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

NCT02649959 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: 00104/Open Label-Autism
• Study Type: interventional
• Target: 405
• Locations: 1 country
• Sites: 31

Brief 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.

Conditions

Autism

Keywords

Autism; A long-term extension study in a pediatric population of children with autism.

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (1)

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

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

Study Arms (1)

Open Label

EXPERIMENTAL

CM-AT

Drug: CM-AT

Interventions

CM-AT DRUG

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

Open Label

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Curemark: lead

Study Sites (31)

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

Phoenix, Arizona, 85006, United States

Location

University of Arizona, Pediatrics Multidisciplinary Research Unit

Tucson, Arizona, 85724, United States

Location

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

Little Rock, Arkansas, 72202, United States

Location

N.R.C. Research Institute

Orange, California, 92868, United States

Location

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

Sacramento, California, 95817, United States

Location

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

San Francisco, California, 94143-0984, United States

Location

IMMUNOe Research Centers

Centennial, Colorado, 80112, United States

Location

Yale Child Study Center

New Haven, Connecticut, 06519, United States

Location

Segal Institute For Clinical Research

North Miami, Florida, 33161, United States

Location

Advent Health -Lake Mary Pediatrics

Orange City, Florida, 32763, United States

Location

A.P.G. Research

Orlando, Florida, 32803, United States

Location

Research Institute of Deaconess Clinic

Newburgh, Indiana, 47630, United States

Location

Lake Charles Clinical Trials

Lake Charles, Louisiana, 70629, United States

Location

L.S.U. Health Sciences Center

Shreveport, Louisiana, 71103, United States

Location

Detroit Clinical Research Center, P.C.

Bingham Farms, Michigan, 48025, United States

Location

Children'S Specialized Hospital

Egg Harbor, New Jersey, 08234, United States

Location

Children's Specialized Hospital

Toms River, New Jersey, 08755, United States

Location

Clinical Research Center of Nj

Voorhees Township, New Jersey, 08043, United States

Location

Lovelace Scientific Resources

Albuquerque, New Mexico, 87108, United States

Location

Richmond Behavioral Associates

Staten Island, New York, 10312, United States

Location

Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program

The Bronx, New York, 10467, United States

Location

Duke Center For Autism and Brain Development

Durham, North Carolina, 27705, United States

Location

Cleveland Clinic Autism Center

Cleveland, Ohio, 44104, United States

Location

Omega Medical Research

Warwick, Rhode Island, 02886, United States

Location

Carolina Clinical Trials, Inc.

Charleston, South Carolina, 29407, United States

Location

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

Nashville, Tennessee, 37232-2551, United States

Location

University of Texas, Houston-Behavioral & Biomedical Sciences

Houston, Texas, 77054, United States

Location

Focus Center of Clinical Research

Clinton, Utah, 84015, United States

Location

University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences

Charlottesville, Virginia, 22903, United States

Location

Neuroscience, Inc

Herndon, Virginia, 20170, United States

Location

Carilion Clinic-Virginia Tech, Carilion School of Medicine

Roanoke, Virginia, 24014, United States

Location

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MeSH Terms

Conditions

Autistic Disorder

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Deborah Pearson, PhD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

• Robert Hendren, DO

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2015
• First Posted: January 8, 2016
• Study Start: October 1, 2015
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): January 1, 2028
• Last Updated: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (31)