Trial Outcomes & Findings for Changing Developmental Trajectories Through Early Treatment (NCT NCT01985022)

NCT ID: NCT01985022

Last Updated: 2023-06-09

Results Overview

Social communication skills were measured with the Communication and Symbolic Behavior Scales (CSBS) Behavior Sample. Interactions between the child and caregiver were videotaped and later converted to scores. The standardized total score has a mean of 100 and standard deviation of 15. Scores below 100 indicate below average performance with the social, speech, and symbolic composites.

Recruitment status

TERMINATED

Study phase

Target enrollment

33 participants

Primary outcome timeframe

Baseline (12 months of age, prior to treatment), 15, 21, and 24 months of age

Results posted on

2023-06-09

Participant Flow

Participants were recruited from Marcus Autism Center in Atlanta, Georgia, USA. Participant enrollment began in May 2013 and follow-up was completed by November 26, 2019.

Infants were enrolled with a parent as parents were a necessary part of the intervention that the infants received. Parents are not considered to be participants who received a study intervention, thus, only infants are described in the modules for Participant Flow, Baseline Characteristics, Outcome Measures, and Adverse Events.

Participant milestones

Participant milestones
Measure	Group-Early Social Interaction (ESI) Infants 12 months of age at risk of developing autism spectrum disorders (ASD) who were randomized to receive the Group-Early Social Interaction (ESI) intervention for 9 months	Individual-ESI Plus Group-ESI Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Overall Study STARTED	16	17
Overall Study COMPLETED	12	14
Overall Study NOT COMPLETED	4	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Group-Early Social Interaction (ESI) Infants 12 months of age at risk of developing autism spectrum disorders (ASD) who were randomized to receive the Group-Early Social Interaction (ESI) intervention for 9 months	Individual-ESI Plus Group-ESI Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Overall Study Lost to Follow-up	0	1
Overall Study Protocol Violation	0	1
Overall Study Withdrawal by Subject	4	1

Baseline Characteristics

Changing Developmental Trajectories Through Early Treatment

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive the Group-ESI intervention for 9 months	Individual-ESI Plus Group-ESI n=17 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.	Total n=33 Participants Total of all reporting groups
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 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	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants	11 Participants n=7 Participants	23 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical <=18 years	16 Participants n=5 Participants	17 Participants n=7 Participants	33 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
Sex: Female, Male Female	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	11 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants	17 Participants n=7 Participants	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	16 Participants n=5 Participants	17 Participants n=7 Participants	33 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (12 months of age, prior to treatment), 15, 21, and 24 months of age

Population: This analysis includes participants remaining in the study at the indicated time point who also completed this assessment. Due to scheduling conflicts with families, not all participants were able to complete every study assessment. If a study visit could not be scheduled within the visit window then that visit was skipped and the next assessment was scheduled.

Outcome measures

Outcome measures
Measure	Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive the Group-ESI intervention for 9 months	Individual-ESI Plus Group-ESI n=17 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Communication and Symbolic Behavior Scales (CSBS) Score Baseline (12 months of age, prior to treatment)	80.44 score on a scale Standard Deviation 3.83	78.71 score on a scale Standard Deviation 5.97
Communication and Symbolic Behavior Scales (CSBS) Score 15 Months of Age	79.20 score on a scale Standard Deviation 10.88	80.36 score on a scale Standard Deviation 8.64
Communication and Symbolic Behavior Scales (CSBS) Score 21 Months of Age	85.60 score on a scale Standard Deviation 12.85	91.20 score on a scale Standard Deviation 11.81
Communication and Symbolic Behavior Scales (CSBS) Score 24 Months of Age	85.79 score on a scale Standard Deviation 17.48	89.92 score on a scale Standard Deviation 15.10

PRIMARY outcome

Timeframe: 15, 24, and 36 months of age

Autism symptoms were measured with the Autism Diagnostic Observation Schedule, Second Edition which is the best gold standard diagnostic measure of ASD. The revised algorithms forming Social-Affect and Restricted Repetitive Behavior ratings of autism symptoms were used. A total of score of 1 to 3 indicates no signs of autism, a score of 4 or 5 suggests that the child may have autism spectrum disorder, while a score of 6 to 10 is indicative of autism.

