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Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) (NF1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00352599
Recruitment Status : Completed
First Posted : July 14, 2006
Last Update Posted : November 22, 2016
Information provided by (Responsible Party):
University of California, Los Angeles

Brief Summary:

Neurofibromatosis type I (NF1) is a genetic disorder that affects approximately 1 in 3500 individuals. Half of people with NF1 inherit the condition from a parent, and half have a new occurrence of the condition. The manifestation of NF1 is highly variable and multiple organ systems are typically affected. Some of the more common symptoms include benign neurofibromas, café au lait spots, Lisch nodules (tan spots on the iris of the eye). Some individuals with NF1 also exhibit more severe associated conditions, such as optic pathway tumors (gliomas) or bones bending or curving. Neurocognitive deficits and specific learning disabilities occur in approximately 30 to 50% of individuals with NF1 and are regarded by some observers and sufferers to be among the most troubling features of a disease. The most commonly reported findings are deficits in visuoperceptual ability, motor coordination, expressive and receptive language, and executive functioning, which requires intact short-term memory and attention. Patients with NF1 also show a slight depression in mean IQ scores compared to healthy adults without the disorder.

While cognitive deficits are now a widely-recognized feature of Neurofibromatosis Type 1 (NF1), the precise cause of these deficits still remain to be determined. Dr. Alcino Silva, a co- investigator on this study, has developed an animal model of NF1 in which mice have a specific mutation of the *NF1* gene. These mice are physically normal but show specific learning impairments. Dr. Silva's lab found that treatment with a medication called lovastatin, a drug typically used for high cholesterol, reversed some of the spatial deficits seen in these animals. Lovastatin is a medication commonly used to treat high cholesterol and has been proven to be relatively safe and tolerable in humans.

The investigators are now conducting a randomized, double-blinded, placebo- controlled, trial of lovastatin in patients with NF1. Participants will be randomly assigned to lovastatin or placebo and treated for approximately 14 weeks with baseline and follow-up assessments to evaluate safety and any effects on neurocognitive test performance.

Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Drug: Lovastatin Drug: placebo pill Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)
Study Start Date : September 2009
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014

Arm Intervention/treatment
Active Comparator: Lovastatin
Drug: Lovastatin
Drug: Lovastatin
Lovastatin capsules daily for 14 weeks (titrated up from 10 mg to 40 mg)

Placebo Comparator: Placebo pill
Placebo pill
Drug: placebo pill

Primary Outcome Measures :
  1. Non-verbal learning /working memory [ Time Frame: 14 weeks ]

Secondary Outcome Measures :
  1. attention [ Time Frame: 14 weeks ]
  2. tolerability of medication [ Time Frame: 14 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. a diagnosis of NF1 by NIH criteria
  2. between 10 and 50 years of age
  3. no evidence of a comorbid neurological disorder (e.g., epilepsy, encephalitis)
  4. not currently taking a statin medication
  5. not suffering from hypercholesterolemia based on self-report, collateral information from physician, or initial medical workup using National Cholesterol Education Program (NCEP, JAMA 2001), guidelines accepted by the American College of Cardiology (ACC) and the American Heart Association (AHA)
  6. does not have any of the aforementioned conditions that contraindicates use of statin medications (such as pregnancy, lactation, liver disease, or use of other medication not recommended for use in conjunction with lovastatin). A negative pregnancy test will be required if the patient is a female in reproductive years.
  7. not mentally retardation (i.e., IQ greater than 70)
  8. no evidence of significant and habitual alcohol or drug abuse or dependence
  9. sufficient acculturation and fluency in the English language to avoid invalidating research measures of thought, language, and speech disorder, and verbal abilities.
  10. lives in Southern California area (or can arrange ~5 visits to Los Angeles over 14 weeks)

Exclusion Criteria:

  1. comorbid neurological conditions
  2. significant drug or alcohol abuse
  3. non-fluency in English

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00352599

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United States, California
Semel Institute for Neuroscience and Human Behavior
Los Angeles, California, United States, 90095
Sponsors and Collaborators
University of California, Los Angeles
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Principal Investigator: Carrie E Bearden, PhD University of California, Los Angeles

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Responsible Party: University of California, Los Angeles Identifier: NCT00352599     History of Changes
Other Study ID Numbers: 05-08-069-01
First Posted: July 14, 2006    Key Record Dates
Last Update Posted: November 22, 2016
Last Verified: November 2016
Keywords provided by University of California, Los Angeles:
Neurofibromatosis Type 1
Additional relevant MeSH terms:
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Neurofibromatosis 1
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
L 647318
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors