural

    No reading (sham) tutoring. Children will receive intensive tutoring on a subject other than reading.

Study groups in NCT02964884
Study groupRoleWhat they receive
NF1: Lovastatin + reading tutoringActive comparatorParticipants (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
Receives: Drug: Lovastatin; Behavioral: reading tutoring intervention
NF1: Placebo + reading tutoringPlacebo comparatorParticipants (NF-1 patients) will receive placebo daily And one week of intensive, one-on-one reading tutoring intervention
Receives: Behavioral: reading tutoring intervention; Drug: Placebo Oral Tablet
RD: Reading tutoringPlacebo comparatorParticipants (RD-only participants) will receive one week of intensive, one-on-one reading tutoring intervention
Receives: Behavioral: reading tutoring intervention
RD: Other Academic (sham) tutoringSham comparatorParticipants (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.
Receives: Behavioral: Other Academic "sham" tutoring

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 24 weeks

  2. 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).

    Measured: 24 weeks

  3. 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.

    Measured: 24 weeks

  4. 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).

    Measured: 24 weeks

  5. 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.

    Measured: 24 weeks

  6. 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.

    Measured: 24 weeks

  7. Delis Kaplan Executive Function System

    Sorting from the D-KEFS will be used to assess higher-level planning and shifting abilities.

    Measured: 24 weeks

  8. 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.

    Measured: 24 weeks

  9. 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.

    Measured: 24 weeks

  10. Clinical Evaluation of Language Fundamentals-Fifth Edition

    CELF-5 will be used to assess aspects of oral language (e.g., syntax, semantics, \& working memory).

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Nov 2016 (estimated)
  2. Study startNov 2016
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
120 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02964884
Sponsor's protocol ID
161159
Other IDs
1R01HD089474-01
Status last verified
Jun 2026
First submitted
9 Nov 2016
Last updated
1 Jul 2026
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Lovastatin and reading tutoring intervention in people with Neurofibromatosis Type 1 and Learning Disability, run by Vanderbilt University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 47 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Woodcock Johnson Tests of Achievement IV (time frame: 24 weeks). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02964884.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2026.

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Interventions for Reading Disabilities in NF1 | AzTrials