Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| MH 082119 |
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| National Institute of Mental Health (NIMH) | NIH |
| Bristol-Myers Squibb | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to develop a better tolerated and more effective pharmacologic treatment with individuals with Pervasive Developmental Disorder. This is a double-blind, placebo-controlled study of aripiprazole in the management of the maladaptive behaviors of Pervasive Developmental Disorder. The investigators hypothesize that aripiprazole will be more effective than placebo for reducing aggression, tantrum and self-injurious behavior in children with Pervasive Developmental Disorder.
Pervasive developmental disorders (PDDs) are characterized by severe impairments in social interaction and communication in addition to restricted patterns of interests and activities. Research suggests that a dysregulation of the dopamine and serotonin systems contributes to these interfering behaviors in individuals with PDD. After benefits of typical neuroleptics were reported in subjects with PDD, research shifted to the atypical antipsychotics which have been shown to be better tolerated and effective in this population. However, the atypical antipsychotics have also been associated with adverse effects. Thus there remains a need for a novel pharmacotherapy that would be safe and effective for children and adolescents with PDDs. The primary objectives of this study are to determine whether aripiprazole is effective and well tolerated for irritability in children and adolescents with PDD not otherwise specified (NOS) during an 8-week acute phase and whether the effectiveness and tolerability of aripiprazole is maintained during a 16-week continuation phase.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1. Aripiprazole oral product | Experimental | Participants will receive Aripiprazole oral product with a minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment. |
|
| Arm 2. Placebo oral capsule | Placebo Comparator | Participants will receive matching (identical in size and appearance to study drug) placebo oral capsules over 8-weeks of treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aripiprazole oral product | Drug | Minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Improved as Measured by the Clinical Global Impression-Global Improvement Scale (Improvement Defined as CGI-I=1 or CGI-I=2) | Clinical Global Impressions (Guy, 1976) global improvement (CGI-I) is designed to take into account all factors to arrive at an assessment of response to treatment. The CGI-I scale ranges from 1 to 7, with lower scores indicating greater improvement (1=very much improved and 2=much improved). Participants with a CGI-I score of 1 or 2 were classified as improved. Four participants assigned to placebo completed an exit interview prior to week 8. One participant assigned to placebo and one participant assigned to aripiprazole withdrew from the study without completing an exit interview. | Double-blind phase study exit - up to 8 weeks |
| Mean Post-baseline Aberrant Behavior Checklist Irritability Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Irritability subscale is 0 to 45. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. Confidence intervals reflect a Bonferroni multiple testing correction accounting for the selection of two primary outcomes. | Weeks 1, 2, 3, 4, 6 and 8 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Post-baseline Aberrant Behavior Checklist Hyperactivity Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 overactivity (7 items), impulsiveness (2 items), inattention (3 items) and noncompliance (4 items). It has been used as a primary outcome measure in several trials in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores is 0 to 48 on the Hyperactivity subscale. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Kimberly A. Stigler, MD | Indiana University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Riley Hospital for Children, Christian Sarkine Autism Treatment Center | Indianapolis | Indiana | 46202 | United States |
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Arm 1. Aripiprazole Oral Product | Participants will receive Aripiprazole oral product with a minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment. Aripiprazole oral product: Minimum dose of 2mg per day to a maximum of 20 mg per day over 8-weeks of treatment. |
| FG001 | Arm 2. Placebo Oral Capsule | Participants will receive matching (identical in size and appearance to study drug) placebo oral capsules over 8-weeks of treatment. Placebo oral capsule: Placebo will identical in size and appearance to study drug. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm 1. Aripiprazole Oral Product | Participants will receive Aripiprazole oral product with a minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment. Aripiprazole oral product: Minimum dose of 2mg per day to a maximum of 20 mg per day over 8-weeks of treatment. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants Improved as Measured by the Clinical Global Impression-Global Improvement Scale (Improvement Defined as CGI-I=1 or CGI-I=2) | Clinical Global Impressions (Guy, 1976) global improvement (CGI-I) is designed to take into account all factors to arrive at an assessment of response to treatment. The CGI-I scale ranges from 1 to 7, with lower scores indicating greater improvement (1=very much improved and 2=much improved). Participants with a CGI-I score of 1 or 2 were classified as improved. Four participants assigned to placebo completed an exit interview prior to week 8. One participant assigned to placebo and one participant assigned to aripiprazole withdrew from the study without completing an exit interview. | All randomized study participants who completed an exit interview | Posted | Number | participants | Double-blind phase study exit - up to 8 weeks |
|
Eight weeks or at the time of latest data collection.
