An Open Label Study of CM-AT for the Treatment of Children With Autism

Study Overview
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Brief Summary
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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
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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.

Official Title
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An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin

Conditions
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Autism

Intervention / Treatment
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* Drug: CM-AT


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open Label<br>CM-AT | Drug: CM-AT<br>* Single unit does powder of active substance (CM-AT) administered 3 times per day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6 |  | Change from Baseline to each post-baseline visit, through study completion Week 72. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6 |  | Change from Baseline to each post-baseline visit, through study completion Week 72. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autism