Clinical trial NCT02649959: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Autism
Keywords: Autism
A long-term extension study in a pediatric population of children with autism.
Lead sponsor: Curemark
Sponsor class: INDUSTRY
First posted: 2016-01-08
Enrollment: 405
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
timeFrame: Change from Baseline to each post-baseline visit, through study completion Week 72.
Secondary outcomes:
measure: Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
timeFrame: Change from Baseline to each post-baseline visit, through study completion Week 72.
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Southwest Autism Research & Resource Center (S.A.R.R.C.)
state: Arizona
city: Tucson
country: United States
facility: University of Arizona, Pediatrics Multidisciplinary Research Unit
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.)
state: Arkansas
city: Orange
country: United States
facility: N.R.C. Research Institute
state: California
city: Sacramento
country: United States
facility: M.I.N.D. Institute (Univ.of California, Davis)
state: California
city: San Francisco
country: United States
facility: University of California (U.C.S.F.)
state: California
city: Centennial
country: United States
facility: IMMUNOe Research Centers
state: Colorado
city: New Haven
country: United States
facility: Yale Child Study Center
state: Connecticut
city: North Miami
country: United States
facility: Segal Institute For Clinical Research
state: Florida
city: Orange City
country: United States
facility: Advent Health -Lake Mary Pediatrics
state: Florida
city: Orlando
country: United States
facility: A.P.G. Research
state: Florida
city: Newburgh
country: United States
facility: Research Institute of Deaconess Clinic
state: Indiana
city: Lake Charles
country: United States
facility: Lake Charles Clinical Trials
state: Louisiana
city: Shreveport
country: United States
facility: L.S.U. Health Sciences Center
state: Louisiana
city: Bingham Farms
country: United States
facility: Detroit Clinical Research Center, P.C.
state: Michigan
city: Egg Harbor Township
country: United States
facility: Children'S Specialized Hospital
state: New Jersey
city: Toms River
country: United States
facility: Children's Specialized Hospital
state: New Jersey
city: Voorhees
country: United States
facility: Clinical Research Center of Nj
state: New Jersey
city: Albuquerque
country: United States
facility: Lovelace Scientific Resources
state: New Mexico
city: Bronx
country: United States
facility: Montefiore Med.Cneter, Autism & Obsessive Compulsive Spectrum Program
state: New York
city: Staten Island
country: United States
facility: Richmond Behavioral Associates
state: New York
city: Durham
country: United States
facility: Duke Center For Autism and Brain Development
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Autism Center
state: Ohio
city: Warwick
country: United States
facility: Omega Medical Research
state: Rhode Island
city: Charleston
country: United States
facility: Carolina Clinical Trials, Inc.
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt Universtiy Med.Center-Treatment&Research Inst. For Asd
state: Tennessee
city: Houston
country: United States
facility: University of Texas, Houston-Behavioral & Biomedical Sciences
state: Texas
city: Clinton
country: United States
facility: Focus Center of Clinical Research
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia, Dept. of Psychiatry and Neurobehavioral Sciences
state: Virginia
city: Herndon
country: United States
facility: Neuroscience, Inc
state: Virginia
city: Roanoke
country: United States
facility: Carilion Clinic-Virginia Tech, Carilion School of Medicine
state: Virginia
Study officials:
affiliation: The University of Texas Health Science Center, Houston
name: Deborah Pearson, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, San Francisco
name: Robert Hendren, DO
role: PRINCIPAL_INVESTIGATOR
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