Back to resultsAn Open Label Study of CM-AT for the Treatment of Children With Autism
Primary Purpose
Autism
Status
Active
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
CM-AT
Sponsored by
About this trial
This is an interventional treatment trial for Autism focused on measuring Autism
Eligibility Criteria
Inclusion Criteria:
- Age between 3 and 8 years, inclusive, at the time of signing informed consent/assent in Sponsor 00103 Study
- Completion of the Sponsor's 00103 Study who continue to meet eligibility requirements
- Currently in the 00102 open label study and continue to meet eligibility requirements
- Subjects who initially qualified for 00103 screening, who subsequently failed Baseline entrance criteria for randomization (@Visit 1) Baseline assessment of the ABC eligibility requirement who continue to meet eligibility requirements
- Up to 20 subjects 9-17 years of age who directly enroll into this study, who meet the current Diagnostic and Statistical Manual for Mental Disorders (DSM-IV-TR) diagnostic criteria for Autistic Disorder (AD), screened by the SCQ and confirmed by the ADI-R
Exclusion Criteria:
- Patient weighing < 13kg
- Allergy to porcine products
- Previous sensitization or allergy to trypsin, pancreatin, or pancrelipase
- History of severe head trauma, as defined by loss of consciousness or hospitalization, skull fracture or stroke.
- Seizure within the last year prior to enrollment, or the need for seizure medications either at present or in the past.
- Evidence or history of severe, moderate or uncontrolled systemic disease
- Ongoing dietary restriction for allergy or other reasons except nut allergies. Lactose free is allowable but not dairy free.
- Inability to ingest the study drug / non-compliance with dosing schedule.
- Inability to follow the prescribed dosing schedule.
- Use of any stimulant or non-stimulant medication or medications given for attention deficit hyperactivity disorder (ADHD) must be discontinued 5 days prior to the initial randomized study period.
- Subjects taking an selective serotonin reuptake inhibitor (SSRI) must be on a stable dose for a minimum of 30 days prior to entering the study.
- History of premature birth <35 weeks gestation.
- Prior history of stroke in utero or other in utero insult.
Sites / Locations
- Southwest Autism Research & Resource Center (S.A.R.R.C.)
- University of Arizona, Pediatrics Multidisciplinary Research Unit
- Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)
- N.R.C. Research Institute
- M.I.N.D. Institute (Univ.of California, Davis)
- University of California (U.C.S.F.)
- IMMUNOe Research Centers
- Yale Child Study Center
- Segal Institute For Clinical Research
- Advent Health -Lake Mary Pediatrics
- A.P.G. Research
- Research Institute of Deaconess Clinic
- Lake Charles Clinical Trials
- L.S.U. Health Sciences Center
- Detroit Clinical Research Center, P.C.
- Children'S Specialized Hospital
- Barbara Enright
- Clinical Research Center of Nj
- Lovelace Scientific Resources
- Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program
- Richmond Behavioral Associates
- Duke Center For Autism and Brain Development
- Cleveland Clinic Autism Center
- Omega Medical Research
- Carolina Clinical Trials, Inc.
- Vanderbilt Universtiy Med.Center-Treatment&Research Inst. For Asd
- University of Texas, Houston-Behavioral & Biomedical Sciences
- Focus Center of Clinical Research
- University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences
- Neuroscience, Inc
- Carilion Clinic-Virginia Tech, Carilion School of Medicine
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open Label
Arm Description
CM-AT
Outcomes
Primary Outcome Measures
Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Secondary Outcome Measures
Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02649959
Brief Title
An Open Label Study of CM-AT for the Treatment of Children With Autism
Official Title
An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 2015 (Actual)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
January 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Curemark
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase III, open label extension study evaluating the continued safety and efficacy of CM-AT in pediatric patients with autism with all levels of fecal chymotrypsin.
