Trial Outcomes & Findings for A Pilot Study of Daytrana TM in Children With Autism Co-Morbid for Attention Deficit Hyperactivity Disorder (ADHD) Symptoms (NCT NCT00541346)

NCT ID: NCT00541346

Last Updated: 2014-02-06

Results Overview

This instrument is a parent rating scale used to assess the frequency of ADHD symptoms based on DSM-IV criteria. Raw scores range from 0-54. Higher scores indicate a higher frequency of ADHD symptoms. Raw scores were used in the analyses described below.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

16 participants

Primary outcome timeframe

Baseline, 8 weeks

Results posted on

2014-02-06

Participant Flow

16 enrolled Study Start Date: September, 2007 Study Completion Date:May, 2009 Seen at OU Physician's Child Study Center.

Participant milestones

Participant milestones
Measure
Methylphenidate Transdermal System
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Baseline
STARTED
16
Baseline
COMPLETED
16
Baseline
NOT COMPLETED
0
Follow-up
STARTED
16
Follow-up
COMPLETED
15
Follow-up
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Methylphenidate Transdermal System
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Follow-up
Adverse Event
1

Baseline Characteristics

A Pilot Study of Daytrana TM in Children With Autism Co-Morbid for Attention Deficit Hyperactivity Disorder (ADHD) Symptoms

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Age, Categorical
<=18 years
16 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
7.83 years
STANDARD_DEVIATION 1.23 • n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
Sex: Female, Male
Male
14 Participants
n=5 Participants
Region of Enrollment
United States
16 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

This instrument is a parent rating scale used to assess the frequency of ADHD symptoms based on DSM-IV criteria. Raw scores range from 0-54. Higher scores indicate a higher frequency of ADHD symptoms. Raw scores were used in the analyses described below.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Attention Deficit Hyperactivity Disorder Rating Scale - IV (ADHD-RS-IV) Total Score From Baseline to 8-week Follow-up Visit
Baseline: ADHD-RS-IV Total Score
39.9 units on a scale
Standard Deviation 7.7
Change in Attention Deficit Hyperactivity Disorder Rating Scale - IV (ADHD-RS-IV) Total Score From Baseline to 8-week Follow-up Visit
Follow-Up: ADHD-RS-IV Total Score
13.8 units on a scale
Standard Deviation 9.0

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week assessment was used to handle missing data for a single drop-out participant who left the study after the 2nd follow-up visit.

The ABC is a behavior rating scale administered by the clinician which is designed to measure behavior changes brought about by drug treatment effects. 16 of these items comprise the hyperactivity, noncompliance factor. Each item is scored on a 3 point scale where 0 indicates the behavior is not a problem and 3 indicates the behavior problem is severe in degree. The minimum score on this factor is 0 (no behavior problems) while the maximum score is 48 ( severe behavior problems).

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Aberrant Behavior Checklist (ABC) Hyperactivity Scores From Baseline to 8-week Follow-up Visit
Baseline: ABC HYPERACTIVITY
34.1 units on a scale
Standard Deviation 8.3
Change in Aberrant Behavior Checklist (ABC) Hyperactivity Scores From Baseline to 8-week Follow-up Visit
Follow-up: ABC HYPERACTIVITY
11.3 units on a scale
Standard Deviation 8.4

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The ABC is a behavior rating scale administered by the clinician which is designed to measure behavior changes brought about by drug treatment effects. 15 of these items comprise the irritability/agitation/crying factor. Each item is scored on a 3 point scale where 0 indicates the behavior is not a problem and 3 indicates the behavior problem is severe in degree. The minimum score on this factor is 0 (no behavior problems) while the maximum score is 45 ( severe behavior problems).

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Aberrant Behavior Checklist (ABC) Irritability Scores From Baseline to 8-week Follow-up Visit
Baseline: ABC Irritability
19.8 units on a scale
Standard Deviation 9.6
Change in Aberrant Behavior Checklist (ABC) Irritability Scores From Baseline to 8-week Follow-up Visit
Follow-up: ABC Irritability
9.1 units on a scale
Standard Deviation 6.5

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The ABC is a behavior rating scale administered by the clinician which is designed to measure behavior changes brought about by drug treatment effects. 16 of these items comprise the lethargy/social withdrawal factor. Each item is scored on a 3 point scale where 0 indicates the behavior is not a problem and 3 indicates the behavior problem is severe in degree. The minimum score on this factor is 0 (no behavior problems) while the maximum score is 48 ( severe behavior problems).

