Trial Outcomes & Findings for A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study) (NCT NCT02410902)

Last Updated: 2023-05-24

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

190 participants

Primary outcome timeframe

Screening through Week 12/Termination

Results posted on

2023-05-24

Participant Flow

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.

Participants were screened for autistic disorder using the Diagnostic Statistical Manual of Mental Disorders Fourth Edition - Text Revised (DSM-IV-TR); and the Social Communication Questionnaire (SCQ), with diagnostic confirmation by the Autism Diagnostic Interview-Revised (ADI-R). All qualifying participants were given a 2 week run-in period and then reassessed for eligibility and then randomized.

Participant milestones

Participant milestones
Measure	CM-AT Active substance in single unit dose powder CM-AT: Single unit dose powder of active substance, 900mg, (CM-AT) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.	Placebo Placebo powder of inactive substance PLACEBO: Single unit dose powder of non-active substance, 900mg, (Placebo) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.
Overall Study STARTED	92	98
Overall Study Randomized	92	98
Overall Study ITT	92	98
Overall Study COMPLETED	71	80
Overall Study NOT COMPLETED	21	18

Reasons for withdrawal

Reasons for withdrawal
Measure	CM-AT Active substance in single unit dose powder CM-AT: Single unit dose powder of active substance, 900mg, (CM-AT) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.	Placebo Placebo powder of inactive substance PLACEBO: Single unit dose powder of non-active substance, 900mg, (Placebo) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.
Overall Study Adverse Event	2	3
Overall Study Lost to Follow-up	4	1
Overall Study Protocol Violation	1	4
Overall Study Withdrawal by Subject	6	8
Overall Study Non-Compliance	8	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CM-AT n=92 Participants Active substance in single unit dose powder CM-AT: Single unit dose powder of active substance, 900mg, (CM-AT) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.	Placebo n=98 Participants Placebo powder of inactive substance PLACEBO: Single unit dose powder of non-active substance, 900mg, (Placebo) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.	Total n=190 Participants Total of all reporting groups
Age, Categorical <=18 years	92 Participants n=5 Participants	98 Participants n=7 Participants	190 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	5.7 Years STANDARD_DEVIATION 1.6 • n=5 Participants	5.7 Years STANDARD_DEVIATION 1.6 • n=7 Participants	5.7 Years STANDARD_DEVIATION 1.6 • n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	19 Participants n=7 Participants	40 Participants n=5 Participants
Sex: Female, Male Male	71 Participants n=5 Participants	79 Participants n=7 Participants	150 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	20 Participants n=5 Participants	80 Participants n=7 Participants	100 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	71 Participants n=5 Participants	18 Participants n=7 Participants	89 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	12 Participants n=5 Participants	10 Participants n=7 Participants	22 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	10 Participants n=5 Participants	8 Participants n=7 Participants	18 Participants n=5 Participants
Race (NIH/OMB) White	61 Participants n=5 Participants	70 Participants n=7 Participants	131 Participants n=5 Participants
Race (NIH/OMB) More than one race	7 Participants n=5 Participants	6 Participants n=7 Participants	13 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Region of Enrollment United States	92 participants n=5 Participants	98 participants n=7 Participants	190 participants n=5 Participants
ABC-I	22.2 units on a scale STANDARD_DEVIATION 7.6 • n=5 Participants	23.4 units on a scale STANDARD_DEVIATION 7.9 • n=7 Participants	22.8 units on a scale STANDARD_DEVIATION 7.75 • n=5 Participants

PRIMARY outcome

Timeframe: Screening through Week 12/Termination

Outcome measures

Outcome measures
Measure	CM-AT n=92 Participants Active substance in single unit dose powder Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT. Participants took 3 sachets of CM-AT in total: One at each meal	Placebo n=98 Participants Placebo powder of inactive substance Participants were between 3 through to 6 years old inclusive and took 900mg Placebo. Participants took 3 sachets of Placebo in total: One at each meal
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 units on a scale Standard Deviation 7.47	-5.5 units on a scale Standard Deviation 9.19

SECONDARY outcome

Timeframe: 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 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.

