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Children's Autism Metabolome Project (CAMP-01)

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ClinicalTrials.gov Identifier: NCT02548442
Recruitment Status : Active, not recruiting
First Posted : September 14, 2015
Last Update Posted : June 12, 2018
Sponsor:
Collaborators:
National Institute of Mental Health (NIMH)
Nancy Lurie Marks Family Foundation
Information provided by (Responsible Party):
Stemina Biomarker Discovery, Inc.

Tracking Information
First Submitted Date September 2, 2015
First Posted Date September 14, 2015
Last Update Posted Date June 12, 2018
Study Start Date August 2015
Estimated Primary Completion Date August 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 10, 2015)
Metabolites in plasma indicative of autism spectrum disorder. [ Time Frame: within 24 months of sample collection ]
The study will be divided into a biomarker discovery/method development phase followed by a validation phase of the analytical methods and algorithm that will be used in the clinical test. The first 375 subjects enrolled in each group (ASD, DD, TD) will be used in the discovery/training set and the remaining 125 subjects will be held aside for use as a validation set. The training set will be used for discovery of the biomarkers and development of the analytical methods intended for the diagnostic test. The validation sample set will be used to evaluate performance of the final clinical methods and algorithms.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02548442 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: September 10, 2015)
Metabolites in plasma that are specific for subtypes of autism spectrum disorder [ Time Frame: Within 24 months of sample collection ]
The study will be divided into a biomarker discovery/method development phase followed by a validation phase of the analytical methods and algorithm that will be used in the clinical test. The first 375 subjects enrolled in each group (ASD, DD, TD) will be used in the identification of subtypes and the remaining 125 subjects will be held aside for use as a validation set. The training set will be used for discovery of the biomarkers and development of the analytical methods intended for the diagnostic test. The validation sample set will be used to evaluate performance of the final clinical methods and algorithms.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Children's Autism Metabolome Project
Official Title Children's Autism Metabolome Project (CAMP): Development and Clinical Evaluation of the Stemina Metabolic Biomarker-Based Test to Diagnose Autism Spectrum Disorder in Early Childhood
Brief Summary Development and Clinical Evaluation of the Stemina Metabolic Biomarker-Based Test to Diagnose Autism Spectrum Disorder in Early Childhood.
Detailed Description

The purpose of this study is to identify a metabolite signature in blood plasma and/or urine using a panel of biomarker metabolites that differentiate children with autism spectrum disorder (ASD) from children with delayed development (DD) and/or typical development (TD), to develop an algorithm that maximizes sensitivity and specificity of the biomarker profile, and to evaluate the algorithm as a diagnostic tool.

A secondary objective is to define metabolites capable of classifying subtypes of ASD that may increase understanding of the metabolic basis of the condition, as well as inform on personalized therapy.

The population targeted for this study includes children aged 18 months to 48 months, diagnosed with ASD or DD using behavioral criteria, and TD children, identified as having no indications of ASD or DD using behavioral criteria. The target size for each of the three groups is 500 children, for a total of 1500 subjects. The targeted male:female ratio is 4:1 in all three groups. If the diagnostic biomarkers identified in the study do not perform well in females during the biomarker discovery phase, the study may be expanded to recruit more females to examine the possibility of a female-specific diagnostic test.

Subjects will be qualified for entry into the study and will be invited to participate. On the first study day, subjects' parents will sign an informed consent form and will be asked questions on the mother's pregnancy and of both parents' medical history. A complete medical history, a physical examination, and information needed to obtain a diagnosis of ASD, DD, or TD will be obtained on the study subject. If possible, a urine sample will be collected during the visit. Up to four tubes of blood (<25 mLs total) will be drawn at the clinic during the visit or within 14 days following this initial visit. An overnight fast is required prior to the visit where blood will be taken from the subject. A subset of the subjects will be asked to return to the clinic 30-60 days later to obtain a replicate metabolic profile.

The study will be divided into a biomarker discovery/method development phase followed by a validation phase of the analytical methods and algorithm that will be used in the clinical test.

The first 375 subjects enrolled in each group (ASD, DD, TD) will be used in the discovery/training set and the remaining 125 subjects will be held aside for use as a validation set. The training set will be used for discovery of the biomarkers and development of the analytical methods intended for the diagnostic test. The validation sample set will be used to evaluate performance of the final clinical methods and algorithms.

Consent will also be sought from all subjects for follow-up contact up to 2 years following enrollment to determine the accuracy of the original behavioral diagnosis. Subjects chosen for follow-up will be identified based on the strength of the diagnosis from the behavioral scores and physician assessments as well as the biomarker profiles observed in individuals.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood plasma for metabolite analysis and blood samples for DNA and RNA expression analysis will be collected from all children as part of the study protocol. In addition, a urine sample for metabolite analysis will also be obtained from children capable of providing the sample while at the clinical site.
Sampling Method Non-Probability Sample
Study Population Subjects will be identified as autism spectrum disorder (ASD) or developmentally delayed (DD) subjects by standard behavioral techniques; Typically developing (TD) subjects will be recruited from the communities in which the autism research centers are situated. The ADOS-2 (research reliable), the Mullen Scales of Early Learning (MSEL), as well as an evaluation based on the DSM-V checklist will be administered to each subject enrolled in the study suspected to have a developmental delay or autism. Typically developing children enrolled in the study will be given an MSEL evaluation to document that they are neither ASD nor DD subjects.
Condition
  • Autism Spectrum Disorder
  • Developmental Disorder
Intervention Not Provided
Study Groups/Cohorts
  • Autism Spectrum Disorder
    Subjects identified as having Autism Spectrum Disorder using behavioral based methods.
  • Developmental Delay
    Subjects identified as having a developmental delay that is not Autism Spectrum Disorder using behavioral methods.
  • Typically Developing Children
    Subjects identified as not having a developmental delay or autism spectrum disorder using behavioral methods as well as not having another serious medical or psychological condition.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: June 11, 2018)
1102
Original Estimated Enrollment
 (submitted: September 10, 2015)
1500
Estimated Study Completion Date August 2020
Estimated Primary Completion Date August 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Age of greater or equal to 18 months and less than or equal to 48 months
  • Fulfills the definition of an autism spectrum disorder, developmentally delayed, or typically developing child in the age range 18-48 months, as determined by a clinician or certified practitioner of the appropriate tests and who is knowledgeable in the field; and
  • Has parental (or other legal guardian ) informed consent to participate.

Exclusion Criteria:

  • Diagnosis with a chronic condition that could interfere with a diagnosis of ASD or DD, (e.g.: a known history of Fragile X, Rett syndrome, Down syndrome, tuberous sclerosis, trisomy 21, inborn errors of metabolism or other genetic disorder that includes some symptoms of autism)
  • Fetal alcohol syndrome, or other serious neurological disorder
  • Other serious metabolic disorder, psychiatric disorder, or medical condition involving the liver, kidney, pulmonary, cardiovascular or endocrine systems
  • A second child within a family in which a sibling has already been enrolled.
  • A child who has previously participated in the CAMP-01 study
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Months to 48 Months   (Child)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02548442
Other Study ID Numbers CAMP-01
R44MH107124-01 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Stemina Biomarker Discovery, Inc.
Study Sponsor Stemina Biomarker Discovery, Inc.
Collaborators
  • National Institute of Mental Health (NIMH)
  • Nancy Lurie Marks Family Foundation
Investigators
Principal Investigator: Robert E Burrier, Ph.D. Stemina Biomarker Discovery
PRS Account Stemina Biomarker Discovery, Inc.
Verification Date June 2018