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

March 16, 2026 updated by: Curemark

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

405

Phase

  • Phase 3

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
      • Phoenix, Arizona, United States, 85006
        • Southwest Autism Research & Resource Center (S.A.R.R.C.)
      • Tucson, Arizona, United States, 85724
        • University of Arizona, Pediatrics Multidisciplinary Research Unit
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)
    • California
      • Orange, California, United States, 92868
        • N.R.C. Research Institute
      • Sacramento, California, United States, 95817
        • M.I.N.D. Institute (Univ.of California, Davis)
      • San Francisco, California, United States, 94143-0984
        • University of California (U.C.S.F.)
    • Colorado
      • Centennial, Colorado, United States, 80112
        • IMMUNOe Research Centers
    • Connecticut
      • New Haven, Connecticut, United States, 06519
        • Yale Child Study Center
    • Florida
      • North Miami, Florida, United States, 33161
        • Segal Institute for Clinical Research
      • Orange City, Florida, United States, 32763
        • Advent Health -Lake Mary Pediatrics
      • Orlando, Florida, United States, 32803
        • A.P.G. Research
    • Indiana
      • Newburgh, Indiana, United States, 47630
        • Research Institute of Deaconess Clinic
    • Louisiana
      • Lake Charles, Louisiana, United States, 70629
        • Lake Charles Clinical Trials
      • Shreveport, Louisiana, United States, 71103
        • L.S.U. Health Sciences Center
    • Michigan
      • Bingham Farms, Michigan, United States, 48025
        • Detroit Clinical Research Center, P.C.
    • New Jersey
      • Egg Harbor, New Jersey, United States, 08234
        • Children's Specialized Hospital
      • Toms River, New Jersey, United States, 08755
        • Children's Specialized Hospital
      • Voorhees Township, New Jersey, United States, 08043
        • Clinical Research Center of Nj
    • New Mexico
      • Albuquerque, New Mexico, United States, 87108
        • Lovelace Scientific Resources
    • New York
      • Staten Island, New York, United States, 10312
        • Richmond Behavioral Associates
      • The Bronx, New York, United States, 10467
        • Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke Center For Autism and Brain Development
    • Ohio
      • Cleveland, Ohio, United States, 44104
        • Cleveland Clinic Autism Center
    • Rhode Island
      • Warwick, Rhode Island, United States, 02886
        • Omega Medical Research
    • South Carolina
      • Charleston, South Carolina, United States, 29407
        • Carolina Clinical Trials, Inc.
    • Tennessee
      • Nashville, Tennessee, United States, 37232-2551
        • Vanderbilt Universtiy Med.Center-Treatment&Research Inst. For Asd
    • Texas
      • Houston, Texas, United States, 77054
        • University of Texas, Houston-Behavioral & Biomedical Sciences
    • Utah
      • Clinton, Utah, United States, 84015
        • Focus Center of Clinical Research
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences
      • Herndon, Virginia, United States, 20170
        • Neuroscience, Inc
      • Roanoke, Virginia, United States, 24014
        • Carilion Clinic-Virginia Tech, Carilion School of Medicine

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

3 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • 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.

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

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Open Label
CM-AT
Drug: CM-AT
Single unit does powder of active substance (CM-AT) administered 3 times per day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
Change from Baseline to each post-baseline visit, through study completion Week 72.

Secondary Outcome Measures

Outcome Measure
Time Frame
Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) 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.
Change from Baseline to each post-baseline visit, through study completion Week 72.

Collaborators and Investigators

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

Sponsor

Curemark

Investigators

  • Principal Investigator: Deborah Pearson, PhD, The University of Texas Health Science Center, Houston
  • Principal Investigator: Robert Hendren, DO, University of California, San Francisco

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 (Actual)

October 1, 2015

Primary Completion (Estimated)

January 1, 2028

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

November 12, 2015

First Submitted That Met QC Criteria

January 6, 2016

First Posted (Estimated)

January 8, 2016

Study Record Updates

Last Update Posted (Actual)

March 18, 2026

Last Update Submitted That Met QC Criteria

March 16, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 00104/Open Label-Autism

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

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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