Autism Spectrum DisorderCAMP-01NCT02548442
Children's Autism Metabolome Project
An observational study of Autism Spectrum Disorder and Developmental Disorder, run by Stemina Biomarker Discovery, Inc. at 8 sites in United States.
Trial summary
Development and Clinical Evaluation of the Stemina Metabolic Biomarker-Based Test to Diagnose Autism Spectrum Disorder in Early Childhood. Read more about this study
- Who is it for?
- Ages 18 to 48 monthsHealthy volunteers can take part
- Conditions the study is for
- Autism Spectrum Disorder, Developmental Disorder
- How many people
- 1,102 participants
- Where
- 8 sitesUnited States
- Study dates
- Aug 2015to Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 48 months
- All sexes
- Healthy volunteers accepted
- Child
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.
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
Lurie Center for Autism
Lexington, Massachusetts, 02142
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UC David MIND Institute
Sacramento, California, 95817
Melmed Center
Scottsdale, Arizona, 85254
About this study
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
Development and Clinical Evaluation of the Stemina Metabolic Biomarker-Based Test to Diagnose Autism Spectrum Disorder in Early Childhood.
Read the 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.
- Conditions
- Autism Spectrum Disorder, Developmental Disorder
- Medical subject headings
- Autism Spectrum Disorder, Developmental Disabilities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Metabolites in plasma indicative of autism spectrum disorder.
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.
Measured: Within 60 months of sample collection
Secondary outcomes (1)
Metabolites in plasma that are specific for subtypes of autism spectrum disorder
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.
Measured: Within 60 months of sample collection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2015 (estimated)
- Study startAug 2015
- Primary completionJan 2023 (estimated)
- Study completionAug 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,102 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stemina Biomarker Discovery, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Institute of Mental Health (NIMH), Nancy Lurie Marks Family Foundation
- Responsible party
- Sponsor
- Investigators
- Robert E Burrier, Ph.D., Principal investigator, Stemina Biomarker Discovery
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02548442
- Sponsor's protocol ID
- CAMP-01
- Other IDs
- R44MH107124-01
- Acronym
- CAMP-01
- Status last verified
- Jun 2020
- First submitted
- 2 Sep 2015
- Last updated
- 29 Jun 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
West PR, Amaral DG, Bais P, Smith AM, Egnash LA, Ross ME, Palmer JA, Fontaine BR, Conard KR, Corbett BA, Cezar GG, Donley EL, Burrier RE. Metabolomics as a tool for discovery of biomarkers of autism spectrum disorder in the blood plasma of children. PLoS One. 2014 Nov 7;9(11):e112445. doi: 10.1371/journal.pone.0112445. eCollection 2014.
PubMed 25380056Smith AM, King JJ, West PR, Ludwig MA, Donley ELR, Burrier RE, Amaral DG. Amino Acid Dysregulation Metabotypes: Potential Biomarkers for Diagnosis and Individualized Treatment for Subtypes of Autism Spectrum Disorder. Biol Psychiatry. 2019 Feb 15;85(4):345-354. doi: 10.1016/j.biopsych.2018.08.016. Epub 2018 Sep 6.
PubMed 30446206Smith AM, Donley ELR, Ney DM, Amaral DG, Burrier RE, Natowicz MR. Metabolomic biomarkers in autism: identification of complex dysregulations of cellular bioenergetics. Front Psychiatry. 2023 Oct 2;14:1249578. doi: 10.3389/fpsyt.2023.1249578. eCollection 2023.
PubMed 37928922
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02548442
- Acronym / sponsor ID
- CAMP-01 / CAMP-01
- Conditions
- Autism Spectrum Disorder, Developmental Disorder
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,102 participants (actual)
- Lead sponsor
- Stemina Biomarker Discovery, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Institute of Mental Health (NIMH), Nancy Lurie Marks Family Foundation
- Study start
- Aug 2015
- Primary completion
- Jan 2023 (estimated)
- Study completion
- Aug 2023 (estimated)
- First posted
- 14 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 to 48 months
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
Metabolites in plasma that are specific for subtypes of autism spectrum disorder
Time frame: Within 60 months of sample collection
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2020.
- Background: PubMed 25380056West PR, Amaral DG, Bais P, Smith AM, Egnash LA, Ross ME, Palmer JA, Fontaine BR, Conard KR, Corbett BA, Cezar GG, Donley EL, Burrier RE. Metabolomics as a tool for discovery of biomarkers of autism spectrum disorder in the blood plasma of children. PLoS One. 2014 Nov 7;9(11):e112445. doi: 10.1371/journal.pone.0112445. eCollection 2014.
- Results publication: PubMed 30446206Smith AM, King JJ, West PR, Ludwig MA, Donley ELR, Burrier RE, Amaral DG. Amino Acid Dysregulation Metabotypes: Potential Biomarkers for Diagnosis and Individualized Treatment for Subtypes of Autism Spectrum Disorder. Biol Psychiatry. 2019 Feb 15;85(4):345-354. doi: 10.1016/j.biopsych.2018.08.016. Epub 2018 Sep 6.
- Derived from this study: PubMed 37928922Smith AM, Donley ELR, Ney DM, Amaral DG, Burrier RE, Natowicz MR. Metabolomic biomarkers in autism: identification of complex dysregulations of cellular bioenergetics. Front Psychiatry. 2023 Oct 2;14:1249578. doi: 10.3389/fpsyt.2023.1249578. eCollection 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert E Burrier, Ph.D. principal investigator
Stemina Biomarker Discovery
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Autism Spectrum Disorder and Developmental Disorder, run by Stemina Biomarker Discovery, Inc. at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Metabolites in plasma indicative of autism spectrum disorder. (time frame: Within 60 months of sample collection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02548442, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2020.