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

Phase 3
Completed
Conditions
Autism
Interventions
Drug: PLACEBO
Registration Number
NCT02410902
Lead Sponsor
Curemark
Brief Summary

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

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
190
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
CM-ATCM-ATActive substance in single unit dose powder
PlaceboPLACEBOPlacebo powder of inactive substance
Primary Outcome Measures
NameTimeMethod
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 VisitScreening 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.

Secondary Outcome Measures
NameTimeMethod
Secondary Outcome Measurements of Changes in the Aberrant Behavior Checklist Checklist Subscale for Lethargy / Social Withdrawal (ABC-L) Between Baseline and Week 12/Termination VisitScreening 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.

Trial Locations

Locations (30)

University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences

🇺🇸

Charlottesville, Virginia, United States

Carilion Clinic-Virginia Tech, Carilion School of Medicine

🇺🇸

Roanoke, Virginia, United States

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

🇺🇸

Nashville, Tennessee, United States

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

🇺🇸

Phoenix, Arizona, United States

University of Arizona, Pediatrics Multidisciplinary Research Unit

🇺🇸

Tucson, Arizona, United States

N.R.C. Research Institute

🇺🇸

Orange, California, United States

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

🇺🇸

Little Rock, Arkansas, United States

Yale Child Study Center

🇺🇸

New Haven, Connecticut, United States

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

🇺🇸

San Francisco, California, United States

IMMUNOe RESEARCH CENTERS

🇺🇸

Centennial, Colorado, United States

Kaley Kildahl

🇺🇸

Orlando, Florida, United States

Lake Charles Clinical Trials

🇺🇸

Lake Charles, Louisiana, United States

L.S.U. Health Sciences Center

🇺🇸

Shreveport, Louisiana, United States

Detroit Clinical Research Center, P.C.

🇺🇸

Bingham Farms, Michigan, United States

Clinical Research Center of Nj

🇺🇸

Voorhees, New Jersey, United States

Children'S Specialized Hospital

🇺🇸

Toms River, New Jersey, United States

Lovelace Scientific Resources

🇺🇸

Albuquerque, New Mexico, United States

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

🇺🇸

Bronx, New York, United States

Richmond Behavioral Associates

🇺🇸

Staten Island, New York, United States

Cleveland Clinic, Center For Autism Research

🇺🇸

Cleveland, Ohio, United States

Omega Medical Research

🇺🇸

Warwick, Rhode Island, United States

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

🇺🇸

Houston, Texas, United States

Ericksen Research & Development

🇺🇸

Clinton, Utah, United States

Neuroscience, Inc.

🇺🇸

Herndon, Virginia, United States

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

🇺🇸

Sacramento, California, United States

Segal Institute For Clinical Research

🇺🇸

North Miami, Florida, United States

Duke Center For Autism and Brain Development

🇺🇸

Durham, North Carolina, United States

Florida Hospital Medical Group-Lake Mary Pediatrics

🇺🇸

Orange City, Florida, United States

Research Institute of Deaconess Clinic

🇺🇸

Evansville, Indiana, United States

Carolina Clinical Trials, Inc.

🇺🇸

Charleston, South Carolina, United States

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