Trial Outcomes & Findings for Guanfacine for the Treatment of Hyperactivity in Pervasive Developmental Disorder (NCT NCT01238575)

NCT ID: NCT01238575

Last Updated: 2020-03-31

Results Overview

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. The 16-item Hyperactivity subscale covers over-activity (7 items), impulsiveness (2 items), inattention (3 items) and noncompliance (4 items). It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. The range of scores is 0 to 48.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

62 participants

Primary outcome timeframe

Week 8

Results posted on

2020-03-31

Participant Flow

81 total patients screened. 13 ineligible. 6 Declined

Participant milestones

Participant milestones
Measure
Extended-release Guanfacine
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Inactive Placebo
placebo: Administered for up to 8 weeks.
Overall Study
STARTED
30
32
Overall Study
COMPLETED
26
28
Overall Study
NOT COMPLETED
4
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Extended-release Guanfacine
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Inactive Placebo
placebo: Administered for up to 8 weeks.
Overall Study
Lack of Efficacy
2
4
Overall Study
Adverse Event
2
0

Baseline Characteristics

Guanfacine for the Treatment of Hyperactivity in Pervasive Developmental Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Total
n=62 Participants
Total of all reporting groups
Age, Continuous
8.44 years
STANDARD_DEVIATION 2.28 • n=5 Participants
8.39 years
STANDARD_DEVIATION 2.23 • n=7 Participants
8.4 years
STANDARD_DEVIATION 2.25 • n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
5 Participants
n=7 Participants
9 Participants
n=5 Participants
Sex: Female, Male
Male
26 Participants
n=5 Participants
27 Participants
n=7 Participants
53 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
Race (NIH/OMB)
Asian
4 Participants
n=5 Participants
1 Participants
n=7 Participants
5 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
7 Participants
n=5 Participants
4 Participants
n=7 Participants
11 Participants
n=5 Participants
Race (NIH/OMB)
White
17 Participants
n=5 Participants
23 Participants
n=7 Participants
40 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
3 Participants
n=7 Participants
4 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

PRIMARY outcome

Timeframe: Week 8

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. The 16-item Hyperactivity subscale covers over-activity (7 items), impulsiveness (2 items), inattention (3 items) and noncompliance (4 items). It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. The range of scores is 0 to 48.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Hyperactivity Subscale
29.7 units on a scale
Interval 25.82 to 33.53
19.3 units on a scale
Interval 15.33 to 23.33

SECONDARY outcome

Timeframe: Week 8

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. The total score can range from 0 to 54, with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Total
38.0 units on a scale
Interval 34.44 to 41.63
25.2 units on a scale
Interval 21.44 to 29.03

SECONDARY outcome

Timeframe: 8 weeks

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. It is a 58 item checklist which takes about 10 - 15 minutes to complete. There are five subscales: a) Irritability and Agitation b) Lethargy and Social Withdrawal c) Stereotypic Behavior d) Hyperactivity and Noncompliance and e) Inappropriate Speech. The higher the number of items (score), the greater the amount of symptoms. Scores can range from 0 to 45.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Irritability Subscale
16.1 units on a scale
Interval 12.68 to 19.54
13.5 units on a scale
Interval 10.01 to 17.06

SECONDARY outcome

Timeframe: 8 weeks

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 48.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Social Withdrawal Subscale
8.6 units on a scale
Interval 6.1 to 11.02
9.8 units on a scale
Interval 7.26 to 12.27

SECONDARY outcome

Timeframe: 8 weeks

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 21.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Sterotypy Subscale
5.9 units on a scale
Interval 4.37 to 7.49
3.6 units on a scale
Interval 2.03 to 5.26

SECONDARY outcome

Timeframe: 8 weeks

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 12.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Inappropriate Speech Subscale
5.99 units on a scale
Interval 4.5 to 6.97
4.2 units on a scale
Interval 3.24 to 5.26

SECONDARY outcome

Timeframe: 8 weeks

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. This subscale can range from 0 to 27 for scoring, with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Inattention Subscale
19.5 units on a scale
Interval 17.52 to 21.56
14.7 units on a scale
Interval 12.55 to 16.78

SECONDARY outcome

Timeframe: 8 weeks

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. This subscale can range from 0 to 27 for scoring,with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Hyperactivity Subscale
18.7 units on a scale
Interval 16.66 to 20.69
10.6 units on a scale
Interval 8.5 to 12.75

SECONDARY outcome

Timeframe: Baseline

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. The 16-item Hyperactivity subscale covers over-activity (7 items), impulsiveness (2 items), inattention (3 items) and noncompliance (4 items). It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. The range of scores is 0 to 48.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Hyperactivity Subscale
34.25 units on a scale
Interval 31.74 to 36.76
34.4 units on a scale
Interval 32.4 to 36.4

SECONDARY outcome

Timeframe: Baseline

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. Scores for this subscale can range from 0 to 45.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Irritability Subscale
18.06 units on a scale
Interval 14.54 to 21.58
20.3 units on a scale
Interval 16.79 to 23.81

SECONDARY outcome

Timeframe: Baseline

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 48.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Social Withdrawal Subscale
12.06 units on a scale
Interval 8.71 to 15.41
13.6 units on a scale
Interval 10.08 to 17.12

SECONDARY outcome

Timeframe: Baseline

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 21.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Sterotypy Subscale
9.31 units on a scale
Interval 7.31 to 11.32
8.53 units on a scale
Interval 6.41 to 10.66

