Trial Outcomes & Findings for A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder (NCT NCT01970345)
NCT ID: NCT01970345
Last Updated: 2024-02-08
Results Overview
Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes.
TERMINATED
PHASE2
3 participants
Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
2024-02-08
Participant Flow
Decision made to terminate due to difficulty in procurement of adequate supply of treatment drug. Decision made on April 2023 to terminate study. However last participant visit was Sept 2017.
Participant milestones
| Measure |
IGF-1 Then Placebo
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
Placebo: Matching Placebo
|
Placebo Then IGF-1
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
Placebo: Matching Placebo
|
|---|---|---|
|
First Treatment Phase 12 Weeks
STARTED
|
1
|
2
|
|
First Treatment Phase 12 Weeks
COMPLETED
|
0
|
1
|
|
First Treatment Phase 12 Weeks
NOT COMPLETED
|
1
|
1
|
|
Crossover Washout Period 4 Weeks
STARTED
|
0
|
1
|
|
Crossover Washout Period 4 Weeks
COMPLETED
|
0
|
1
|
|
Crossover Washout Period 4 Weeks
NOT COMPLETED
|
0
|
0
|
|
Second Treatment Phase 12 Weeks
STARTED
|
0
|
1
|
|
Second Treatment Phase 12 Weeks
COMPLETED
|
0
|
0
|
|
Second Treatment Phase 12 Weeks
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
IGF-1 Then Placebo
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
Placebo: Matching Placebo
|
Placebo Then IGF-1
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
Placebo: Matching Placebo
|
|---|---|---|
|
First Treatment Phase 12 Weeks
Withdrawal by Subject
|
1
|
1
|
|
Second Treatment Phase 12 Weeks
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Baseline characteristics by cohort
| Measure |
Children With Autism Spectrum Disorders
n=3 Participants
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.
|
|---|---|
|
Age, Categorical
<=18 years
|
3 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
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8Population: Results for participants who were still on study.
Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Aberrant Behavior Checklist - Social Withdrawal Subscale
Week 4, phase 1
|
20 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
20 score on a scale
Standard Deviation 18.4
|
|
Aberrant Behavior Checklist - Social Withdrawal Subscale
Week 12, Phase 1
|
—
|
4 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Aberrant Behavior Checklist - Social Withdrawal Subscale
Week 4, phase 2
|
3 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Aberrant Behavior Checklist - Social Withdrawal Subscale
Week 8, Phase 2
|
2 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Week 4 of Phase 1Population: Data provided for participants who completed week 4
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as "0" (does not show the behavior), "1" (rarely shows the behavior), "2" (sometimes/partially shows the behavior), "3" (usually/typically shows the behavior), or "?" (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Sensory
|
21 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
16 score on a scale
Standard Deviation 14.1
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Ritual
|
9 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
13 score on a scale
Standard Deviation 14.1
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pragmatic
|
20 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
14 score on a scale
Standard Deviation 0
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Semantic Pragmatic
|
19 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
0.5 score on a scale
Standard Deviation 0.7
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Arouse
|
23 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
20.5 score on a scale
Standard Deviation 6.4
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Specific Fears
|
13 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
25 score on a scale
Standard Deviation 1.4
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Aggressiveness
|
7 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
30 score on a scale
Standard Deviation 25.5
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Approach
|
75 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
31 score on a scale
Standard Deviation 15.6
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Expressive
|
36 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
18.5 score on a scale
Standard Deviation 20.5
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Learning Memory
|
0 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
8.5 score on a scale
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: Week 12 of Phase 1Population: Data provided for participants who completed week 12
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as "0" (does not show the behavior), "1" (rarely shows the behavior), "2" (sometimes/partially shows the behavior), "3" (usually/typically shows the behavior), or "?" (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Outcome measures
| Measure |
IGF-1
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=1 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Sensory
|
—
|
9 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Ritual
|
—
|
4 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pragmatic
|
—
|
16 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Semantic Pragmatic
|
—
|
8 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Arouse
|
—
|
12 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Specific Fears
|
—
|
22 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Aggressiveness
|
—
|
18 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Approach
|
—
|
59 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Expressive
|
—
|
27 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Learning Memory
|
—
|
15 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
SECONDARY outcome
Timeframe: Week 4 of Phase 2Population: Data provided for participants who completed week 4
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as "0" (does not show the behavior), "1" (rarely shows the behavior), "2" (sometimes/partially shows the behavior), "3" (usually/typically shows the behavior), or "?" (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Sensory
|
11 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Ritual
|
2 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pragmatic
|
13 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Semantic Pragmatic
|
5 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Arouse
|
16 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Specific Fears
|
10 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Aggressiveness
|
10 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Approach
|
52 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Expressive
|
36 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Learning Memory
|
14 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Week 8 of Phase 2Population: Data provided for participants who completed week 8. Data not provided by participant for one subscale.