Detailed Description
Autism is clearly a significant cause of disability in the pediatric population. Treatment is based on the observation that many children with autism do not digest protein. CM-AT is a proprietary enzyme that is designed as a granulated powder taken three times daily.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism
Keywords
Autism
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
405 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Open Label
Arm Type
Experimental
Arm Description
CM-AT
Intervention Type
Drug
Intervention Name(s)
CM-AT
Intervention Description
Single unit does powder of active substance (CM-AT) administered 3 times per day
Primary Outcome Measure Information:
Title
Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Time Frame
Change from Baseline to each post-baseline visit, through study completion Week 72.
Secondary Outcome Measure Information:
Title
Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Time Frame
Change from Baseline to each post-baseline visit, through study completion Week 72.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Age between 3 and 8 years, inclusive, at the time of signing informed consent/assent in Sponsor 00103 Study
Completion of the Sponsor's 00103 Study who continue to meet eligibility requirements
Currently in the 00102 open label study and continue to meet eligibility requirements
Subjects who initially qualified for 00103 screening, who subsequently failed Baseline entrance criteria for randomization (@Visit 1) Baseline assessment of the ABC eligibility requirement who continue to meet eligibility requirements
Up to 20 subjects 9-17 years of age who directly enroll into this study, who meet the current Diagnostic and Statistical Manual for Mental Disorders (DSM-IV-TR) diagnostic criteria for Autistic Disorder (AD), screened by the SCQ and confirmed by the ADI-R
Exclusion Criteria:
Patient weighing < 13kg
Allergy to porcine products
Previous sensitization or allergy to trypsin, pancreatin, or pancrelipase
History of severe head trauma, as defined by loss of consciousness or hospitalization, skull fracture or stroke.
Seizure within the last year prior to enrollment, or the need for seizure medications either at present or in the past.
Evidence or history of severe, moderate or uncontrolled systemic disease
Ongoing dietary restriction for allergy or other reasons except nut allergies. Lactose free is allowable but not dairy free.
Inability to ingest the study drug / non-compliance with dosing schedule.
Inability to follow the prescribed dosing schedule.
Use of any stimulant or non-stimulant medication or medications given for attention deficit hyperactivity disorder (ADHD) must be discontinued 5 days prior to the initial randomized study period.
Subjects taking an selective serotonin reuptake inhibitor (SSRI) must be on a stable dose for a minimum of 30 days prior to entering the study.
History of premature birth <35 weeks gestation.
Prior history of stroke in utero or other in utero insult.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Deborah Pearson, PhD
Organizational Affiliation
The University of Texas Health Science Center, Houston
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Robert Hendren, DO
Organizational Affiliation
University of California, San Francisco
Official's Role
Principal Investigator
Facility Information:
Facility Name
Southwest Autism Research & Resource Center (S.A.R.R.C.)
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
University of Arizona, Pediatrics Multidisciplinary Research Unit
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
N.R.C. Research Institute
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
M.I.N.D. Institute (Univ.of California, Davis)
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California (U.C.S.F.)
City
San Francisco
State/Province
California
ZIP/Postal Code
94143-0984
Country
United States
Facility Name
IMMUNOe Research Centers
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Yale Child Study Center
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
Facility Name
Segal Institute For Clinical Research
City
North Miami
State/Province
Florida
ZIP/Postal Code
33161
Country
United States
Facility Name
Advent Health -Lake Mary Pediatrics
City
Orange City
State/Province
Florida
ZIP/Postal Code
32763
Country
United States
Facility Name
A.P.G. Research
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Research Institute of Deaconess Clinic
City
Newburgh
State/Province
Indiana
ZIP/Postal Code
47630
Country
United States
Facility Name
Lake Charles Clinical Trials
City
Lake Charles
State/Province
Louisiana
ZIP/Postal Code
70629
Country
United States
Facility Name
L.S.U. Health Sciences Center
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71103
Country
United States
Facility Name
Detroit Clinical Research Center, P.C.