Adverse event information was collected via a structured side effect rating scale completed with the participant and their primary caregiver. This included a list of side-effects that have been reported with aripiprazole at a rate greater than 1%. The physician also asked about any visits to the doctor, new medication use, or any other complaints. Events that were not present at baseline and occurred or worsened after the date of randomization are reported.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm 1. Aripiprazole Oral Product | Participants will receive Aripiprazole oral product with a minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment. Aripiprazole oral product: Minimum dose of 2mg per day to a maximum of 20 mg per day over 8-weeks of treatment. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
The PI who conducted the trial left the institution, Indiana University, prior to data analysis. The institution negotiated/executed a data transfer agreement for the PI's primary mentor, Dr. Christopher McDougle, to analyze the data on her behalf.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Christopher J. McDougle, MD | Indiana University | 781-860-1783 | cmcdougle@partners.org |
Not provided
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| ID | Term |
|---|---|
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| D000068180 | Aripiprazole |
| D000073893 | Sugars |
| ID | Term |
|---|---|
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D015363 | Quinolones |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo oral capsule | Drug | Placebo will be identical in size and appearance to study drug. |
|
|
| Weeks 1, 2, 3, 4, 6 and 8 |
| Mean Post-baseline Aberrant Behavior Checklist Inappropriate Speech Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Inappropriate Speech subscale is 0 to 12. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | Weeks 1, 2, 3, 4, 6 and 8 |
| Mean Post-baseline Aberrant Behavior Checklist Social Withdrawal Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Social Withdrawal subscale is 0 to 48. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | Weeks 1, 2, 3, 4, 6 and 8 |
| Mean Post-baseline Aberrant Behavior Checklist Stereotypy Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Stereotypy subscale is 0 to 21. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | Weeks 1, 2, 3, 4, 6 and 8 |
| Adverse Event |
|
| Arm 2. Placebo Oral Capsule |
Participants will receive matching (identical in size and appearance to study drug) placebo oral capsules over 8-weeks of treatment. Placebo oral capsule: Placebo will identical in size and appearance to study drug. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Participants will receive Aripiprazole oral product with a minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment.
Aripiprazole oral product: Minimum dose of 2 mg per day to a maximum dose of 20 mg per day over 8-weeks of treatment.
| OG001 | Arm 2. Placebo Oral Capsule | Participants will receive matching (identical in size and appearance to study drug) placebo oral capsules over 8-weeks of treatment. Placebo oral capsule: Placebo will be identical in size and appearance to study drug. |
|
|
| Primary | Mean Post-baseline Aberrant Behavior Checklist Irritability Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Irritability subscale is 0 to 45. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. Confidence intervals reflect a Bonferroni multiple testing correction accounting for the selection of two primary outcomes. | All randomized study participants | Posted | Mean | 95% Confidence Interval | units on a scale | Weeks 1, 2, 3, 4, 6 and 8 |
|
|
|
| Secondary | Mean Post-baseline Aberrant Behavior Checklist Hyperactivity Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 overactivity (7 items), impulsiveness (2 items), inattention (3 items) and noncompliance (4 items). It has been used as a primary outcome measure in several trials in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores is 0 to 48 on the Hyperactivity subscale. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | All randomized study participants | Posted | Mean | 95% Confidence Interval | units on a scale | Weeks 1, 2, 3, 4, 6 and 8 |
|
|
|
| Secondary | Mean Post-baseline Aberrant Behavior Checklist Inappropriate Speech Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Inappropriate Speech subscale is 0 to 12. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | All randomized study participants | Posted | Mean | 95% Confidence Interval | units on a scale | Weeks 1, 2, 3, 4, 6 and 8 |
