Ignite Creation Date:
2024-05-05 @ 11:20 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00007124
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2000-12-07
Brief Title:
Ketogenic Diet in Lafora Disease
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Trial of Ketogenic Diet in Lafora Disease
Status:
COMPLETED
Status Verified Date:
2002-11
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:
None
Brief Summary:
This study will examine the effect of a restricted-carbohydrate diet ketogenic diet on Lafora disease-a severe neurological disease in which brain cells die because of abnormal accumulation of glucose a type of sugar Patients with Lafora disease have rapid neurological deterioration with myoclonus brief muscle jerks seizures and mental decline At present there is no treatment to halt disease progression
Patients 10 years of age and older with relatively advanced Lafora disease may be eligible for this study Participants will be admitted to the Clinical Center for the first 4 weeks of this 6-month study for baseline testing and to start the ketogenic diet They will have a complete medical history and physical examination plus a detailed neurological examination and blood and urine tests Procedures include
Magnetic resonance imaging MRI brain scans to provide information about brain chemistry
Lumbar puncture spinal tap to analyze chemicals in cerebrospinal fluid
Metabolic and endocrinological tests including a glucose tolerance test to evaluate the bodys response to a large intake of oral glucose
Standard neuropsychological tests
Magnetic resonance spectroscopy of the brain and muscle
Electroencephalography EEG to measure brain wave activity
Electromyography EMG to measure muscle activity
Evoked potentials SEP and VEP to study brain responses to mild electric or visual stimulation
Transcranial magnetic stimulation magnetic stimulation of the brain may also be done to study the function of the brain cortex outer nervous tissue of the brain and the effects of treatment on brain excitability
The ketogenic diet will begin after the tests are completed The diet provides mainly fats to fuel the body plus the recommended amount of protein and minimum carbohydrate Vitamin and mineral supplements are provided to meet daily requirements After 2 weeks on the diet the patient will be discharged from the hospital and seen daily as an outpatient for another 1 to 2 weeks During this time the patient or caregiver is trained in preparing the ketogenic diet and then the patient is discharged to home Throughout the study disease symptoms will be assessed using standardized rating scales Blood and urine tests will be done as needed as will follow-up brain imaging neuropsychological and neurophysiological evaluations
A skin andor muscle biopsy may be done at the first clinic visit to grow skin cells in culture and to analyze the skin and muscle under a microscope The biopsy area is numbed with an anesthetic and a small piece of tissue is removed either with a needle an instrument similar to a cookie-cutter or a knife The skin cells may be used for metabolic studies and to obtain DNA for genetic testing
At the end of the study patients who responded well to the treatment with no significant adverse side effects may continue the diet for another 12 months They will be followed at 3-month intervals to monitor side effects and treatment response
Patients 10 years of age and older with relatively advanced Lafora disease may be eligible for this study Participants will be admitted to the Clinical Center for the first 4 weeks of this 6-month study for baseline testing and to start the ketogenic diet They will have a complete medical history and physical examination plus a detailed neurological examination and blood and urine tests Procedures include
Magnetic resonance imaging MRI brain scans to provide information about brain chemistry
Lumbar puncture spinal tap to analyze chemicals in cerebrospinal fluid
Metabolic and endocrinological tests including a glucose tolerance test to evaluate the bodys response to a large intake of oral glucose
Standard neuropsychological tests
Magnetic resonance spectroscopy of the brain and muscle
Electroencephalography EEG to measure brain wave activity
Electromyography EMG to measure muscle activity
Evoked potentials SEP and VEP to study brain responses to mild electric or visual stimulation
Transcranial magnetic stimulation magnetic stimulation of the brain may also be done to study the function of the brain cortex outer nervous tissue of the brain and the effects of treatment on brain excitability
The ketogenic diet will begin after the tests are completed The diet provides mainly fats to fuel the body plus the recommended amount of protein and minimum carbohydrate Vitamin and mineral supplements are provided to meet daily requirements After 2 weeks on the diet the patient will be discharged from the hospital and seen daily as an outpatient for another 1 to 2 weeks During this time the patient or caregiver is trained in preparing the ketogenic diet and then the patient is discharged to home Throughout the study disease symptoms will be assessed using standardized rating scales Blood and urine tests will be done as needed as will follow-up brain imaging neuropsychological and neurophysiological evaluations
A skin andor muscle biopsy may be done at the first clinic visit to grow skin cells in culture and to analyze the skin and muscle under a microscope The biopsy area is numbed with an anesthetic and a small piece of tissue is removed either with a needle an instrument similar to a cookie-cutter or a knife The skin cells may be used for metabolic studies and to obtain DNA for genetic testing
At the end of the study patients who responded well to the treatment with no significant adverse side effects may continue the diet for another 12 months They will be followed at 3-month intervals to monitor side effects and treatment response
Detailed Description:
The objective of this study is to evaluate the acute effect and potential disease modifying effects of a restrictive minimum carbohydrate diet ketogenic diet in patients with Lafora Disease Untreated Lafora Disease is rapidly progressive to death over about 10 years In an open label proof-of-principle clinical trial the efficacy of the ketogenic diet will be assessed through the use of validated clinical scales as well as surrogate neurophysiological and biochemical measures Safety will be monitored by means of frequent clinical evaluations and laboratory tests
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 01-N-0042 | None | None | None |