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

May 22, 2023 updated by: Curemark

A Double Blind, Randomized, Placebo-Controlled Study of CM-AT for the Treatment of Autism in Children With All Levels of Fecal Chymotrypsin (FCT)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

190

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
        • Florida Hospital Medical Group-Lake Mary Pediatrics
      • Orlando, Florida, United States, 32803
        • Kaley Kildahl
    • Indiana
      • Evansville, Indiana, United States, 47713
        • 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 Township, New Jersey, United States, 08234
        • Children's Specialized Hospital
      • Toms River, New Jersey, United States, 08755
        • Children's Specialized Hospital
      • Voorhees, New Jersey, United States, 08043
        • Clinical Research Center of NJ
    • New Mexico
      • Albuquerque, New Mexico, United States, 87108
        • Lovelace Scientific Resources
    • New York
      • Bronx, New York, United States, 10467
        • Montefiore Med.Center, Autism & Obsessive Compulsive Spectrum Prog.
      • Staten Island, New York, United States, 10312
        • Richmond Behavioral Associates
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke Center For Autism and Brain Development
    • Ohio
      • Cleveland, Ohio, United States, 44104
        • Cleveland Clinic, Center For Autism Research
    • 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 University Med.Center -Treatment & Research Inst. For Asd
    • Texas
      • Houston, Texas, United States, 77054
        • University of Texas, Houston Dept. of Psychiatry and Behavioral Sciences
    • Utah
      • Clinton, Utah, United States, 84015
        • Ericksen Research & Development
    • 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 8 years (Child)

Accepts Healthy Volunteers

Yes

Description

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

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CM-AT
Active substance in single unit dose powder
Drug: CM-AT
Single unit dose powder of active substance (CM-AT) administered 3 times per day for 90 days
Placebo Comparator: Placebo
Placebo powder of inactive substance
Drug: PLACEBO
Single unit dose powder of non-active substance administered 3 times per day for 90 days
Other Names:
  • placebo powder

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Primary Outcome Measurements to Determine Efficacy of Treatment With CM-AT Versus Placebo for Changes in the Aberrant Behavior Checklist Subscale for Irritability / Agitation (ABC-I) Between Baseline and Week 12/Termination Visit
Time Frame: Screening through Week 12/Termination
Primary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist (ABC) - Community sub scale for Irritability/Agitation (ABC-I) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-I is one of five discrete sub scales measured by the ABC. The scale range is 0-45. A higher score reflects higher severity of symptoms (irritability). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree. The score was automatically calculated by the EDC.
Screening through Week 12/Termination

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Secondary Outcome Measurements of Changes in the Aberrant Behavior Checklist Checklist Subscale for Lethargy / Social Withdrawal (ABC-L) Between Baseline and Week 12/Termination Visit
Time Frame: Screening through Week 12/Termination.
Secondary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist- Community (ABC) sub scale for Lethargy / Social Withdrawal (ABC-L) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-L is one of five discrete sub scales measured by the ABC. The scale range is 0-48. A higher score reflects higher severity of symptoms (lethargy). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree.
Screening through Week 12/Termination.

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)

May 13, 2015

Primary Completion (Actual)

December 22, 2017

Study Completion (Actual)

December 22, 2017

Study Registration Dates

First Submitted

March 19, 2015

First Submitted That Met QC Criteria

April 2, 2015

First Posted (Estimate)

April 8, 2015

Study Record Updates

Last Update Posted (Actual)

May 24, 2023

Last Update Submitted That Met QC Criteria

May 22, 2023

Last Verified

September 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 00103

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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