| Dec 15, 2009 |
| Protocol Number: 09-CH-0203
Active Accrual, Protocols Recruiting New Patients Title: Cholesterol in Autism Spectrum Disorder (ASD): Characterization and Treatment Number: 09-CH-0203 Summary: 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. Sponsoring Institute: National Institute of Child Health and Human Development (NICHD) Recruitment Detail Type: Participants currently recruited/enrolled Gender: Male & Female Referral Letter Required: No Population Exclusion(s): None Eligibility Criteria: 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. Special Instructions: Currently Not Provided Keywords: Autism Autism Spectrum Disorder Cholesterol Smith-Lemli-Opitz Syndrome Recruitment Keyword(s): Autism Spectrum Disorder Autism ASD Cholesterol Pervasive Developmental Disorder PPD-NOS Condition(s): Autism Asperger Disorder PDD-NOS Investigational Drug(s): None Investigational Device(s): None Intervention(s): Other: Cholesterol Supporting Site: National Institute of Child Health and Human Development Contact(s): Patient Recruitment and Public Liaison Office Building 61 10 Cloister Court Bethesda, Maryland 20892-4754 Toll Free: 1-800-411-1222 TTY: 301-594-9774 (local),1-866-411-1010 (toll free) Fax: 301-480-9793 Electronic Mail:prpl@mail.cc.nih.gov Citation(s): Elias ER, Irons MB, Hurley AD, Tint GS, Salen G. Clinical effects of cholesterol supplementation in six patients with the Smith-Lemli-Opitz syndrome (SLOS). Am J Med Genet. 1997 Jan 31;68(3):305-10. Irons M, Elias ER, Abuelo D, Bull MJ, Greene CL, Johnson VP, Keppen L, Schanen C, Tint GS, Salen G. Treatment of Smith-Lemli-Opitz syndrome: results of a multicenter trial. Am J Med Genet. 1997 Jan 31;68(3):311-4. Kaufmann WE, Cortell R, Kau AS, Bukelis I, Tierney E, Gray RM, Cox C, Capone GT, Stanard P. Autism spectrum disorder in fragile X syndrome: communication, social interaction, and specific behaviors. Am J Med Genet A. 2004 Sep 1;129A(3):225-34. |
Attachments: 09-CH-0203 ASD Flyer 3 R IRB App 11-19-09_Layout 1.pdf
09-CH-0203 ASD Flyer Tear Off R IRB App 11-19-09_Layout 1.pdf
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