Trial Outcomes & Findings for Folinic Acid in Autism (NCT NCT03771560)

NCT ID: NCT03771560

Last Updated: 2019-12-03

Results Overview

The Aberrant Behavior Checklist - parent reported version measures aberrant behavior in children and young adults. There are 58 questions.The scoring of one question can range from 0 (not a problem) to 3 (severe) points on a Likert scale. The total possible score range for the ABC is 0 - 174. Analysis will be performed for mean of total score change over time. Scoring from 0-3 Not a problem = 0, Slightly = 1, Moderately Serious =2, Severe =3 Lower score indicates better performance.

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 18 participants
• Primary outcome timeframe: Baseline to Week 12
• Results posted on: 2019-12-03

Participant Flow

Participant milestones

Measure	Folinic Acid Open-label In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Overall Study STARTED	18
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Folinic Acid in Autism

Baseline characteristics by cohort

Measure	Folinic Acid Open-label n=12 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Age, Categorical <=18 years	12 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	16 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 12

The Aberrant Behavior Checklist - parent reported version measures aberrant behavior in children and young adults. There are 58 questions.The scoring of one question can range from 0 (not a problem) to 3 (severe) points on a Likert scale. The total possible score range for the ABC is 0 - 174. Analysis will be performed for mean of total score change over time.

Scoring from 0-3

Not a problem = 0, Slightly = 1, Moderately Serious =2, Severe =3

Lower score indicates better performance.

Outcome measures

Measure	Folinic Acid Open-label n=12 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Aberrant Behavior Checklist (ABC) - Parent Reported Change	-2.4 score on a scale Interval -11.3 to 6.4

PRIMARY outcome

Timeframe: Baseline to Week 12

Population: Deleted one outlier for data analysis; deleted one subject because of missing survey time point from teacher.

The Aberrant Behavior Checklist - teacher reported version measures aberrant behavior in children and young adults. There are 58 questions. The scoring of ABC questions can range from 0 (not a problem) to 3 (severe) points on a likert scale. The total possible score range for the ABC is 0 - 174. Analysis will be performed for mean of total score change over time.

Scoring from 0-3

Not a problem = 0, Slightly = 1, Moderately Serious =2, Severe =3

Lower score indicates better performance.

Outcome measures

Measure	Folinic Acid Open-label n=10 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Aberrant Behavior Checklist (ABC) - Teacher Reported Change	1.2 score on a scale Interval -5.2 to 7.6

SECONDARY outcome

Timeframe: Baseline to Week 12

The Social Responsiveness Scale - parent reported version measures social ability in children and young adults. There are 65 questions. The questions on the scale with anchors 1 (Not True) - 4 (Almost Always True). The scoring of SRS questions can range from 0-3 (with possible reverse scoring) based on scoring instructions for data analysis. The total possible score range for the SRS is 0 - 195. Analysis will be performed for mean of total score change over time.

Anchors Not True = 1 Sometimes True = 2 Often True = 3 Almost Always True = 4

Lower score indicates better performance.

Outcome measures

Measure	Folinic Acid Open-label n=12 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Social Responsiveness Scale (SRS) - Parent Reported Change	-7.8 score on a scale Interval -17.3 to 1.6

SECONDARY outcome

Timeframe: Baseline to Week 12

Population: Deleted one outlier for data analysis; deleted one subject because of missing survey time point from teacher.

The Social Responsiveness Scale - teacher reported version measures social ability in children and young adults. There are 65 questions. The questions on the scale with anchors 1 (Not True) - 4 (Almost Always True). The scoring of SRS questions can range from 0-3 (with possible reverse scoring) based on scoring instructions for data analysis. The total possible score range for the SRS is 0 - 195. Analysis will be performed for mean of total score change over time.

Anchors Not True = 1 Sometimes True = 2 Often True = 3 Almost Always True = 4

Lower score indicates better performance.

Outcome measures

Measure	Folinic Acid Open-label n=10 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Social Responsiveness Scale (SRS) - Teacher Reported Change	-0.5 score on a scale Interval -7.0 to 13.5

SECONDARY outcome

Timeframe: Baseline to Week 12

Pediatric Quality of Life is reported by parent only and it assesses improvement of the child's overall quality of life through questions about physical, emotional, social and school functioning. There are 23 questions. The scoring of PedsQL questions can range from 0 (Never) to 4 (Almost Always) points on a Likert scale. Questions are reversed scored and linearly transformed to a 0 - 100 scale for data analysis as follows: 0=100, 1=75, 2=50, 3=23, 4=0. The total score = sum of all the questions over the number of items answered on. The total possible score range for the PedsQL is 0 - 100. Analysis will be performed for mean of total score change over time.

Scoring from 0 to 4

Never = 0, Almost Never = 1, Sometimes = 2, Often = 3, Almost Always =4

Higher score indicates better performance.

Outcome measures

Measure	Folinic Acid Open-label n=12 Participants In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Pediatric Quality of Life (PedsQL) - Parent Reported Change	-0.8 score on a scale Interval -5.2 to 3.5

Adverse Events

Folinic Acid Open-label

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Folinic Acid Open-label n=12 participants at risk In this open-label trial, all subjects will receive the twice daily dose of folinic acid. Folinic acid will be delivered in pill form at a weight-based dose. folinic acid: subject will take folinic acid daily for 12 weeks
Gastrointestinal disorders Upset Stomach	8.3% 1/12 • Number of events 1 • 4 months
General disorders Dry mouth	8.3% 1/12 • Number of events 1 • 4 months
Respiratory, thoracic and mediastinal disorders Cold	8.3% 1/12 • Number of events 1 • 4 months
Psychiatric disorders Increased aggression	8.3% 1/12 • Number of events 1 • 4 months
Gastrointestinal disorders Blood in Stool	8.3% 1/12 • Number of events 1 • 4 months

Additional Information

Dr. Robert Hendren

UCSF

Phone: 415-502-3500

Email: STAR@ucsf.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place