Ignite Creation Date:
2024-05-05 @ 4:57 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00352599
Status:
COMPLETED
Last Update Posted:
2016-11-22
First Post:
2006-07-13
Brief Title:
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I NF1
Sponsor:
University of California Los Angeles
Organization:
University of California Los Angeles
Study Overview
Official Title:
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I NF1
Status:
COMPLETED
Status Verified Date:
2016-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
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 Silvas 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
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 Silvas 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None