A Phase 3 interventional study of CM-AT and PLACEBO in Autism, sponsored by Curemark. Completed at 31 sites in United States. Open to participants aged 3 Years to 8 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-05-24.
Sponsored by Curemark · Phase 3, Interventional, and Treatment
The purpose of this study is to determine whether CM-AT is safe and effective in treating the core symptoms of autism in children with all levels of fecal chymotrypsin.
Autism is clearly a significant cause of disability in the pediatric population. Many children with Autism exhibit impaired protein digestion which may or may not manifest in self-restricted diets. The inability to digest protein affects the availability of essential amino acids in the body. CM-AT is designed to enhance protein digestion thereby potentially restoring the pool of essential amino acids. Essential amino acids play a critical role in the expression of several genes important to neurological function and serve as precursors to key neurotransmitters such as serotonin and dopamine. CM-AT is a proprietary enzyme that is designed as a granulated powder taken three times daily.
1,344 studies on the registry are indexed under Autistic Disorder; 334 are open to participants now.
This study's enrollment of 190 is above the median of 45 across 1,044 interventional studies indexed under Autistic Disorder.
Browse Autistic Disorder studies →Curemark is the lead sponsor of 4 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Active substance in single unit dose powder
Drug: CM-AT
Placebo powder of inactive substance
Drug: PLACEBO
Single unit dose powder of active substance (CM-AT) administered 3 times per day for 90 days
Single unit dose powder of non-active substance administered 3 times per day for 90 days
Also known as: placebo powder
Primary Outcome Measurements to Determine Efficacy of Treatment With CM-AT Versus Placebo for Changes in the Aberrant Behavior Checklist Subscale for Irritability / Agitation (ABC-I) Between Baseline and Week 12/Termination Visit
Primary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist (ABC) - Community sub scale for Irritability/Agitation (ABC-I) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-I is one of five discrete sub scales measured by the ABC. The scale range is 0-45. A higher score reflects higher severity of symptoms (irritability). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree. The score was automatically calculated by the EDC.
Time frame: Screening through Week 12/Termination
Secondary Outcome Measurements of Changes in the Aberrant Behavior Checklist Checklist Subscale for Lethargy / Social Withdrawal (ABC-L) Between Baseline and Week 12/Termination Visit
Secondary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist- Community (ABC) sub scale for Lethargy / Social Withdrawal (ABC-L) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-L is one of five discrete sub scales measured by the ABC. The scale range is 0-48. A higher score reflects higher severity of symptoms (lethargy). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree.
Time frame: Screening through Week 12/Termination.
Participants were recruited across 33 sites in the USA. The First Subject First Visit (FSFV) occurred on 03 June 2015 (First screening visit date) and the Last Subject Last Visit (LSLV) occurred on 20 December 2017.
| Milestone | CM-AT | Placebo |
|---|---|---|
| Started | 92 | 98 |
| Randomized | 92 | 98 |
| Itt | 92 | 98 |
| Completed | 71 | 80 |
| Not completed | 21 | 18 |
| Withdrew: Adverse event | 2 | 3 |
| Withdrew: Lost to follow-up | 4 | 1 |
| Withdrew: Protocol violation | 1 | 4 |
| Withdrew: Withdrawal by subject | 6 | 8 |
| Withdrew: Non-compliance | 8 | 2 |
Primary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist (ABC) - Community sub scale for Irritability/Agitation (ABC-I) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-I is one of five discrete sub scales measured by the ABC. The scale range is 0-45. A higher score reflects higher severity of symptoms (irritability). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree. The score was automatically calculated by the EDC.
| units on a scale | CM-AT | Placebo |
|---|---|---|
| Primary Outcome Measurements to Determine Efficacy of Treatment With CM-AT Versus Placebo for Changes in the Aberrant Behavior Checklist Subscale for Irritability / Agitation (ABC-I) Between Baseline and Week 12/Termination Visit | -8.0 ± 7.47 | -5.5 ± 9.19 |
Secondary outcome measurements to determine efficacy of treatment with CM-AT versus Placebo for changes in the Aberrant Behavior Checklist- Community (ABC) sub scale for Lethargy / Social Withdrawal (ABC-L) between baseline (subject's initial measurement) and Week 12/Termination (subject's final measurement) visit. Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT or Placebo three times daily. The ABC-L is one of five discrete sub scales measured by the ABC. The scale range is 0-48. A higher score reflects higher severity of symptoms (lethargy). Scores are obtained via Parent Rated Questionnaire. Parents respond to a series of questions on a scale directly into an electronic data capture system (EDC), responding: 0 = not at all a problem 1 = the behavior is a problem but slight in degree 2 = the problem is moderately serious 3 = the problem is severe in degree.
| units on a scale | CM-AT | Placebo |
|---|---|---|
| Secondary Outcome Measurements of Changes in the Aberrant Behavior Checklist Checklist Subscale for Lethargy / Social Withdrawal (ABC-L) Between Baseline and Week 12/Termination Visit | -7.9 ± 6.96 | -6.6 ± 9.52 |
Collected over Adverse Events were recorded from the time of consent through 30-days following subject completion of or withdrawal from study, equalling a maximum of 128 days.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CM-AT | 0/92 (0%) | 0/92 (0%) | 79/92 (85.9%) |
| Placebo | 0/98 (0%) | 0/98 (0%) | 90/98 (91.8%) |
| Event | CM-AT | Placebo |
|---|---|---|
| NasopharyngitisInfections and infestations | 24/92 | 28/98 |
| Stool pH DecreasedInvestigations | 15/92 | 22/98 |
| ConstipationGastrointestinal disorders | 17/92 | 14/98 |
| PyrexiaGeneral disorders | 15/92 | 17/98 |
| VomitingGastrointestinal disorders | 11/92 | 14/98 |
| DiarrhoeaGastrointestinal disorders | 5/92 | 14/98 |
| Upper Respiratory Tract InfectionsInfections and infestations | 11/92 | 14/98 |
| Stool pH IncreasedInvestigations | 4/92 | 12/98 |
| Stool Analysis AbnormalInvestigations | 3/92 | 11/98 |
| Oropharyngeal PainRespiratory, thoracic and mediastinal disorders | 10/92 | 3/98 |
| Age, Categorical(Participants) | CM-AT | Placebo | Total |
|---|---|---|---|
| <=18 years | 92 | 98 | 190 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Age, Continuous(Years) | CM-AT | Placebo | Total |
|---|---|---|---|
| Mean | 5.7 ± 1.6 | 5.7 ± 1.6 | 5.7 ± 1.6 |
| Sex: Female, Male(Participants) | CM-AT | Placebo | Total |
|---|---|---|---|
| Female | 21 | 19 | 40 |
| Male | 71 | 79 | 150 |
| Ethnicity (NIH/OMB)(Participants) | CM-AT | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 20 | 80 | 100 |
| Not Hispanic or Latino | 71 | 18 | 89 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | CM-AT | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 12 | 10 | 22 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 10 | 8 | 18 |
| White | 61 | 70 | 131 |
| More than one race | 7 | 6 | 13 |
| Unknown or Not Reported | 2 | 3 | 5 |
| Region of Enrollment(participants) | CM-AT | Placebo | Total |
|---|---|---|---|
| United States | 92 | 98 | 190 |
| ABC-I(units on a scale) | CM-AT | Placebo | Total |
|---|---|---|---|
| Mean | 22.2 ± 7.6 | 23.4 ± 7.9 | 22.8 ± 7.75 |
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