Children's Autism Metabolome Project (CAMP-01)

June 25, 2020 updated by: Stemina Biomarker Discovery, Inc.

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

Development and Clinical Evaluation of the Stemina Metabolic Biomarker-Based Test to Diagnose Autism Spectrum Disorder in Early Childhood.

Study Overview

Status

Active, not recruiting

Conditions

Detailed Description

The purpose of this study is to identify metabolitic signatures 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 overall 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 original target size for the study was 1500 subjects divided equally between the three groups. The targeted male:female ratio is 4:1 in all three groups. During the study, it was determined that biomarkers capable of identifying ASD subjects could be obtained using a total of 1100 subjects divided with 58% ASD, 25%TD, and 17% DD. 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 subjects will be randomized and divided equally between a discovery/training set and 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 5 years following enrollment of the last subject enrolled to determine the accuracy of the original behavioral diagnosis over time. 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

Enrollment (Actual)

1102

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85254
        • Melmed Center
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Sacramento, California, United States, 95817
        • UC David MIND Institute
    • Massachusetts
      • Lexington, Massachusetts, United States, 02142
        • Lurie Center for Autism
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 4 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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 and a Social Communications Questionnaire (SCQ) evaluation to document that they are neither ASD nor DD subjects.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Metabolites in plasma indicative of autism spectrum disorder.
Time Frame: Within 60 months of sample collection
Study participants were randomized into two independent groups: a biomarker discovery/method development (training set) and a validation set. The validation set is used to assess the performance of the biomarkers identified in the training set once the analytical methods and algorithms are developed using the training set. These biomarkers and algorithms will be used in the clinical diagnostic tests. Using this approach and CLIA validated assays, two diagnostic panels are now able to correctly identify metabolic signatures in 53% of the ASD population in the CAMP study with a specificity of 91% when tested against the validation set of subject samples.
Within 60 months of sample collection

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Metabolites in plasma that are specific for subtypes of autism spectrum disorder
Time Frame: Within 60 months of sample collection
Using the training set or the validation subject set described in the primary aim, the study will seek to identify additional biomarkers such that >75% of the ASD population can be correctly identified with the same high specificity of >90%. In addition, the study will attempt to link the new biomarkers to important metabolic pathways associated with ASD to give insight into the underlying pathophysiologies of the disorder.
Within 60 months of sample collection

Collaborators and Investigators

This is where you will find people and organizations involved with this 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2015

Primary Completion (Anticipated)

January 1, 2023

Study Completion (Anticipated)

August 1, 2023

Study Registration Dates

First Submitted

September 2, 2015

First Submitted That Met QC Criteria

September 10, 2015

First Posted (Estimate)

September 14, 2015

Study Record Updates

Last Update Posted (Actual)

June 29, 2020

Last Update Submitted That Met QC Criteria

June 25, 2020

Last Verified

June 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAMP-01
  • R44MH107124-01 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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