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

Phase 3

Active, not recruiting

• Conditions: Autism
• Interventions: Drug: CM-AT

• Registration Number: NCT02649959
• Lead Sponsor: Curemark

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.

Study Timeline

• Study Start: 2015-10
• Study First Submitted: 2015-11-12
• Study First Submitted QC: 2016-01-06
• Study First Posted: 2016-01-08
• Status Verified: 2025-01
• Last Update Submitted: 2025-02-11
• Last Update Posted: 2025-02-13
• Primary Completion: 2028-01
• Study Completion: 2028-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 405

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open Label	CM-AT	CM-AT

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (31)

Southwest Autism Research & Resource Center (S.A.R.R.C.); 🇺🇸 Phoenix, Arizona, United States

University of Arizona, Pediatrics Multidisciplinary Research Unit; 🇺🇸 Tucson, Arizona, United States

Arkansas Children'S Hosp. Research Institute (A.C.H.R.I.); 🇺🇸 Little Rock, Arkansas, United States

N.R.C. Research Institute; 🇺🇸 Orange, California, United States

M.I.N.D. Institute (Univ.of California, Davis); 🇺🇸 Sacramento, California, United States

University of California (U.C.S.F.); 🇺🇸 San Francisco, California, United States

IMMUNOe Research Centers; 🇺🇸 Centennial, Colorado, United States

Yale Child Study Center; 🇺🇸 New Haven, Connecticut, United States

Segal Institute For Clinical Research; 🇺🇸 North Miami, Florida, United States

Advent Health -Lake Mary Pediatrics; 🇺🇸 Orange City, Florida, United States

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