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.
Brief Title
An Open Label Study of CM-AT for the Treatment of Children With Autism
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.
Categories
Completion Date
Completion Date Type
Estimated
Conditions
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.
* 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.
Inclusion 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
* 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
Gender
All
Gender Based
false
Keywords
Autism
A long-term extension study in a pediatric population of children with autism.
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
17 Years
Minimum Age
3 Years
NCT Id
NCT02649959
Org Class
Industry
Org Full Name
Curemark
Org Study Id
00104/Open Label-Autism
Overall Status
Active, not recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin
Primary Outcomes
Outcome Measure
Aberrant Behavioral Checklist: Subscale of Irritability / Agitation (ABC-I) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Outcome Time Frame
Change from Baseline to each post-baseline visit, through study completion Week 72.
Secondary Outcomes
Outcome Time Frame
Change from Baseline to each post-baseline visit, through study completion Week 72.
Outcome Measure
Aberrant Behavior Checklist: Subscale of Lethargy / Social Withdrawal (ABC-L) at fecal chymotrypsin (FCT) levels less than or equal to 12.6
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
17
Minimum Age Number (converted to Years and rounded down)
3
Investigators
Investigator Type
Principal Investigator
Investigator Name
Eric Hollander
Investigator Email
eholland@montefiore.org
Investigator Phone
718-920-4287