|
|
|
| Secondary | Mean Post-baseline Aberrant Behavior Checklist Social Withdrawal Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Social Withdrawal subscale is 0 to 48. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | All randomized study participants | Posted | Mean | 95% Confidence Interval | units on a scale | Weeks 1, 2, 3, 4, 6 and 8 |
|
|
|
| Secondary | Mean Post-baseline Aberrant Behavior Checklist Stereotypy Subscale Score, Parent Report, Double-blind Phase | The Aberrant Behavior Checklist (ABC) is a symptom checklist for assessing problem behaviors in individuals ages 6 to 54 years-old 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 in children with developmental disabilities. The interpretation of the tool and its subscales is that a greater number of items indicates greater severity. The range of scores for the Stereotypy subscale is 0 to 21. Means were estimated using a repeated measures linear regression model with treatment group, baseline score, study week (in categories), and Tanner stage as covariates. A linear contrast estimated the average across study timepoints. | All randomized study participants | Posted | Mean | 95% Confidence Interval | units on a scale | Weeks 1, 2, 3, 4, 6 and 8 |
|
|
|
| 0 |
| 17 |
| 0 |
| 17 |
| 16 |
| 17 |
| EG001 | Arm 2. Placebo Oral Capsule | Participants will receive matching (identical in size and appearance to study drug) placebo oral capsules over 8-weeks of treatment. Placebo oral capsule: Placebo will identical in size and appearance to study drug. | 0 | 16 | 0 | 16 | 13 | 16 |
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Stomach or abdominal discomfort | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Appetite decrease | General disorders | Systematic Assessment |
|
| Appetite increase | General disorders | Systematic Assessment |
|
| Dry mouth | General disorders | Systematic Assessment |
|
| Fever | General disorders | Systematic Assessment |
|
| Hypersalivation | General disorders | Systematic Assessment |
|
| Increased thirst | General disorders | Systematic Assessment |
|
| Other Pain | General disorders | Systematic Assessment |
|
| Sedation/Drowsiness | General disorders | Systematic Assessment |
|
| Tiredness/Fatigue | General disorders | Systematic Assessment |
|
| Allergies Not Otherwise Specified | Immune system disorders | Systematic Assessment |
|
| Flu or upper respiratory problems | Infections and infestations | Systematic Assessment |
|
| Nasal congestion or Cold | Infections and infestations | Systematic Assessment |
|
| Sore throat | Infections and infestations | Systematic Assessment |
|
| Weight gain | Metabolism and nutrition disorders | Systematic Assessment |
|
| Weight loss | Metabolism and nutrition disorders | Systematic Assessment |
|
| Muscle/bone/joint pain/condition | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Tremor | Nervous system disorders | Systematic Assessment |
|
| Unspecified or not otherwise listed head | Nervous system disorders | Systematic Assessment |
|
| Anxiety/Nervousness/Worry | Psychiatric disorders | Systematic Assessment |
|
| Change in speech | Psychiatric disorders | Systematic Assessment |
|
| Concentration difficulty | Psychiatric disorders | Systematic Assessment |
|
| Daydreaming | Psychiatric disorders | Systematic Assessment |
|
| Difficulty falling asleep | Psychiatric disorders | Systematic Assessment |
|
| Echolalia | Psychiatric disorders | Systematic Assessment |
|
| Emotional outburst | Psychiatric disorders | Systematic Assessment |
|
| Hypertalkativeness | Psychiatric disorders | Systematic Assessment |
|
| Increased motor activity | Psychiatric disorders | Systematic Assessment |
|
| Interrupted sleep/other sleep problems | Psychiatric disorders | Systematic Assessment |
|
| Irritability | Psychiatric disorders | Systematic Assessment |
|
| Restlessness/Agitation including fidgety | Psychiatric disorders | Systematic Assessment |
|
| Stereotypy | Psychiatric disorders | Systematic Assessment |
|
| Unspecified or not otherwise listed psych | Psychiatric disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Generalized rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Localized rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Intermittent nosebleed | Vascular disorders | Systematic Assessment |
|
Not provided
Not provided
| D011804 |
| Quinolines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D002241 | Carbohydrates |