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Could a cholesterol drug boost reading skills in kids with NF1?

NCT ID NCT02964884

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 01, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastatin or a placebo daily for 12 weeks, then undergo one week of specialized reading or sham tutoring. The goal is to see if the medication enhances the brain's response to tutoring by targeting a protein deficiency linked to learning and memory in NF1.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Lovastatin
What this could lead to
If it works, this could point toward a treatment that combines medication with tutoring to improve reading skills in children with NF1.
What could go wrong
This is a Phase 2 study with a small number of participants, so results may not apply to everyone. Lovastatin may cause side effects like muscle pain or digestive issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 120 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2016

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

8 to 20 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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: 1. Individuals ages 8-20 (all participants) 2. Documented NF-1 (NF patients only) 3. If female, participant is post-menarche (NF patients only) 4. If male, participant has reached Tanner Stage 2 (NF patients only) 5. Participant able to swallow capsule (NF patients only, may be confirmed via swallowability test, described below) 6. Participant's English is sufficient for school (all participants) 7. 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: 1. Child 7 years of age or less; 2. known uncorrectable visual impairment; 3. documented hearing impairment greater than or equal to a 25 dB loss; 4. medical contraindication to MRI procedures (e.g., metal devices); 5. any psychiatric, behavioral, or developmental disorder that would preclude active participation in in-depth tutoring sessions 6. Pregnant at time of screening. 7. Known conditions which are contraindicated to Lovastatin 1. Hypersensitivity to the medication 2. Uncontrolled Epilepsy 3. Metabolic Syndrome X, High Blood Sugar, 4. Muscle Damage Due to Autoimmunity 5. Stroke caused by Bleeding in the Brain, Loss of Memory, 6. Severely Low Blood Pressure 7. Liver Problems including Abnormal Liver Function Tests 8. Severe Renal Impairment, 9. Serious Muscle Damage that may Lead to Kidney Failure, 10. Recent Operation or significant Injury 11. Muscle Pain or Tenderness with Increase Creatinine Kinase, 12. Habit of Drinking Too Much Alcohol 13. 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. 1. Children ages 8-17 2. Participant is a native English speaker 3. 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: 1. Children 7 years of age or younger 2. previous diagnosis of Intellectual Disability; 3. known uncorrectable visual impairment; 4. documented hearing impairment greater than or equal to a 25 dB loss; 5. medical contraindication to MRI procedures (e.g., metal devices); 6. known IQ below 70; 7. a pervasive developmental disorder; and 8. any known neurologic pathology, including epilepsy, spina bifida, cerebral palsy, traumatic brain injury, optic gliomas, and brain tumors (other than UBOs). 9. Comorbid severe psychiatric disorders will also be excluded.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Vanderbilt University

    Nashville, Tennessee, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.