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AutismPhase 3NCT02649959

An Open Label Study of CM-AT for the Treatment of Children With Autism

A Phase 3 study testing CM-AT in people with Autism, run by Curemark at 31 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a Phase III, open label extension study evaluating the continued safety and efficacy of CM-AT in pediatric patients with autism with all levels of fecal chymotrypsin. Read more about this study

Who is it for?
Ages 3 to 17 years
Conditions the study is for
Autism
What is being tested?
CM-AT
Could I receive a placebo?
No, everyone receives an active treatment
How many people
405 participants
Where
31 sitesUnited States
Study dates
Oct 2015to Jan 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

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 3 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Age between 3 and 8 years, inclusive, at the time of signing informed consent/assent in Sponsor 00103 Study
  • Completion of the Sponsor's 00103 Study who continue to meet eligibility requirements
  • Currently in the 00102 open label study and continue to meet eligibility requirements
  • Subjects who initially qualified for 00103 screening, who subsequently failed Baseline entrance criteria for randomization (@Visit 1) Baseline assessment of the ABC eligibility requirement who continue to meet eligibility requirements
  • Up to 20 subjects 9-17 years of age who directly enroll into this study, who meet the current Diagnostic and Statistical Manual for Mental Disorders (DSM-IV-TR) diagnostic criteria for Autistic Disorder (AD), screened by the SCQ and confirmed by the ADI-R

Who cannot join

  • Patient weighing < 13kg
  • Allergy to porcine products
  • Previous sensitization or allergy to trypsin, pancreatin, or pancrelipase
  • History of severe head trauma, as defined by loss of consciousness or hospitalization, skull fracture or stroke.
  • Seizure within the last year prior to enrollment, or the need for seizure medications either at present or in the past.
  • Evidence or history of severe, moderate or uncontrolled systemic disease
Show all 13Show fewer
  • Ongoing dietary restriction for allergy or other reasons except nut allergies. Lactose free is allowable but not dairy free.
  • Inability to ingest the study drug / non-compliance with dosing schedule.
  • Inability to follow the prescribed dosing schedule.
  • Use of any stimulant or non-stimulant medication or medications given for attention deficit hyperactivity disorder (ADHD) must be discontinued 5 days prior to the initial randomized study period.
  • Subjects taking an selective serotonin reuptake inhibitor (SSRI) must be on a stable dose for a minimum of 30 days prior to entering the study.
  • History of premature birth <35 weeks gestation.
  • Prior history of stroke in utero or other in utero insult.

Trial locations and contacts

31 sites in 1 country.

31 sites in 1 country

United States31 sites

  • Lovelace Scientific Resources

    Albuquerque, New Mexico, 87108

  • Detroit Clinical Research Center, P.C.

    Bingham Farms, Michigan, 48025

  • IMMUNOe Research Centers

    Centennial, Colorado, 80112

  • Carolina Clinical Trials, Inc.

    Charleston, South Carolina, 29407

  • University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences

    Charlottesville, Virginia, 22903

  • Cleveland Clinic Autism Center

    Cleveland, Ohio, 44104

  • Focus Center of Clinical Research

    Clinton, Utah, 84015

  • Duke Center For Autism and Brain Development

    Durham, North Carolina, 27705

  • Children'S Specialized Hospital

    Egg Harbor, New Jersey, 08234

  • Neuroscience, Inc

    Herndon, Virginia, 20170

  • University of Texas, Houston-Behavioral & Biomedical Sciences

    Houston, Texas, 77054

  • Lake Charles Clinical Trials

    Lake Charles, Louisiana, 70629

  • Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)

    Little Rock, Arkansas, 72202

  • Vanderbilt Universtiy Med.Center-Treatment&Research Inst. For Asd

    Nashville, Tennessee, 37232-2551

  • Yale Child Study Center

    New Haven, Connecticut, 06519

  • Research Institute of Deaconess Clinic

    Newburgh, Indiana, 47630

  • Segal Institute For Clinical Research

    North Miami, Florida, 33161

  • N.R.C. Research Institute

    Orange, California, 92868

  • Advent Health -Lake Mary Pediatrics

    Orange City, Florida, 32763

  • A.P.G. Research

    Orlando, Florida, 32803

  • Southwest Autism Research & Resource Center (S.A.R.R.C.)

