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

NCT02410902 | Completed Phase 3 Results

• 1 other identifier: 00103
• Study Type: interventional
• Target: 190
• Locations: 1 country
• Sites: 31

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.

Conditions

Autism

Keywords

Autism

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• 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

  Screening through Week 12/Termination.

Study Arms (2)

CM-AT

EXPERIMENTAL

Active substance in single unit dose powder

Drug: CM-AT

Placebo

PLACEBO COMPARATOR

Placebo powder of inactive substance

Drug: PLACEBO

Interventions

CM-AT DRUG

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

CM-AT

PLACEBO DRUG

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

Also known as: placebo powder

Placebo

Eligibility Criteria

• Age: 3 Years - 8 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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;

You may not qualify if:

• 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;
• 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

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

Florida Hospital Medical Group-Lake Mary Pediatrics

Orange City, Florida, 32763, United States

Location

Kaley Kildahl

Orlando, Florida, 32803, United States

Location

Research Institute of Deaconess Clinic

Evansville, Indiana, 47713, 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.Center, Autism & Obsessive Compulsive Spectrum Prog.

The Bronx, New York, 10467, United States

Location

Duke Center For Autism and Brain Development

Durham, North Carolina, 27705, United States

Location

Cleveland Clinic, Center For Autism Research

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

Nashville, Tennessee, 37232-2551, United States

Location

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

Houston, Texas, 77054, United States

Location

Ericksen Research & Development

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

Related Publications (29)

• McPheeters ML, Warren Z, Sathe N, Bruzek JL, Krishnaswami S, Jerome RN, Veenstra-Vanderweele J. A systematic review of medical treatments for children with autism spectrum disorders. Pediatrics. 2011 May;127(5):e1312-21. doi: 10.1542/peds.2011-0427. Epub 2011 Apr 4.

  PMID: 21464191 BACKGROUND

• Autism and Developmental Disabilities Monitoring Network Surveillance Year 2008 Principal Investigators; Centers for Disease Control and Prevention. Prevalence of autism spectrum disorders--Autism and Developmental Disabilities Monitoring Network, 14 sites, United States, 2008. MMWR Surveill Summ. 2012 Mar 30;61(3):1-19.

  PMID: 22456193 BACKGROUND

• Developmental Disabilities Monitoring Network Surveillance Year 2010 Principal Investigators; Centers for Disease Control and Prevention (CDC). Prevalence of autism spectrum disorder among children aged 8 years - autism and developmental disabilities monitoring network, 11 sites, United States, 2010. MMWR Surveill Summ. 2014 Mar 28;63(2):1-21.

  PMID: 24670961 BACKGROUND

• Peacock G, Amendah D, Ouyang L, Grosse SD. Autism spectrum disorders and health care expenditures: the effects of co-occurring conditions. J Dev Behav Pediatr. 2012 Jan;33(1):2-8. doi: 10.1097/DBP.0b013e31823969de.

  PMID: 22157409 BACKGROUND

• Ganz ML. The lifetime distribution of the incremental societal costs of autism. Arch Pediatr Adolesc Med. 2007 Apr;161(4):343-9. doi: 10.1001/archpedi.161.4.343.

  PMID: 17404130 BACKGROUND

• McElhanon BO, McCracken C, Karpen S, Sharp WG. Gastrointestinal symptoms in autism spectrum disorder: a meta-analysis. Pediatrics. 2014 May;133(5):872-83. doi: 10.1542/peds.2013-3995.

  PMID: 24777214 BACKGROUND

• Buie T, Campbell DB, Fuchs GJ 3rd, Furuta GT, Levy J, Vandewater J, Whitaker AH, Atkins D, Bauman ML, Beaudet AL, Carr EG, Gershon MD, Hyman SL, Jirapinyo P, Jyonouchi H, Kooros K, Kushak R, Levitt P, Levy SE, Lewis JD, Murray KF, Natowicz MR, Sabra A, Wershil BK, Weston SC, Zeltzer L, Winter H. Evaluation, diagnosis, and treatment of gastrointestinal disorders in individuals with ASDs: a consensus report. Pediatrics. 2010 Jan;125 Suppl 1:S1-18. doi: 10.1542/peds.2009-1878C.

  PMID: 20048083 BACKGROUND

• Samsam M, Ahangari R, Naser SA. Pathophysiology of autism spectrum disorders: revisiting gastrointestinal involvement and immune imbalance. World J Gastroenterol. 2014 Aug 7;20(29):9942-51. doi: 10.3748/wjg.v20.i29.9942.

  PMID: 25110424 BACKGROUND

• Elsabbagh M, Divan G, Koh YJ, Kim YS, Kauchali S, Marcin C, Montiel-Nava C, Patel V, Paula CS, Wang C, Yasamy MT, Fombonne E. Global prevalence of autism and other pervasive developmental disorders. Autism Res. 2012 Jun;5(3):160-79. doi: 10.1002/aur.239. Epub 2012 Apr 11.

  PMID: 22495912 BACKGROUND

• Wasfy M, Oyofo B, Elgindy A, Churilla A. Comparison of preservation media for storage of stool samples. J Clin Microbiol. 1995 Aug;33(8):2176-8. doi: 10.1128/jcm.33.8.2176-2178.1995.

  PMID: 7559972 BACKGROUND

• Cavallini G, Benini L, Brocco G, Riela A, Bovo P, Pederzoli P, Angelini G, Pelle C, Bertelli G, Scuro LA. The fecal chymotrypsin photometric assay in the evaluation of exocrine pancreatic capacity. Comparison with other direct and indirect pancreatic function tests. Pancreas. 1989;4(3):300-4. doi: 10.1097/00006676-198906000-00005.