Outcome measures

Outcome measures
Measure	Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive the Group-ESI intervention for 9 months	Individual-ESI Plus Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Autism Diagnostic Observation Schedule (ADOS) Score 15 Months of Age	3.88 score on a scale Standard Deviation 2.03	3.75 score on a scale Standard Deviation 2.24
Autism Diagnostic Observation Schedule (ADOS) Score 24 Months of Age	5.14 score on a scale Standard Deviation 2.69	5.23 score on a scale Standard Deviation 3.00
Autism Diagnostic Observation Schedule (ADOS) Score 36 Months of Age	3.79 score on a scale Standard Deviation 2.12	4.53 score on a scale Standard Deviation 3.56

PRIMARY outcome

Timeframe: Baseline (12 months of age, prior to treatment), 24, and 36 months of age

The Mullen Scales of Early Learning (MSEL) measures developmental level with standardized scores in five domains: Visual Reception, Receptive Language, Expressive Language, Fine Motor Skills, and Gross Motor Skills. Domain scores are represented in T-scores, with a mean of 50 and standard deviation of 10. A Verbal Composite score is calculated by averaging T-scores of the domains for Receptive and Expressive Language. A Nonverbal Composite score is calculated by averaging T-scores for Visual Reception and Fine Motor domains. Scores below 50 indicate below average performance.

Outcome measures

Outcome measures
Measure	Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive the Group-ESI intervention for 9 months	Individual-ESI Plus Group-ESI n=17 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Mullen Scales of Early Learning (MSEL) Score Baseline (12 months of age, prior to treatment) - Verbal Composite Score	37.50 t-score Standard Deviation 8.43	37.03 t-score Standard Deviation 6.16
Mullen Scales of Early Learning (MSEL) Score 24 Months of Age - Verbal Composite Score	40.86 t-score Standard Deviation 14.20	45.19 t-score Standard Deviation 11.02
Mullen Scales of Early Learning (MSEL) Score 36 Months of Age - Verbal Composite Score	44.43 t-score Standard Deviation 12.94	43.63 t-score Standard Deviation 11.73
Mullen Scales of Early Learning (MSEL) Score Baseline (12 months of age, prior to treatment) - Nonverbal Composite Score	54.13 t-score Standard Deviation 5.66	52.79 t-score Standard Deviation 8.78
Mullen Scales of Early Learning (MSEL) Score 24 Months of Age - Nonverbal Composite Score	52.36 t-score Standard Deviation 6.34	47.19 t-score Standard Deviation 10.70
Mullen Scales of Early Learning (MSEL) Score 36 Months of Age - Nonverbal Composite Score	48.93 t-score Standard Deviation 13.73	47.13 t-score Standard Deviation 10.17

PRIMARY outcome

Timeframe: Baseline (12 months of age, prior to treatment), 24, and 36 months of age

The Vineland II assesses adaptive behavior by providing a standard score in four domains of Communication, Daily Living Skills, Socialization, and Motor Skills. Domain scores and the Adaptive Behavior Composite score are standardized with a mean of 100 with a standard deviation of 15. Scores less than 100 indicate below average performance.

Outcome measures

Outcome measures
Measure	Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive the Group-ESI intervention for 9 months	Individual-ESI Plus Group-ESI n=16 Participants Infants 12 months of age at risk of developing ASD who were randomized to receive Individual-ESI intervention in addition to the Group-ESI intervention for 9 months.
Vineland Adaptive Behavior Scales, Second Edition (Vineland II) Score Baseline (12 months of age, prior to treatment)	87.69 score on a scale Standard Deviation 8.51	87.19 score on a scale Standard Deviation 7.26
Vineland Adaptive Behavior Scales, Second Edition (Vineland II) Score 24 Months of Age	90.64 score on a scale Standard Deviation 8.91	92.14 score on a scale Standard Deviation 7.08
Vineland Adaptive Behavior Scales, Second Edition (Vineland II) Score 36 Months of Age	90.13 score on a scale Standard Deviation 10.75	84.57 score on a scale Standard Deviation 6.21

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline (12 months of age, prior to treatment), 15, 21, 24, and 36 months of age

Population: Expressive language phase was to be coded based on the CSBS, however, this coding requires extensive time and effort by a trained staff member. As the study terminated early and research priorities shifted with the Coronavirus disease 2019 (COVID-19) pandemic, staffing was no longer available to review the video recordings specifically to code language phase. Data analyses for this study focused on addressing the primary aims of the study and data for this outcome were never created.

The child's expressive language phase will be determined at each assessment based on the benchmarks delineated by Tager-Flusberg and colleagues and recommended for evaluating the efficacy and comparing outcomes across NIH-funded intervention studies. Based on multiple sources of information about language benchmarks, the language will be categorized as: Preverbal Communication, First Words, Word Combinations, or Sentences, to provide a clinically meaningful measure of language outcome.

Outcome measures

Outcome data not reported

Adverse Events

Group-ESI

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Individual-ESI Plus Group-ESI

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Nathan Call, PhD

Emory University

Phone: 404-785-9400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place