CM-AT for Autism

Not currently recruiting at 34 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Curemark

Must not be taking: Stimulants, Non-stimulants, ADHD meds

Disqualifiers: Seizures, Severe trauma, Systemic disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of CM-AT, a treatment for children with autism. The goal is to evaluate how well CM-AT works and its safety for long-term use in children with autism and varying levels of the digestive enzyme fecal chymotrypsin. Eligible participants have either taken part in related studies or meet specific diagnostic criteria for autism. The trial is open to children who can take the medication as directed and do not have severe allergies or other major health issues. As a Phase 3 trial, this is the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment for autism.

Will I have to stop taking my current medications?

If you are taking medication for ADHD, you must stop 5 days before the study starts. If you are on an SSRI, you need to be on a stable dose for at least 30 days before joining the study.

Is there any evidence suggesting that CM-AT is likely to be safe for humans?

Research has shown that CM-AT, a treatment aiding digestion, is well tolerated by children with autism. In a study involving 190 children aged 3 to 6 years, CM-AT addressed challenging behaviors. The study found that children handled the treatment well, with no major safety issues. No serious side effects related to the treatment were reported.

These results suggest that CM-AT is generally safe for children with autism. The treatment's presence in Phase 3 trials indicates strong safety data already supports its use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

CM-AT is unique because it targets the digestive system, aiming to improve enzyme activity related to protein breakdown, which might be linked to autism symptoms. Unlike traditional autism treatments that focus on behavioral therapy or medications to manage symptoms, CM-AT addresses potential underlying metabolic issues. Researchers are excited about CM-AT because it could offer a novel approach by improving protein digestion, potentially leading to better overall outcomes for individuals with autism.

What evidence suggests that CM-AT might be an effective treatment for autism?

Research has shown that CM-AT, the treatment under study in this trial, might help with core symptoms of autism. In a study involving 190 children aged 3 to 6 years with autism spectrum disorder (ASD), those who received CM-AT showed improvements in behaviors such as irritability. Another study on core ASD behaviors found that children taking CM-AT experienced positive changes. These findings suggest CM-AT could help reduce some challenging behaviors in children with autism. Although more research is needed, early results appear promising. ^{1 3 4 5 6}

Who Is on the Research Team?

Deborah Pearson, PhD

Principal Investigator

The University of Texas Health Science Center, Houston

Robert Hendren, DO

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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

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

Exclusion Criteria

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

Ongoing dietary restriction for allergy or other reasons except nut allergies. Lactose free is allowable but not dairy free

History of premature birth <35 weeks gestation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CM-AT, a proprietary enzyme, taken as a granulated powder three times daily

12 weeks

Open-label extension

Continued evaluation of the safety and efficacy of CM-AT in pediatric patients with autism

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CM-AT

Trial Overview 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.

How Is the Trial Designed?

Group I: Open Label Experimental Treatment 1 Intervention

CM-AT

Who Is Running the Clinical Trial?

Curemark

Lead Sponsor

Trials

4

Recruited

940+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02410902

A Trial of CM-AT in Children With Autism With All Levels ... 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 ...

2. jamanetwork.com jamanetwork.com/journals/jamanetworkopen/fullarticle/2812393

Pancreatic Replacement Therapy for Maladaptive ... In this cohort study, 190 children aged 3 to 6 years with ASD treated with high-protease pancreatic therapy improved the maladaptive behaviors of irritability ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4494609/

Outcome predictors in autism spectrum disorders ... The ANN model used in this study seems to be a promising tool for the identification of the variables involved in the positive response to TAU in autism.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT00881452

NCT00881452 | A Trial of CM-AT in Children With Autism Inclusion Criteria: Meets the current Diagnostic and Statistical Manual for Mental Disorders (DSM-IV-TR) diagnostic criteria for autistic disorder (AD).

5. nature.com nature.com/articles/s41591-022-01683-9

Safety and target engagement of an oral small-molecule ... Exploratory outcomes show improvement of core ASD behavior. In addition, we captured several domains of behavioral data for all study ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38032645/

Pancreatic Replacement Therapy for Maladaptive ... Our high-protease pancreatic replacement (CM-AT) was well tolerated with no emergent safety concerns or related serious adverse events noted.