  PMID: 2734275 BACKGROUND

• Matthews DM. Intestinal absorption of amino acids and peptides. Proc Nutr Soc. 1972 Sep;31(2):171-7. doi: 10.1079/pns19720033. No abstract available.

  PMID: 4563292 BACKGROUND

• Coutinho AM, Oliveira G, Morgadinho T, Fesel C, Macedo TR, Bento C, Marques C, Ataide A, Miguel T, Borges L, Vicente AM. Variants of the serotonin transporter gene (SLC6A4) significantly contribute to hyperserotonemia in autism. Mol Psychiatry. 2004 Mar;9(3):264-71. doi: 10.1038/sj.mp.4001409.

  PMID: 15094787 BACKGROUND

• Naushad SM, Jain JM, Prasad CK, Naik U, Akella RR. Autistic children exhibit distinct plasma amino acid profile. Indian J Biochem Biophys. 2013 Oct;50(5):474-8.

  PMID: 24772971 BACKGROUND

• 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.

  PMID: 25155956 BACKGROUND

• Balasubramanian MN, Butterworth EA, Kilberg MS. Asparagine synthetase: regulation by cell stress and involvement in tumor biology. Am J Physiol Endocrinol Metab. 2013 Apr 15;304(8):E789-99. doi: 10.1152/ajpendo.00015.2013. Epub 2013 Feb 12.

  PMID: 23403946 BACKGROUND

• Fairclough PD, Hegarty JE, Silk DB, Clark ML. Comparison of the absorption of two protein hydrolysates and their effects on water and electrolyte movements in the human jejunum. Gut. 1980 Oct;21(10):829-34. doi: 10.1136/gut.21.10.829.

  PMID: 7192244 BACKGROUND

• Arnold GL, Hyman SL, Mooney RA, Kirby RS. Plasma amino acids profiles in children with autism: potential risk of nutritional deficiencies. J Autism Dev Disord. 2003 Aug;33(4):449-54. doi: 10.1023/a:1025071014191.

  PMID: 12959424 BACKGROUND

• Munasinghe SA, Oliff C, Finn J, Wray JA. Digestive enzyme supplementation for autism spectrum disorders: a double-blind randomized controlled trial. J Autism Dev Disord. 2010 Sep;40(9):1131-8. doi: 10.1007/s10803-010-0974-2.

  PMID: 20204691 BACKGROUND

• Schreck KA, Williams K, Smith AF. A comparison of eating behaviors between children with and without autism. J Autism Dev Disord. 2004 Aug;34(4):433-8. doi: 10.1023/b:jadd.0000037419.78531.86.

  PMID: 15449518 BACKGROUND

• Bailey DB Jr, Raspa M, Olmsted M, Holiday DB. Co-occurring conditions associated with FMR1 gene variations: findings from a national parent survey. Am J Med Genet A. 2008 Aug 15;146A(16):2060-9. doi: 10.1002/ajmg.a.32439.

  PMID: 18570292 BACKGROUND

• Lecavalier L. An evaluation of the Gilliam Autism Rating Scale. J Autism Dev Disord. 2005 Dec;35(6):795-805. doi: 10.1007/s10803-005-0025-6.

  PMID: 16283084 BACKGROUND

• Marcus RN, Owen R, Kamen L, Manos G, McQuade RD, Carson WH, Aman MG. A placebo-controlled, fixed-dose study of aripiprazole in children and adolescents with irritability associated with autistic disorder. J Am Acad Child Adolesc Psychiatry. 2009 Nov;48(11):1110-1119. doi: 10.1097/CHI.0b013e3181b76658.

  PMID: 19797985 BACKGROUND

• Yerys BE, Wallace GL, Sokoloff JL, Shook DA, James JD, Kenworthy L. Attention deficit/hyperactivity disorder symptoms moderate cognition and behavior in children with autism spectrum disorders. Autism Res. 2009 Dec;2(6):322-33. doi: 10.1002/aur.103.

  PMID: 19998356 BACKGROUND

• Schreck KA, Williams K. Food preferences and factors influencing food selectivity for children with autism spectrum disorders. Res Dev Disabil. 2006 Jul-Aug;27(4):353-63. doi: 10.1016/j.ridd.2005.03.005. Epub 2005 Jul 25.

  PMID: 16043324 BACKGROUND

• Borowitz D. Update on the evaluation of pancreatic exocrine status in cystic fibrosis. Curr Opin Pulm Med. 2005 Nov;11(6):524-7. doi: 10.1097/01.mcp.0000181474.08058.b3.

  PMID: 16217179 BACKGROUND

• Penn AH, Hugli TE, Schmid-Schonbein GW. Pancreatic enzymes generate cytotoxic mediators in the intestine. Shock. 2007 Mar;27(3):296-304. doi: 10.1097/01.shk.0000235139.20775.7f.

  PMID: 17304111 BACKGROUND

• Williams K, Wheeler DM, Silove N, Hazell P. Selective serotonin reuptake inhibitors (SSRIs) for autism spectrum disorders (ASD). Cochrane Database Syst Rev. 2010 Aug 4;(8):CD004677. doi: 10.1002/14651858.CD004677.pub2.

  PMID: 20687077 BACKGROUND

• 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.

  PMID: 38032645 DERIVED

MeSH Terms

Conditions

Autistic Disorder

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: William E. Gannon, Jr MD, Medical Director
• Organization: Curemark

william.gannon@curemark.com

914-824-9716

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

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2015
• First Posted: April 8, 2015
• Study Start: May 13, 2015
• Primary Completion: December 22, 2017
• Study Completion: December 22, 2017
• Last Updated: May 24, 2023
• Results First Posted: October 26, 2022

Record last verified: 2022-09

Locations

US (31)