    Phoenix, Arizona, 85006

  • Carilion Clinic-Virginia Tech, Carilion School of Medicine

    Roanoke, Virginia, 24014

  • M.I.N.D. Institute (Univ.of California, Davis)

    Sacramento, California, 95817

  • University of California (U.C.S.F.)

    San Francisco, California, 94143-0984

  • L.S.U. Health Sciences Center

    Shreveport, Louisiana, 71103

  • Richmond Behavioral Associates

    Staten Island, New York, 10312

  • Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program

    The Bronx, New York, 10467

  • Children's Specialized Hospital

    Toms River, New Jersey, 08755

  • University of Arizona, Pediatrics Multidisciplinary Research Unit

    Tucson, Arizona, 85724

  • Clinical Research Center of Nj

    Voorhees Township, New Jersey, 08043

  • Omega Medical Research

    Warwick, Rhode Island, 02886

Other trials in United States

About this study

Official title: An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin

This is a Phase III, open label extension study evaluating the continued safety and efficacy of CM-AT in pediatric patients with autism with all levels of fecal chymotrypsin.

Read the detailed description

Autism is clearly a significant cause of disability in the pediatric population. Treatment is based on the observation that many children with autism do not digest protein. CM-AT is a proprietary enzyme that is designed as a granulated powder taken three times daily.

Conditions
Autism
Being tested
CM-AT
Keywords
Autism, A long-term extension study in a pediatric population of children with autism.
Medical subject headings
Autistic Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CM-ATDrug

    Single unit does powder of active substance (CM-AT) administered 3 times per day

Study groups in NCT02649959
Study groupRoleWhat they receive
Open LabelExperimentalCM-AT
Receives: Drug: CM-AT

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6

    Measured: Change from Baseline to each post-baseline visit, through study completion Week 72.

Secondary outcomes (1)

  1. Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6

    Measured: Change from Baseline to each post-baseline visit, through study completion Week 72.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Jan 2016 (estimated)
  2. Study startOct 2015
  3. Primary completionJan 2028 (estimated)
  4. Study completionJan 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
405 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02649959
Sponsor's protocol ID
00104/Open Label-Autism
Status last verified
Mar 2026
First submitted
12 Nov 2015
Last updated
18 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • McPheeters ML, Warren Z, Sathe N, Bruzek JL, Krishnaswami S, Jerome RN, Veenstra-Vanderweele J. A systematic review of medical treatments for children with autism spectrum disorders. Pediatrics. 2011 May;127(5):e1312-21. doi: 10.1542/peds.2011-0427. Epub 2011 Apr 4.

    PubMed 21464191

  • Autism and Developmental Disabilities Monitoring Network Surveillance Year 2008 Principal Investigators; Centers for Disease Control and Prevention. Prevalence of autism spectrum disorders--Autism and Developmental Disabilities Monitoring Network, 14 sites, United States, 2008. MMWR Surveill Summ. 2012 Mar 30;61(3):1-19.

    PubMed 22456193

  • Baio, J. Prevalence of Autism Spectrum Disorders - Autism and Developmental Disabilities Monitoring Network, 14 Sites, United States.(2010). Retrieved from http://www.cdc.gov/mmwr/pdf/ss/ss6302.pdf

  • Peacock G, Amendah D, Ouyang L, Grosse SD. Autism spectrum disorders and health care expenditures: the effects of co-occurring conditions. J Dev Behav Pediatr. 2012 Jan;33(1):2-8. doi: 10.1097/DBP.0b013e31823969de.

    PubMed 22157409

  • Ganz ML. The lifetime distribution of the incremental societal costs of autism. Arch Pediatr Adolesc Med. 2007 Apr;161(4):343-9. doi: 10.1001/archpedi.161.4.343.