Outcome measures

Outcome measures
Measure	CM-AT n=92 Participants Active substance in single unit dose powder Participants were between 3 through to 6 years old inclusive and took 900mg CM-AT. Participants took 3 sachets of CM-AT in total: One at each meal	Placebo n=98 Participants Placebo powder of inactive substance Participants were between 3 through to 6 years old inclusive and took 900mg Placebo. Participants took 3 sachets of Placebo in total: One at each meal
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 units on a scale Standard Deviation 6.96	-6.6 units on a scale Standard Deviation 9.52

Adverse Events

CM-AT

Serious events: 0 serious events

Other events: 79 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 90 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

William E. Gannon, Jr MD, Medical Director

Curemark

Phone: 914-824-9716

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publications include input from PI and Curemark reflected in authorship. Institution's results may be submitted after results of the multicenter study are published, 18m after completion, or if multicenter publication isn't planned. Then PI may to publish, subject to confidentiality agreement. PI shall give to Curemark 45 days prior for review. Submission of such publication of results be can't delayed more than 105d after being received by Curemark. After 105 days, the PI may publish.
Publication restrictions are in place

Restriction type: OTHER

Measure	CM-AT n=92 participants at risk Active substance in single unit dose powder CM-AT: Single unit dose powder of active substance, 900mg, (CM-AT) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.	Placebo n=98 participants at risk Placebo powder of inactive substance PLACEBO: Single unit dose powder of non-active substance, 900mg, (Placebo) administered 3 times per day, one at each meal for 90 days. Participants were between 3 and 8 years old inclusive.
Infections and infestations Nasopharyngitis	26.1% 24/92 • Number of events 28 • 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.	28.6% 28/98 • Number of events 35 • 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.
Investigations Stool pH Decreased	16.3% 15/92 • Number of events 17 • 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.	22.4% 22/98 • Number of events 28 • 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.
Gastrointestinal disorders Constipation	18.5% 17/92 • Number of events 25 • 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.	14.3% 14/98 • Number of events 18 • 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.
General disorders Pyrexia	16.3% 15/92 • Number of events 22 • 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.	17.3% 17/98 • Number of events 27 • 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.
Gastrointestinal disorders Vomiting	12.0% 11/92 • Number of events 12 • 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.	14.3% 14/98 • Number of events 16 • 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.
Gastrointestinal disorders Diarrhoea	5.4% 5/92 • Number of events 10 • 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.	14.3% 14/98 • Number of events 17 • 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.
Infections and infestations Upper Respiratory Tract Infections	12.0% 11/92 • Number of events 16 • 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.	14.3% 14/98 • Number of events 19 • 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.
Investigations Stool Analysis Abnormal	3.3% 3/92 • 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.	11.2% 11/98 • 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.
Investigations Clostridium Test Positive	7.6% 7/92 • Number of events 8 • 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.	8.2% 8/98 • Number of events 8 • 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.
Nervous system disorders Headache	4.3% 4/92 • Number of events 5 • 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.	5.1% 5/98 • Number of events 7 • 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.
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	10.9% 10/92 • Number of events 13 • 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.	3.1% 3/98 • Number of events 3 • 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.
Respiratory, thoracic and mediastinal disorders Cough	7.6% 7/92 • Number of events 7 • 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.	8.2% 8/98 • Number of events 12 • 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.
Investigations Stool pH Increased	4.3% 4/92 • Number of events 4 • 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.	12.2% 12/98 • 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.
Ear and labyrinth disorders Ear Pain	8.7% 8/92 • Number of events 8 • 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.	1.0% 1/98 • 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.
Immune system disorders Seasonal Allergy	5.4% 5/92 • Number of events 5 • 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.	1.0% 1/98 • 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.
Skin and subcutaneous tissue disorders Rash	4.3% 4/92 • 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.	5.1% 5/98 • 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.