SECONDARY outcome

Timeframe: Baseline

The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. The full ABC is a 58-item parent-rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. It has been used as a primary outcome measure in several trials of children with developmental disabilities. The interpretation of the tool and its sub-scales is that a greater number of items, indicates greater severity. This subscale's scores can range from 0 to 12.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Aberrant Behavior Checklist Inappropriate Speech Subscale
6.84 units on a scale
Interval 5.63 to 8.06
6.33 units on a scale
Interval 5.02 to 7.65

SECONDARY outcome

Timeframe: Baseline

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. This subscale can range from 0 to 27 for scoring, with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Inattention Subscale
20.41 units on a scale
Interval 18.75 to 22.06
20.53 units on a scale
Interval 19.17 to 21.9

SECONDARY outcome

Timeframe: Baseline

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. This subscale can range from 0 to 27 for scoring, with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Hyperactivity Subscale
19.5 units on a scale
Interval 17.71 to 21.29
19.00 units on a scale
Interval 17.38 to 20.62

SECONDARY outcome

Timeframe: Baseline

The ADHD Rating Scale (ADHD-RS) is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder with established reliability, validity and sensitivity to change. The ADHD Rating Scale-IV is completed independently by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. Results from this rating scale alone should not be used to make a diagnosis. The total score can range from 0 to 54, with a higher score indicating greater severity.

Outcome measures

Outcome measures
Measure
Inactive Placebo
n=32 Participants
placebo: Administered for up to 8 weeks.
Extended-release Guanfacine
n=30 Participants
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
ADHD Rating Scale - Total
39.91 units on a scale
Interval 37.5 to 42.32
39.53 units on a scale
Interval 37.33 to 41.73

Adverse Events

Extended-release Guanfacine

Serious events: 1 serious events
Other events: 30 other events
Deaths: 0 deaths

Inactive Placebo

Serious events: 0 serious events
Other events: 25 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Extended-release Guanfacine
n=30 participants at risk
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Inactive Placebo
n=32 participants at risk
placebo: Administered for up to 8 weeks.
Psychiatric disorders
Aggressive
3.3%
1/30
0.00%
0/32

Other adverse events

Other adverse events
Measure
Extended-release Guanfacine
n=30 participants at risk
extended-release guanfacine: 1 mg tablets; flexible dosing up to 4 mg/day for up to 16 weeks
Inactive Placebo
n=32 participants at risk
placebo: Administered for up to 8 weeks.
Nervous system disorders
Drowsiness
86.7%
26/30
9.4%
3/32
General disorders
Fatigue
63.3%
19/30
9.4%
3/32
General disorders
Decreased Appetite
43.3%
13/30
6.2%
2/32
General disorders
Emotional/tearful
40.0%
12/30
9.4%
3/32
Gastrointestinal disorders
Dry Mouth
40.0%
12/30
3.1%
1/32
General disorders
Irritability
36.7%
11/30
9.4%
3/32
Psychiatric disorders
Anxiety
30.0%
9/30
3.1%
1/32
Nervous system disorders
Headache
30.0%
9/30
18.8%
6/32
General disorders
Increased Energy
30.0%
9/30
18.8%
6/32
Psychiatric disorders
Mid sleep awakening
30.0%
9/30
6.2%
2/32
Gastrointestinal disorders
Stomachache
26.7%
8/30
12.5%
4/32
Gastrointestinal disorders
Constipation
23.3%
7/30
6.2%
2/32
Psychiatric disorders
Increased Repetitive Behavior
16.7%
5/30
9.4%
3/32
Psychiatric disorders
Aggression
16.7%
5/30
6.2%
2/32
Psychiatric disorders
Depressed Mood
13.3%
4/30
3.1%
1/32
Respiratory, thoracic and mediastinal disorders
Cough/Congestion
13.3%
4/30
12.5%
4/32
Psychiatric disorders
Self-injury
10.0%
3/30
0.00%
0/32
Gastrointestinal disorders
Nausea
10.0%
3/30
6.2%
2/32
Psychiatric disorders
Trouble Falling Asleep
10.0%
3/30
6.2%
2/32
Nervous system disorders
Dizziness
10.0%
3/30
6.2%
2/32
Psychiatric disorders
Silly
10.0%
3/30
15.6%
5/32
General disorders
Weakness
10.0%
3/30
3.1%
1/32
Gastrointestinal disorders
Diarrhea
10.0%
3/30
0.00%
0/32
Gastrointestinal disorders
Vomiting
10.0%
3/30
9.4%
3/32
Gastrointestinal disorders
Increased Appetite
6.7%
2/30
6.2%
2/32
Psychiatric disorders
Excessive Talking
6.7%
2/30
28.1%
9/32
Eye disorders
Blurred Vision
6.7%
2/30
0.00%
0/32
Skin and subcutaneous tissue disorders
Skin rash/Eczema
6.7%
2/30
12.5%
4/32
Psychiatric disorders
Nightmares
6.7%
2/30
0.00%
0/32
Renal and urinary disorders
Enuresis
6.7%
2/30
6.2%
2/32
Nervous system disorders
Motor Tics
3.3%
1/30
6.2%
2/32
Skin and subcutaneous tissue disorders
Skin Picking
3.3%
1/30
9.4%
3/32
General disorders
Fever
3.3%
1/30
9.4%
3/32

Additional Information

Lawrence Scahill, MSN, PhD

Emory University

Results disclosure agreements

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