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

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Brief Title

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

Official Title

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

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.
    


Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Non-verbal learning /working memory

Secondary Outcome

 attention

Condition

Neurofibromatosis 1

Intervention

Lovastatin

Study Arms / Comparison Groups

 Lovastatin
Description:  Lovastatin

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

44

Start Date

September 2009

Completion Date

March 2014

Primary Completion Date

March 2014

Eligibility Criteria

        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
      

Gender

All

Ages

10 Years - 50 Years

Accepts Healthy Volunteers

No

Contacts

Carrie E Bearden, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00352599

Organization ID

05-08-069-01


Responsible Party

Sponsor

Study Sponsor

University of California, Los Angeles


Study Sponsor

Carrie E Bearden, PhD, Principal Investigator, University of California, Los Angeles


Verification Date

November 2016