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Trial to Evaluate the Safety of Lovastatin in Adults With Neurofibromatosis Type I (NF1)
This study is currently recruiting participants.
Verified by University of California, Los Angeles, July 2006
Sponsored by: University of California, Los Angeles
Information provided by: University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT00352599
  Purpose

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 NF1, the precise cause of these deficits still remain to be determined. Dr. Alcino Silva, a co-investigator on the proposed project, 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; however, there is no information about its safety specifically among people with NF1. Our goal is to first establish the safety of this medication among individuals with NF1 and then, if safe, move on to conducting a study to evaluate whether this medication might be helpful in treatment of cognitive problems in individuals diagnosed with NF1. We are conducting a randomized, double-blinded, placebo-controlled, safety study of lovastatin in patients with NF1. Participants will be randomly assigned to lovastatin or placebo and treated for approximately 8 weeks with baseline and follow-up assessments to evaluate safety and any effects on neurocognitive test performance.


Condition Intervention Phase
Neurofibromatosis 1
Drug: Lovastatin
Phase I

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies neurofibromatosis type 1 neurofibromatosis type 2
MedlinePlus related topics: Neurofibromatosis
Drug Information available for: Lovastatin
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title: Trial to Evaluate the Safety of Lovastatin in Adults With Neurofibromatosis Type I (NF1)

Further study details as provided by University of California, Los Angeles:

Estimated Enrollment: 45
  Eligibility

Ages Eligible for Study:   18 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. a diagnosis of NF1 by NIH criteria
  2. between 18 and 45 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.

Exclusion Criteria:

  1. comorbid neurological conditions
  2. significant drug or alcohol abuse
  3. non-fluency in English
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00352599

Contacts
Contact: Molly E Hardt (310) 794-9730 hardt@lifesci.ucla.edu

Locations
United States, California
Clinical Neuroscience Laboratory Recruiting
Los Angeles, California, United States, 90095
Contact: Molly E Hardt     310-794-9730     hardt@lifesci.ucla.edu    
Principal Investigator: Tyrone Cannon, PhD            
Sponsors and Collaborators
University of California, Los Angeles
Investigators
Principal Investigator: Tyrone Cannon, PhD University of California, Los Angeles
  More Information

Study ID Numbers: 05-08-069-01
Study First Received: July 13, 2006
Last Updated: July 13, 2006
ClinicalTrials.gov Identifier: NCT00352599  
Health Authority: United States: Institutional Review Board

Keywords provided by University of California, Los Angeles:
Neurofibromatosis Type 1
NF1
Lovastatin
statin

Study placed in the following topic categories:
Neurodegenerative Diseases
Neurofibromatosis 1
Neurofibromatosis type 1
Neoplastic Syndromes, Hereditary
Heredodegenerative Disorders, Nervous System
Neurofibroma
Neuromuscular Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neurofibromatoses
Nerve Sheath Neoplasms
Lovastatin
Neurocutaneous Syndromes

Additional relevant MeSH terms:
Antimetabolites
Neoplasms
Neoplasms by Histologic Type
Molecular Mechanisms of Pharmacological Action
Therapeutic Uses
Antilipemic Agents
Nervous System Diseases
Neoplasms, Nerve Tissue
Enzyme Inhibitors
Anticholesteremic Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009