NCT02410902

A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study)

Completed Phase 3 Results posted Last updated 24 May 2023

What this trial tests

Phase 3 trial testing CM-AT in Autism in 190 participants. Completed in 22 December 2017.

Timeline

13 May 2015

Primary endpoint
22 December 2017

22 December 2017

Quick facts

Lead sponsor	Curemark
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	190
Start date	13 May 2015
Primary completion	22 December 2017
Estimated completion	22 December 2017
Sites	31 locations across United States

Drugs / interventions tested

• CM-AT — full drug profile →
• PLACEBO

Conditions studied

• Autism — all drugs for Autism →

Sponsor

Curemark — full company profile →

Who can join

Adults 3 to 8, any sex, with Autism. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

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 · Screening through Week 12/Termination

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

Group	Value	95% CI
CM-AT	-8.0	± 7.47
Placebo	-5.5	± 9.19

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 · Screening through Week 12/Termination.

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 obtai

Group	Value	95% CI
CM-AT	-7.9	± 6.96
Placebo	-6.6	± 9.52

Adverse events — posted to ClinicalTrials.gov

Time frame: 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.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

CM-AT

Serious: 0/92 (0%)

Deaths: 0/92

Placebo

Serious: 0/98 (0%)

Deaths: 0/98

Other adverse events (16 terms — click to expand)

Reaction	System	CM-AT	Placebo
Nasopharyngitis	Infections and infestations	—	—
Stool pH Decreased	Investigations	—	—
Constipation	Gastrointestinal disorders	—	—
Pyrexia	General disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Diarrhoea	Gastrointestinal disorders	—	—
Upper Respiratory Tract Infections	Infections and infestations	—	—
Stool pH Increased	Investigations	—	—
Stool Analysis Abnormal	Investigations	—	—
Oropharyngeal Pain	Respiratory, thoracic and mediastinal disorders	—	—
Clostridium Test Positive	Investigations	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Ear Pain	Ear and labyrinth disorders	—	—
Headache	Nervous system disorders	—	—
Seasonal Allergy	Immune system disorders	—	—
Rash	Skin and subcutaneous tissue disorders	—	—

Data from ClinicalTrials.gov NCT02410902 adverse events section.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Pancreatic Replacement Therapy for Maladaptive Behaviors in Preschool Children With Autism Spectrum Disorder.
   Pearson DA, Hendren RL, Heil MF, McIntyre WR, et al · · 2023 · cited 5× · PMID 38032645 · DOI 10.1001/jamanetworkopen.2023.44136