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Aberrant Behavior Checklist (ABC) Lethargy Scores From Baseline to 8-week Follow-up Visit
Baseline: ABC Lethargy
11.3 units on a scale
Standard Deviation 4.9
Change in Aberrant Behavior Checklist (ABC) Lethargy Scores From Baseline to 8-week Follow-up Visit
Follow-up: ABC Lethargy
4.5 units on a scale
Standard Deviation 3.6

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The ABC is a behavior rating scale administered by the clinician which is designed to measure behavior changes brought about by drug treatment effects. 7 of these items comprise the stereotypic behavior factor. Each item is scored on a 3 point scale where 0 indicates the behavior is not a problem and 3 indicates the behavior problem is severe in degree. The minimum score on this factor is 0 (no behavior problems) while the maximum score is 21 ( severe behavior problems).

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Aberrant Behavior Checklist (ABC) Stereotypy Scores From Baseline to 8-week Follow-up Visit
Baseline: ABC Stereotypy
5.6 units on a scale
Standard Deviation 5.4
Change in Aberrant Behavior Checklist (ABC) Stereotypy Scores From Baseline to 8-week Follow-up Visit
Follow-up: ABC Stereotypy
1.3 units on a scale
Standard Deviation 2.3

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The ABC is a behavior rating scale administered by the clinician which is designed to measure behavior changes brought about by drug treatment effects. 4 of these items comprise the lethargy/social withdrawal factor. Each item is scored on a 3 point scale where 0 indicates the behavior is not a problem and 3 indicates the behavior problem is severe in degree. The minimum score on this factor is 0 (no behavior problems) while the maximum is 12 ( severe behavior problems).

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Aberrant Behavior Checklist (ABC) Inappropriate Speech Scores From Baseline to 8-week Follow-up Visit
Baseline: ABC Inappropriate Speech
5.5 units on a scale
Standard Deviation 2.9
Change in Aberrant Behavior Checklist (ABC) Inappropriate Speech Scores From Baseline to 8-week Follow-up Visit
Follow-up: ABC Inappropriate Speech
2.1 units on a scale
Standard Deviation 2.3

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The LPS was developed to capture treatment-related improvements in adaptive functioning including quality of life, social development, and emotion regulation. There are 24 items scored using a 4-point Likert frequency scale (0=Never or Seldom, 1=Sometimes, 2=Often, 3=Very Often). A summed scale score (possible range of 0 to 72) was used in the analyses described below. Higher scores indicate more adaptive functioning.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Lifetime Participation Scale (LPS) Total Scores From Baseline to 8-week Follow-up Visit
Baseline: LPS Total
26.6 units on a scale
Standard Deviation 9.0
Change in Lifetime Participation Scale (LPS) Total Scores From Baseline to 8-week Follow-up Visit
Follow-up: LPS Total
39.9 units on a scale
Standard Deviation 12.2

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week follow-up assessment was used to handle missing data for a single drop-out participant who left the study after a 2nd follow-up visit.

The Family Assessment measure is a self-report instrument that provides quantitative indices of family strengths and weaknesses. Each items is rated 0 (strongly agree) to 3 (strongly disagree). The General scale produces seven subscales: task accomplishment, role performance, communication, affective expression, involvement, control and values and norms. The minimum score for each subscale is 0 while the maximum score is 15. Higher raw scores indicate a higher number of family problems reported. A total summed score of all scale scores was used in the analyses described below. The possible range of this total score was 0 to 105. Like the subscales, higher values for this total summed score indicate a higher number of family problems reported.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Family III General Scale Summed Score From Baseline to 8-week Follow-up Visit
Baseline: FAM-III Overall
55.7 units on a scale
Standard Deviation 4.1
Change in Family III General Scale Summed Score From Baseline to 8-week Follow-up Visit
Follow-up: FAM-III Overall
53.5 units on a scale
Standard Deviation 5.2

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week assessment was used to handle missing data for a single drop-out participant who left the study after the 2nd follow-up visit.

The PEDI Caregiver Assistance measures rate the child's function in three domains: Self-care, Mobility, and Social Function. Items are scored 0 (total, where the child is completely dependent on assistance) to 5 (independent, where no assistance is given or required). Scale scores represent summed item scores within each domain. The Self-Care scale score ranges from 0 to 40. A higher score indicates a higher degree of independence in the self-care area.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Pediatric Evaluation Disability Inventory (PEDI) Caregiver Assistance: Self-Care From Baseline to 8-week Follow-up Visit
Baseline: PEDI Self-Care
28.8 units on a scale
Standard Deviation 7.0
Change in Pediatric Evaluation Disability Inventory (PEDI) Caregiver Assistance: Self-Care From Baseline to 8-week Follow-up Visit
Follow-up: PEDI Self-Care
32.5 units on a scale
Standard Deviation 6.5

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week assessment was used to handle missing data for a single drop-out participant who left the study after the 2nd follow-up visit.

