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View Clinical Trial (Medical Research Study)
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Cholesterol in ASD: Characterization and Treatment - NCT00965068-43210 (Clinical Trial 306821)
Permalink: http://www.ClinicalConnection.com/exp/ExpandedPatientViewStudy306821.aspx
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| City: |
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Columbus |
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State:
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OH |
| Zip Code: |
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43210 |
| Conditions: |
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Autism - Asperger Disorder - PDD-NOS |
| Purpose: |
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Background:
- Autism spectrum disorders (ASD) are developmental disabilities characterized by impaired
social interaction and repetitive and/or stereotypical behaviors. Research studies suggest
that some individuals with ASD have very low blood cholesterol levels. This low cholesterol
level and other abnormal sterol levels may be important markers for
subtypes of ASD. Providing additional cholesterol to the diets of children with ASD may help
improve behavior.
- These findings will guide the medical community in identifying individuals who should be
tested for sterol disorders. This study will also help researchers learn whether adding
extra cholesterol to the diet will improve behavioral and other autism spectrum
characteristics seen in individuals with ASD and low cholesterol.
Objectives:
- To determine cholesterol levels in children with autism spectrum disorders.
- To compare behavioral and other characteristics among children who have autism spectrum
disorders and high, low, or normal cholesterol levels.
- To determine whether adding cholesterol to the diet will improve behavioral and other
characteristics in individuals with ASD and low cholesterol.
Eligibility:
- Children between the ages of 4 and 12 who have been diagnosed with an autism spectrum
disorder.
Design:
- Initial screening study will involve a collection of blood samples (for study purposes
and cholesterol testing).
- Children who have low cholesterol levels will take part in a study in which they will
receive either cholesterol supplementation or a placebo, and will have detailed
physical and psychological examinations to measure possible improvement in behavioral
or other characteristics.
- Children who have high or normal cholesterol levels will have further blood samples
taken, and will undergo an additional set of examinations for comparison purposes.
- Researchers may request blood or DNA samples from other family members (parents or
siblings), which will be collected through blood draws and cheek swabs....
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| Study summary: |
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Pilot work suggests that some individuals with autism spectrum disorders (ASD) have very low
blood cholesterol levels. This low cholesterol level and other abnormal sterol levels may be
important markers for subtypes of ASD. The proposed trial aims to test the response of
individuals with ASD and low cholesterol to increased cholesterol in the diet.
Evidence for the role of low cholesterol in causing ASD in a subgroup of individuals comes
from five sources. First, half of individuals with Smith-Lemli-Opitz syndrome (SLOS) meet
the behavioral criteria for autistic disorder (Tierney et al, 2001), and three quarters have
some type of ASD (Sikora et al, 2006). Second, in individuals with SLOS, the lower the
cholesterol was in the blood and cerebrospinal fluid, the more severe were the autism and IQ
and adaptive function deficits. Third, in SLOS, improvement was found in social and
communication abilities with added dietary cholesterol. Fourth, cholesterol was low in a
pilot study of 100 children with autism of unknown cause. Fifth, it is becoming increasingly
clear that cholesterol plays a pivotal role in several aspects of brain development.
This proposal is designed to 1) determine the prevalence of hypocholesterolemia (greater
than 2 standard deviations below the mean) in ASD individuals (ASD+Hypo); 2) determine the
prevalence of hypercholesterolemia (greater than 2 standard deviations above the mean) in
ASD individuals (ASD+Hyper); 3) determine the rate of SLOS in the ASD subjects; 4) determine
the phenotype (physical, behavioral, and developmental) of ASD individuals with either
hypocholesterolemia, hypercholesterolemia or normal cholesterol (ASD+Normal); 5) test the
efficacy of dietary cholesterol supplementation in ASD individuals with hypocholesterolemia;
and 6) create a repository of biomaterial samples from individuals with ASD and their
biological family members.
Three sites (Kennedy Krieger Institute (KKI), Ohio State University (OSU), and the National
Institutes of Health (NIH) will collaborate to accomplish the objectives of this study. In
addition to defining the frequency of altered cholesterol homeostasis in ASD, 60 youths (20
at each site) with ASD plus hypocholesterolemia will enter a 12-week, double-blind,
placebo-controlled trial immediately followed by a 12-week open-label cholesterol trial to
test the efficacy of dietary cholesterol supplementation. Outcome measures will include
standard tests of behavior, communication, and other autism features.