City
Bingham Farms
State/Province
Michigan
ZIP/Postal Code
48025
Country
United States
Facility Name
Children'S Specialized Hospital
City
Egg Harbor Township
State/Province
New Jersey
ZIP/Postal Code
08234
Country
United States
Facility Name
Barbara Enright
City
Toms River
State/Province
New Jersey
ZIP/Postal Code
08755
Country
United States
Facility Name
Clinical Research Center of Nj
City
Voorhees
State/Province
New Jersey
ZIP/Postal Code
08043
Country
United States
Facility Name
Lovelace Scientific Resources
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87108
Country
United States
Facility Name
Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Richmond Behavioral Associates
City
Staten Island
State/Province
New York
ZIP/Postal Code
10312
Country
United States
Facility Name
Duke Center For Autism and Brain Development
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Cleveland Clinic Autism Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44104
Country
United States
Facility Name
Omega Medical Research
City
Warwick
State/Province
Rhode Island
ZIP/Postal Code
02886
Country
United States
Facility Name
Carolina Clinical Trials, Inc.
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29407
Country
United States
Facility Name
Vanderbilt Universtiy Med.Center-Treatment&Research Inst. For Asd
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-2551
Country
United States
Facility Name
University of Texas, Houston-Behavioral & Biomedical Sciences
City
Houston
State/Province
Texas
ZIP/Postal Code
77054
Country
United States
Facility Name
Focus Center of Clinical Research
City
Clinton
State/Province
Utah
ZIP/Postal Code
84015
Country
United States
Facility Name
University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
Neuroscience, Inc
City
Herndon
State/Province
Virginia
ZIP/Postal Code
20170
Country
United States
Facility Name
Carilion Clinic-Virginia Tech, Carilion School of Medicine
City
Roanoke
State/Province
Virginia
ZIP/Postal Code
24014
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
21464191
Citation
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.
Results Reference
background
Citation
Baio, J. Prevalence of Autism Spectrum Disorders - Autism and Developmental Disabilities Monitoring Network, 14 Sites, United States. (2008), Retrieved from http://www.cdc.gov/mmwr/pdf/ss/ss6103.pdf
Results Reference
background
Citation
Baio, J. Prevalence of Autism Spectrum Disorders - Autism and Developmental Disabilities Monitoring Network, 14 Sites, United States.(2010). Retrieved from http://www.cdc.gov/mmwr/pdf/ss/ss6302.pdf
Results Reference
background
PubMed Identifier
22157409
Citation
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.
Results Reference
background
PubMed Identifier
17404130
Citation
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.
Results Reference
background
PubMed Identifier
24777214
Citation
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.
Results Reference
background
PubMed Identifier
20048083
Citation
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.
Results Reference
background
PubMed Identifier
25110424
Citation
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.
Results Reference
background
PubMed Identifier
22495912
Citation
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.
Results Reference
background
PubMed Identifier
7559972
Citation
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.
Results Reference
background
PubMed Identifier
2734275
Citation
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.
Results Reference
background
PubMed Identifier
4563292
Citation
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.
Results Reference
background
PubMed Identifier
15094787
Citation
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.
Results Reference
background
PubMed Identifier
24772971
Citation
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.
Results Reference
background
PubMed Identifier
20687077
Citation
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.
Results Reference
background
PubMed Identifier
25155956
Citation
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.
Results Reference
background
PubMed Identifier
23403946
Citation
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.
Results Reference
background
PubMed Identifier
7192244
Citation
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.
Results Reference
background
PubMed Identifier
12959424
Citation
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.
Results Reference
background
PubMed Identifier
20204691
Citation
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.
Results Reference
background
PubMed Identifier
15449518
Citation
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.
Results Reference
background
PubMed Identifier
18570292
Citation
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.
Results Reference
background
PubMed Identifier
16283084
Citation
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.
Results Reference
background
PubMed Identifier
19797985
Citation
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.
Results Reference
background
PubMed Identifier
19998356
Citation
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.
Results Reference
background
PubMed Identifier
16043324
Citation
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.
Results Reference
background
PubMed Identifier
16217179
Citation
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.
Results Reference
background
PubMed Identifier
17304111
Citation
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.
Results Reference
background
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An Open Label Study of CM-AT for the Treatment of Children With Autism
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