The PEDI Caregiver Assistance measures rate the child's function in three domains: Self-care, Mobility, and Social Function. Items are scored 0 (total, where the child is completely dependent on assistance) to 5 (independent, where no assistance is given or required). Scale scores represent summed item scores within each domain. The Social-Function scale score ranges from 0 to 25. A higher score indicates a higher degree of independence in the Social-Function area.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=16 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Pediatric Evaluation Disability Inventory (PEDI) Social Function From Baseline to 8-week Follow-up Visit
Baseline: PEDI Social Function
9.3 units on a scale
Standard Deviation 5.0
Change in Pediatric Evaluation Disability Inventory (PEDI) Social Function From Baseline to 8-week Follow-up Visit
Follow-up: PEDI Social Function
14.9 units on a scale
Standard Deviation 5.8

SECONDARY outcome

Timeframe: Baseline, 8 weeks

Population: Intention to Treat (ITT). Bayesian posterior-predictive imputation of the 8th-week assessment was used to handle missing data for a single drop-out participant who left the study after the 2nd follow-up visit.

This instrument is a teacher rating scale used to assess the frequency of ADHD symptoms based on DSM-IV criteria. Raw scores range from 0-54. Higher scores indicate a higher frequency of ADHD symptoms. Raw scores were used in the analyses described below.

Outcome measures

Outcome measures
Measure
Methylphenidate Transdermal System
n=12 Participants
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Change in Attention Deficit Hyperactivity Disorder Rating Scale IV: Teacher Assessment Total Scores From Baseline to 8-week Follow-up Visit
Baseline: ADHDRS Teacher Total
32.6 units on a scale
Standard Deviation 8.9
Change in Attention Deficit Hyperactivity Disorder Rating Scale IV: Teacher Assessment Total Scores From Baseline to 8-week Follow-up Visit
Follow-up: ADHDRS Teacher Total
28.0 units on a scale
Standard Deviation 14.4

Adverse Events

Methylphenidate Transdermal System

Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Methylphenidate Transdermal System
n=16 participants at risk
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Nervous system disorders
tic
6.2%
1/16 • 8 weeks

Other adverse events

Other adverse events
Measure
Methylphenidate Transdermal System
n=16 participants at risk
10mg for one week, with weekly stepwise increases to 15mg, 20mg, and 30mg for additional 7 weeks, if symptom reports remained elevated. Titration decreased one stepwise dosage when significant side-effects were present.
Nervous system disorders
Headache
18.8%
3/16 • 8 weeks
Infections and infestations
fever
43.8%
7/16 • 8 weeks
Psychiatric disorders
irritability
25.0%
4/16 • 8 weeks
Metabolism and nutrition disorders
appetite, loss of
18.8%
3/16 • 8 weeks
Gastrointestinal disorders
Abdominal pain
6.2%
1/16 • 8 weeks
General disorders
nose, inflammation of
6.2%
1/16 • 8 weeks
Psychiatric disorders
sleeping, difficulty
6.2%
1/16 • 8 weeks
Gastrointestinal disorders
vomiting
12.5%
2/16 • 8 weeks
Gastrointestinal disorders
diarrhea
12.5%
2/16 • 8 weeks
Gastrointestinal disorders
increased salivation
6.2%
1/16 • 8 weeks
Gastrointestinal disorders
blood in stool
6.2%
1/16 • 8 weeks
Gastrointestinal disorders
constipation
6.2%
1/16 • 8 weeks
Eye disorders
eyes bloodshot
6.2%
1/16 • 8 weeks
Respiratory, thoracic and mediastinal disorders
Rhinitis Allergic
6.2%
1/16 • 8 weeks
Respiratory, thoracic and mediastinal disorders
sore throat
6.2%
1/16 • 8 weeks
Respiratory, thoracic and mediastinal disorders
congestion
6.2%
1/16 • 8 weeks
Ear and labyrinth disorders
earache
6.2%
1/16 • 8 weeks
Respiratory, thoracic and mediastinal disorders
rhinorrhea
25.0%
4/16 • 8 weeks
Respiratory, thoracic and mediastinal disorders
nasal congestion
6.2%
1/16 • 8 weeks
Psychiatric disorders
enuresis
6.2%
1/16 • 8 weeks
Skin and subcutaneous tissue disorders
skin, dry
6.2%
1/16 • 8 weeks
Skin and subcutaneous tissue disorders
sores under nose
6.2%
1/16 • 8 weeks
Skin and subcutaneous tissue disorders
irritation 'burning' application site, patch
6.2%
1/16 • 8 weeks
Skin and subcutaneous tissue disorders
hives
6.2%
1/16 • 8 weeks
Psychiatric disorders
less social
6.2%
1/16 • 8 weeks
Immune system disorders
allergies
6.2%
1/16 • 8 weeks
Eye disorders
itchy eyes
6.2%
1/16 • 8 weeks

Additional Information

Thomas M. Lock, M.D.

University of Oklahoma Health Sciences Center

Phone: 405-271-5700

Results disclosure agreements

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

Restriction type: LTE60