What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) include behavioral therapies, medications, and dietary supplements. Behavioral therapies generally have minimal side effects but can be time-consuming and require significant effort. Medications such as antipsychotics (e.g., risperidone and aripiprazole) can cause weight gain, drowsiness, and hormonal changes. Dietary supplements, including omega-3 fatty acids, may cause gastrointestinal distress like nausea and diarrhea. Enzyme replacement therapies like CM-AT, aimed at improving digestive function, are still under study, but potential side effects could include gastrointestinal issues similar to other enzyme supplements.

What prior FDA approvals exist?

Currently, there are no FDA-approved enzyme replacement or supplement therapies specifically for the treatment of Autism Spectrum Disorder (ASD) aimed at improving digestive function, such as CM-AT. The FDA has approved medications like risperidone and aripiprazole to address irritability associated with ASD, but these are antipsychotic drugs and do not target digestive issues. Other treatments under investigation, such as memantine, have not demonstrated clinical efficacy for core symptoms of ASD. Therefore, enzyme replacement or supplement therapies like CM-AT represent a novel approach in the treatment landscape for ASD.

What other types of research exist?

One promising alternative to CM-AT for ASD patients is omega-3 fatty acid supplementation. Although evidence is inconsistent, some studies suggest small beneficial effects on language, social deficits, and associated symptoms like inattention and irritability. Another potential option is the use of probiotics, which have shown benefits in gastrointestinal health and may indirectly improve ASD symptoms. Both options should be discussed with a healthcare provider to tailor treatment to the individual needs of the patient.

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CM-AT for Autism

Q: What is the mechanism of action of this treatment?

Enzyme replacement or supplement therapies, such as CM-AT, aim to improve digestive function by enhancing the breakdown and absorption of nutrients, which may be impaired in some individuals with Autism Spectrum Disorder (ASD). This is based on the hypothesis that better nutrient absorption can positively influence brain function and behavior. Other common treatments include dietary interventions like gluten-free casein-free (GFCF) diets, which are thought to reduce gut permeability and subsequent behavioral issues, although evidence is limited. Pharmacological treatments often target neurotransmitter systems to alleviate symptoms like irritability and hyperactivity. These treatments matter for ASD patients as they offer potential improvements in core symptoms and overall quality of life, though their efficacy and safety vary and should be carefully monitored by healthcare providers.

Phase 3

Waitlist Available

Led By Deborah Pearson, PhD

Research Sponsored by Curemark

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

History of severe head trauma, as defined by loss of consciousness or hospitalization, skull fracture or stroke

Patient weighing < 13kg

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a special enzyme powder called CM-AT to help children with autism digest protein better.

Who is the study for?

This trial is for children with autism aged 3-8 who participated in a prior study (Sponsor's 00103 Study) or are currently in the open label study (00102). It also includes new participants aged 9-17 meeting DSM-IV-TR criteria for Autistic Disorder. Exclusions include severe head trauma, allergy to porcine products, SSRI use without stable dosing, significant systemic disease, dietary restrictions except nut allergies and lactose-free diets, weight under 13kg, trypsin sensitivities/allergies, ADHD medication use within five days of starting the trial.

What is being tested?

The clinical trial is testing CM-AT on pediatric patients with autism to evaluate its safety and effectiveness over time. This Phase III trial allows all levels of fecal chymotrypsin among participants and does not involve a control group as it is an open-label extension study.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include allergic reactions due to porcine product ingredients in CM-AT. Participants should be monitored for any adverse effects related to digestive enzymes present in the treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 9-17 years old and have been diagnosed with Autism.

Select...

I am between 3 and 8 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have had a severe head injury, including loss of consciousness, hospitalization, skull fracture, or stroke.

Select...

I weigh less than 13 kilograms.

Select...

I have had a seizure or needed seizure medication in the last year.

Select...

I cannot take the study drug as prescribed.

Select...

I have a severe or uncontrolled illness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2017 Phase 3 trial • 190 Patients • NCT02410902

26%

Nasopharyngitis

18%

Constipation

16%

Pyrexia

16%

Stool pH Decreased

12%

Vomiting

12%

Upper Respiratory Tract Infections

11%

Oropharyngeal Pain

Ear Pain

Clostridium Test Positive

Cough

Seasonal Allergy

Diarrhoea

Rash

Headache

Stool pH Increased

Stool Analysis Abnormal

100%

80%

60%

40%

20%

Study treatment Arm

CM-AT

Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

CM-AT

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CM-AT

2010

Completed Phase 3

~540

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Enzyme replacement or supplement therapies, such as CM-AT, aim to improve digestive function by enhancing the breakdown and absorption of nutrients, which may be impaired in some individuals with Autism Spectrum Disorder (ASD). This is based on the hypothesis that better nutrient absorption can positively influence brain function and behavior. Other common treatments include dietary interventions like gluten-free casein-free (GFCF) diets, which are thought to reduce gut permeability and subsequent behavioral issues, although evidence is limited. Pharmacological treatments often target neurotransmitter systems to alleviate symptoms like irritability and hyperactivity. These treatments matter for ASD patients as they offer potential improvements in core symptoms and overall quality of life, though their efficacy and safety vary and should be carefully monitored by healthcare providers.

Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis.Nutritional interventions for autism spectrum disorder.Dietary Supplement for Core Symptoms of Autism Spectrum Disorder: Where Are We Now and Where Should We Go?