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as "0" (does not show the behavior), "1" (rarely shows the behavior), "2" (sometimes/partially shows the behavior), "3" (usually/typically shows the behavior), or "?" (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Sensory
|
5 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Ritual
|
1 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Semantic Pragmatic
|
9 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Arouse
|
10 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Specific Fears
|
7 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Aggressiveness
|
6 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Approach
|
60 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Expressive
|
43 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Learning Memory
|
14 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8Population: Data provided for participants who were on study
Repetitive Behavior: Repetitive Behavior Scale (RBS) - Includes 43 items scored 0 (behavior does not occur), 1 (behavior occurs and is a mild problem), 2 (behavior occurs and is a moderate problem), 3 (behavior occurs and is a severe problem). Full scale from 0-129, with higher scores indicating worse outcomes.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Repetitive Behavior
Week 4 of Phase 1
|
31 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
44 score on a scale
Standard Deviation 41.0
|
|
Repetitive Behavior
Week 12 of Phase 1
|
—
|
11 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Repetitive Behavior
Week 4 of Phase 2
|
11 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Repetitive Behavior
Week 8 of Phase 2
|
8 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8Population: Data provided for participants who were on study
Caregiver Strain Index - 21 question tool, with response 1 (not al all) - 5 (very much). Total score on scale from 21-105. Higher score indicates poorer health outcomes.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Caregiver Strain
Week 4, Phase 1
|
71 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
87 score on a scale
Standard Deviation 9.9
|
|
Caregiver Strain
Week 12, Phase 1
|
—
|
73 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
|
Caregiver Strain
Week 4, Phase 2
|
54 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
|
Caregiver Strain
Week 8, Phase 2
|
54 score on a scale
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8Population: data not collected
Clinician-rated interview designed to evaluate repetitive behavior in children with pervasive developmental disorders (PDDs). It is a modification of the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS), developed to assess typically-developing children with obsessive compulsive behavior. Because of language limitations in children with PDDs the CY-BOCS-PDD only includes the five compulsion items: Time Spent, Interference, Distress, Resistance of repetitive behavior, and Control of repetitive behavior. Each item is rated from 0 (none) through 4 (extreme), and scores can range from 0 to 20, with higher scores reflecting more severe symptoms. Usually a score 8 is considered clinically significant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8Population: Data provided for participants who were on study
The CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating. Full scale is 1 (normal, not at all ill) to 7 (extremely ill).
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
The Clinical Global Impression - Severity Scale (CGI-S)
Week 4, Phase 1
|
5 score on a scale
Standard Deviation NA
N=1 so no measure of dispersion
|
5 score on a scale
Standard Deviation 1.4
|
|
The Clinical Global Impression - Severity Scale (CGI-S)
Week 12, Phase 1
|
—
|
4 score on a scale
Standard Deviation NA
N=1 so no measure of dispersion
|
|
The Clinical Global Impression - Severity Scale (CGI-S)
Week 4, Phase 2
|
—
|
4 score on a scale
Standard Deviation NA
N=1 so no measure of dispersion
|
|
The Clinical Global Impression - Severity Scale (CGI-S)
Week 8, Phase 2
|
4 score on a scale
Standard Deviation NA
N=1 so no measure of dispersion
|
—
|
SECONDARY outcome
Timeframe: Baseline and Phase 1 Week 12Population: Data collected at baseline and Week 12 and only for participants on study.
The Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form) yields an overall standard score (Adaptive Behavior Composite, ABC) and age standard scores in four domains. ABC scores have a mean of 100 and a standard deviation of 15 (range = 20 to 160). Higher scores suggest a higher level of adaptive functioning.
Outcome measures
| Measure |
IGF-1
n=1 Participants
IGF-1: Dose titration initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 Participants
Placebo
Placebo/saline: Placebo
|
|---|---|---|
|
Vineland Adaptive Behavior Composite Score
Baseline
|
55 T-score
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
51 T-score
Standard Deviation 8.5
|
|
Vineland Adaptive Behavior Composite Score
Week 12, Phase 1
|
—
|
57 T-score
Standard Deviation NA
N=1 therefore there is no measure of dispersion
|
Adverse Events
IGF-1
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
IGF-1
n=2 participants at risk
Dose titration will be initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established.
|
Placebo
n=2 participants at risk
Placebo/saline: Placebo
|
|---|---|---|
|
Metabolism and nutrition disorders
Increased appetite
|
50.0%
1/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
General disorders
Sleep disturbance
|
100.0%
2/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
Psychiatric disorders
Irritability
|
50.0%
1/2 • 36 weeks
|
100.0%
2/2 • 36 weeks
|
|
Blood and lymphatic system disorders
Bruising easily
|
0.00%
0/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
General disorders
Increased energy
|
50.0%
1/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
Psychiatric disorders
Aggression towards others
|
0.00%
0/2 • 36 weeks
|
100.0%
2/2 • 36 weeks
|
|
Psychiatric disorders
oppositional behavior
|
0.00%
0/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
Eye disorders
Eye blinking
|
0.00%
0/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
General disorders
Canker sore
|
0.00%
0/2 • 36 weeks
|
50.0%
1/2 • 36 weeks
|
|
Renal and urinary disorders
Enuresis
|
0.00%
0/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
Gastrointestinal disorders
Vomiting/diarrhea
|
100.0%
2/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
50.0%
1/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
General disorders
Congestion/allergies
|
100.0%
2/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
General disorders
Fever/congestion
|
100.0%
2/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
Psychiatric disorders
Tic- head shake
|
0.00%
0/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
|
Psychiatric disorders
Anxiety
|
50.0%
1/2 • 36 weeks
|
0.00%
0/2 • 36 weeks
|
Additional Information
Dr. Alexander Kolevzon
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place