    PubMed 17404130

  • McElhanon BO, McCracken C, Karpen S, Sharp WG. Gastrointestinal symptoms in autism spectrum disorder: a meta-analysis. Pediatrics. 2014 May;133(5):872-83. doi: 10.1542/peds.2013-3995.

    PubMed 24777214

  • Buie T, Campbell DB, Fuchs GJ 3rd, Furuta GT, Levy J, Vandewater J, Whitaker AH, Atkins D, Bauman ML, Beaudet AL, Carr EG, Gershon MD, Hyman SL, Jirapinyo P, Jyonouchi H, Kooros K, Kushak R, Levitt P, Levy SE, Lewis JD, Murray KF, Natowicz MR, Sabra A, Wershil BK, Weston SC, Zeltzer L, Winter H. Evaluation, diagnosis, and treatment of gastrointestinal disorders in individuals with ASDs: a consensus report. Pediatrics. 2010 Jan;125 Suppl 1:S1-18. doi: 10.1542/peds.2009-1878C.

    PubMed 20048083

  • Samsam M, Ahangari R, Naser SA. Pathophysiology of autism spectrum disorders: revisiting gastrointestinal involvement and immune imbalance. World J Gastroenterol. 2014 Aug 7;20(29):9942-51. doi: 10.3748/wjg.v20.i29.9942.

    PubMed 25110424

  • Elsabbagh M, Divan G, Koh YJ, Kim YS, Kauchali S, Marcin C, Montiel-Nava C, Patel V, Paula CS, Wang C, Yasamy MT, Fombonne E. Global prevalence of autism and other pervasive developmental disorders. Autism Res. 2012 Jun;5(3):160-79. doi: 10.1002/aur.239. Epub 2012 Apr 11.

    PubMed 22495912

  • Wasfy M, Oyofo B, Elgindy A, Churilla A. Comparison of preservation media for storage of stool samples. J Clin Microbiol. 1995 Aug;33(8):2176-8. doi: 10.1128/jcm.33.8.2176-2178.1995.

    PubMed 7559972

  • Cavallini G, Benini L, Brocco G, Riela A, Bovo P, Pederzoli P, Angelini G, Pelle C, Bertelli G, Scuro LA. The fecal chymotrypsin photometric assay in the evaluation of exocrine pancreatic capacity. Comparison with other direct and indirect pancreatic function tests. Pancreas. 1989;4(3):300-4. doi: 10.1097/00006676-198906000-00005.

    PubMed 2734275

  • Matthews DM. Intestinal absorption of amino acids and peptides. Proc Nutr Soc. 1972 Sep;31(2):171-7. doi: 10.1079/pns19720033. No abstract available.

    PubMed 4563292

  • Coutinho AM, Oliveira G, Morgadinho T, Fesel C, Macedo TR, Bento C, Marques C, Ataide A, Miguel T, Borges L, Vicente AM. Variants of the serotonin transporter gene (SLC6A4) significantly contribute to hyperserotonemia in autism. Mol Psychiatry. 2004 Mar;9(3):264-71. doi: 10.1038/sj.mp.4001409.

    PubMed 15094787

  • Naushad SM, Jain JM, Prasad CK, Naik U, Akella RR. Autistic children exhibit distinct plasma amino acid profile. Indian J Biochem Biophys. 2013 Oct;50(5):474-8.

    PubMed 24772971

  • Williams K, Wheeler DM, Silove N, Hazell P. Selective serotonin reuptake inhibitors (SSRIs) for autism spectrum disorders (ASD). Cochrane Database Syst Rev. 2010 Aug 4;(8):CD004677. doi: 10.1002/14651858.CD004677.pub2.

    PubMed 20687077

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing CM-AT in people with Autism, run by Curemark at 31 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 5 inclusion and 13 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: under way, not looking for participants, as of the sponsor's last update on 18 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 31 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.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6 (time frame: Change from Baseline to each post-baseline visit, through study completion Week 72.). 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 NCT02649959, which also lists 29 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 18 Mar 2026.

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