Autism
Conditions
Keywords
Autism, A long-term extension study in a pediatric population of children with autism.
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.
Interventions
DRUGCM-AT
Single unit does powder of active substance (CM-AT) administered 3 times per day
Sponsors
Curemark
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | 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
| Measure | 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. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORDeborah Pearson, PhD
The University of Texas Health Science Center, Houston
PRINCIPAL_INVESTIGATORRobert Hendren, DO
University of California, San Francisco
Outcome results
None listed