These findings will guide the medical community in identifying individuals who should be
tested for sterol disorders. This study will also help researchers learn whether adding
extra cholesterol to the diet will improve behavioral and other autism spectrum
characteristics seen in individuals with ASD and low cholesterol. The results of this study
may help individuals with hypocholesterolemic ASD by the knowledge of the therapeutic value
and safety of the use of cholesterol supplementation both biochemically and behaviorally. If
improvement is demonstrated, it opens a new window to understanding the neurologic
mechanisms of ASD. This knowledge may also be helpful for hypocholesterolemic individuals
with ASD in that this newly identified population will benefit from such supplementation.
Even if cholesterol supplementation is found to not be effective, important behavioral
phenotype and developmental information will be obtained that might be useful in identifying
subjects with ASD plus cholesterol abnormalities. |
| Criteria: |
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- INCLUSION/EXCLUSION CRITERIA:
Screening Visit:
Inclusion:
- Parents agree not to use dietary supplements if their children are selected to
continue to the Characterization Visit and not to use any dietary supplements
throughout the clinical trial other than the supplements supplied by the research
study.
- Male or female between the ages of 4.0-11.75 years at the time of consent/assent.
- Clinically diagnosed with an ASD for which no cause has been detected.
- Anticonvulsants used for the treatment of a seizure disorder will be permitted if the
dosage has been stable for 3 months, and the subject is seizure free for at least 3
months.
Exclusion:
- Known pregnancy.
- Subject has SLOS or known cholesterol synthesis/regulation disorder.
- The subject has had an anticonvulsant dose change in the preceding 3 months or a
seizure in the preceding 3 months.
- Child has an abnormal karyotype (such as rearrangement or fragile X syndrome).
- DSM-IV diagnosis of Rett Disorder, childhood disintegrative disorder, schizophrenia,
another psychotic disorder, or substance abuse.
- A significant medical condition such as heart disease, hypertension, liver or renal
failure, pulmonary disease, diabetes, or unstable seizure disorder identified by
history, physical examination, or laboratory tests.
- Currently on or has taken a statin medication within the preceding 3 months.
- Currently on or has taken dietary cholesterol supplementation within the preceding 3
months.
- Subjects will be excluded if they are on other medications that affect cholesterol or
other lipid levels.
(No subjects will be excluded based on race, ethnicity or gender).
Characterization Visit:
Inclusion:
- Same inclusion criteria as the Screening Visit.
- Participants must have a mental age of 18 months as measured at the Characterization
Visit by the age-appropriate form of the Stanford Binet-V or the Mullen Scales of
Early Learning or DAS (N.8. potential subjects who test below 18 months of age, but
are otherwise eligible, may be admitted to the study following a case review convened
by the Multisite Steering Committee).
- Child must have an ASD diagnosis using DSM-IV and clinical judgment in order to
proceed to the other components of the Characterization Visit.
- Neuroleptic medication free for are least three months (ASD+Hyper and ASD+Normal
only).
Exclusion:
- Same exclusion criteria as the Screening Visit.
- Non-verbal mental age below18 months as measured by the developmentally-appropriate
form of the Standard -Binet-V or the Mullen Scales of Early Learning or DAS (N.B.
potential subjects who test below 18 months of age, but are otherwise eligible, may
be admitted to the study following a case review convened by the Multisite Steering
Committee).
- Did not meet the criteria for an ASD & during the CharacterizationVisit.
Double-Blind, Placebo-Controlled trial phase for ASD+Hypo Only:
Inclusion:
- Met Characterization Visit inclusion criteria and completed Characterization Visit
procedures.
- Met criteria for hypocholesterolemia in ASD.
Exclusion:
-Same exclusion criteria as the Characterization Visit.
Open.Label Trial for ASD+Hypo only:
Inclusion:
-Subject continues to meet double-blind, placebo-controlled trial phase inclusion
criteria.
Exclusion:
-Subject started a neuroleptic medication or medication that affects cholesterol synthesis
or metabolism. |
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| Study is available at: |
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Ohio State University
Columbus, OH 43210
United States
Primary Contact:
Patient Recruitment and Public Liaison Office
Email: prpl@mail.cc.nih.gov
Phone: (800) 411-1222 |
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If you are interested in this clinical trial please use the contact information above. If you would like to get additional information about this clinical trial please visit ClinicalTrials.gov.
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| Data Source: |
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ClinicalTrials.gov |
| Date Processed: |
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March 16, 2010 |
Modifications to
this listing: |
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Only selected fields are shown, please use the link
above to view all information about this clinical trial. |
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Clinical trials are medical research studies designed to test the safety and/or
effectiveness of new drugs, devices, or treatments in humans. These studies are
conducted worldwide for a range of conditions and illnesses. Learn more about
clinical research and participating in a study at
About Clinical Trials.
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