A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study)

NCT ID: NCT02410902

Last Updated: 2023-05-24

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

190 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-05-13

Study Completion Date

2017-12-22

Brief Summary

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The purpose of this study is to determine whether CM-AT is safe and effective in treating the core symptoms of autism in children with all levels of fecal chymotrypsin.

Detailed Description

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Autism is clearly a significant cause of disability in the pediatric population. Many children with Autism exhibit impaired protein digestion which may or may not manifest in self-restricted diets. The inability to digest protein affects the availability of essential amino acids in the body. CM-AT is designed to enhance protein digestion thereby potentially restoring the pool of essential amino acids. Essential amino acids play a critical role in the expression of several genes important to neurological function and serve as precursors to key neurotransmitters such as serotonin and dopamine. CM-AT is a proprietary enzyme that is designed as a granulated powder taken three times daily.

Conditions

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Autism

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CM-AT

Active substance in single unit dose powder

Group Type EXPERIMENTAL

CM-AT

Intervention Type DRUG

Single unit dose powder of active substance (CM-AT) administered 3 times per day for 90 days

Placebo

Placebo powder of inactive substance

Group Type PLACEBO_COMPARATOR

PLACEBO

Intervention Type DRUG

Single unit dose powder of non-active substance administered 3 times per day for 90 days

Interventions

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CM-AT

Single unit dose powder of active substance (CM-AT) administered 3 times per day for 90 days

Intervention Type DRUG

PLACEBO

Single unit dose powder of non-active substance administered 3 times per day for 90 days

Intervention Type DRUG

Other Intervention Names

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placebo powder

Eligibility Criteria

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Inclusion Criteria

* Meets the current Diagnostic and Statistical Manual with Mental Disorders (DSM-IV-TR) for Autism (Autistic Disorder), screened by SCQ and confirmed by ADI-R;

Exclusion Criteria

* Patient weighing \< 13kg (28.6 lbs)
* Previous allergy to porcine (pork) products
* Previous history of severe head trauma or stroke, loss of consciousness, seizure (or need for seizure medication either present or past) within one year of entering study or uncontrolled systemic disease
* Diagnosis of: HIV, cerebral palsy, endocrine disorder, pancreatic disease, muscular dystrophy, known genetic disorder, blood dyscrasia, ongoing GI disease
* Evidence of severe, moderate or uncontrolled systemic disease; and/or any co-morbid condition which in the Investigator's or Medical Director's opinion makes it undesirable for the subject to participate in the study or jeopardizes compliance with the protocol;
* Within 30 days of starting the study, certain supplementation, chelation or dietary restriction (a 30 day washout period would be required for inclusion);
* Ongoing dietary restriction for allergy or other reasons except nut allergies (lactose-free allowable);
* Use of of any stimulant medication must be discontinued 5 days prior to entering the study.
* Subject must have a stable dose of SSRI's for at least 30 days.
* Inability to ingest study drug and/or follow prescribed dosing schedule
Minimum Eligible Age

3 Years

Maximum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Curemark

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Deborah Pearson, PhD

Role: PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Robert Hendren, DO

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Locations

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

Phoenix, Arizona, United States

Site Status

University of Arizona, Pediatrics Multidisciplinary Research Unit

Tucson, Arizona, United States

Site Status

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

Little Rock, Arkansas, United States

Site Status

N.R.C. Research Institute

Orange, California, United States

Site Status

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

Sacramento, California, United States

Site Status

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

San Francisco, California, United States

Site Status

IMMUNOe RESEARCH CENTERS

Centennial, Colorado, United States

Site Status

Yale Child Study Center

New Haven, Connecticut, United States

Site Status

Segal Institute For Clinical Research

North Miami, Florida, United States

Site Status

Florida Hospital Medical Group-Lake Mary Pediatrics

Orange City, Florida, United States

Site Status

Kaley Kildahl

Orlando, Florida, United States

Site Status

Research Institute of Deaconess Clinic

Evansville, Indiana, United States

Site Status

Lake Charles Clinical Trials

Lake Charles, Louisiana, United States

Site Status

L.S.U. Health Sciences Center

Shreveport, Louisiana, United States

Site Status

Detroit Clinical Research Center, P.C.

Bingham Farms, Michigan, United States

Site Status

Children'S Specialized Hospital

Egg Harbor, New Jersey, United States

Site Status

Children'S Specialized Hospital

Toms River, New Jersey, United States

Site Status

Clinical Research Center of Nj

Voorhees Township, New Jersey, United States

Site Status

Lovelace Scientific Resources

Albuquerque, New Mexico, United States

Site Status

Richmond Behavioral Associates

Staten Island, New York, United States

Site Status

Montefiore Med.Center, Autism & Obsessive Compulsive Spectrum Prog.

The Bronx, New York, United States

Site Status

Duke Center For Autism and Brain Development

Durham, North Carolina, United States

Site Status

Cleveland Clinic, Center For Autism Research

Cleveland, Ohio, United States

Site Status

Omega Medical Research

Warwick, Rhode Island, United States

Site Status

Carolina Clinical Trials, Inc.

Charleston, South Carolina, United States

Site Status

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

Nashville, Tennessee, United States

Site Status

University of Texas, Houston Dept. of Psychiatry and Behavioral Sciences

Houston, Texas, United States

Site Status

Ericksen Research & Development

Clinton, Utah, United States

Site Status

University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences

Charlottesville, Virginia, United States

Site Status

Neuroscience, Inc.

Herndon, Virginia, United States

Site Status

Carilion Clinic-Virginia Tech, Carilion School of Medicine

Roanoke, Virginia, United States

Site Status

Countries

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United States

References

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Pearson DA, Hendren RL, Heil MF, McIntyre WR, Raines SR. Pancreatic Replacement Therapy for Maladaptive Behaviors in Preschool Children With Autism Spectrum Disorder. JAMA Netw Open. 2023 Nov 1;6(11):e2344136. doi: 10.1001/jamanetworkopen.2023.44136.

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Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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00103

Identifier Type: -

Identifier Source: org_study_id

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