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

Autism Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02410902
• Other study ID #: 00103
• Status: Completed
• Phase: Phase 3
• Start date: May 13, 2015
• Est. completion date: December 22, 2017

Study information

• Verified date: September 2022
• Source: Curemark
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 190
• Est. completion date: December 22, 2017
• Est. primary completion date: December 22, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 3 Years to 8 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism
• Autistic Disorder

Intervention

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

Locations

Country	Name	City	State
United States	Lovelace Scientific Resources	Albuquerque	New Mexico
United States	Detroit Clinical Research Center, P.C.	Bingham Farms	Michigan
United States	Montefiore Med.Center, Autism & Obsessive Compulsive Spectrum Prog.	Bronx	New York
United States	IMMUNOe RESEARCH CENTERS	Centennial	Colorado
United States	Carolina Clinical Trials, Inc.	Charleston	South Carolina
United States	University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences	Charlottesville	Virginia
United States	Cleveland Clinic, Center For Autism Research	Cleveland	Ohio
United States	Ericksen Research & Development	Clinton	Utah
United States	Duke Center For Autism and Brain Development	Durham	North Carolina
United States	Children'S Specialized Hospital	Egg Harbor Township	New Jersey
United States	Research Institute of Deaconess Clinic	Evansville	Indiana
United States	Neuroscience, Inc.	Herndon	Virginia
United States	University of Texas, Houston Dept. of Psychiatry and Behavioral Sciences	Houston	Texas
United States	Lake Charles Clinical Trials	Lake Charles	Louisiana
United States	Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)	Little Rock	Arkansas
United States	Vanderbilt University Med.Center -Treatment & Research Inst. For Asd	Nashville	Tennessee
United States	Yale Child Study Center	New Haven	Connecticut
United States	Segal Institute For Clinical Research	North Miami	Florida
United States	N.R.C. Research Institute	Orange	California
United States	Florida Hospital Medical Group-Lake Mary Pediatrics	Orange City	Florida
United States	Kaley Kildahl	Orlando	Florida
United States	Southwest Autism Research & Resource Center (S.A.R.R.C.)	Phoenix	Arizona
United States	Carilion Clinic-Virginia Tech, Carilion School of Medicine	Roanoke	Virginia
United States	M.I.N.D. Institute (Univ.of California, Davis)	Sacramento	California
United States	University of California (U.C.S.F.)	San Francisco	California
United States	L.S.U. Health Sciences Center	Shreveport	Louisiana
United States	Richmond Behavioral Associates	Staten Island	New York
United States	Children'S Specialized Hospital	Toms River	New Jersey
United States	University of Arizona, Pediatrics Multidisciplinary Research Unit	Tucson	Arizona
United States	Clinical Research Center of Nj	Voorhees	New Jersey
United States	Omega Medical Research	Warwick	Rhode Island

Sponsors (1)

Lead Sponsor	Collaborator
Curemark

Country where clinical trial is conducted

United States, 

References & Publications (28)

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Tang G, Gudsnuk K, Kuo SH, Cotrina ML, Rosoklija G, Sosunov A, Sonders MS, Kanter E, Castagna C, Yamamoto A, Yue Z, Arancio O, Peterson BS, Champagne F, Dwork AJ, Goldman J, Sulzer D. Loss of mTOR-dependent macroautophagy causes autistic-like synaptic pruning deficits. Neuron. 2014 Sep 3;83(5):1131-43. doi: 10.1016/j.neuron.2014.07.040. Epub 2014 Aug 21. Erratum In: Neuron. 2014 Sep 17;83(6):1482. — View Citation

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* Note: There are 28 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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	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	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.