Insomnia, Psychomotor Agitation, Sleep Disorders, Circadian Rhythm
Conditions
Keywords
Kabuki Syndrome, Actigraphy, Treatment Response Measurement
Brief summary
The study examines and illustrates the utility of utilizing actigraphic measurements to assess treatment response.
Detailed description
Actigraphic measurements will be recorded every 30 seconds and will be recorded for an 11 day baseline period, followed by a 10 day period of the initial pharmacological interventions. The non-invasive nature of watch-like actigraphy devices is particularly attractive for use in pediatric populations. Meaningful treatment response measurements are obtained when actigraphic data is combined with psychometric and observational repeated measurements. Subsequent to the initial data collection the parent consented and the child assented to extend the study and continue measurements. The case study included baseline and repeated psychological, observational and actigraphic measurements that were initiated prior to treatment with risperidone and repeated throughout the treatment process with the final data collected in July 2010. The Personality Inventory for Children (PIC) is an objective multidimensional measurement of affect, behavior, ability and family function. The PIC was administered prior to treatment with risperidone and repeated after 22 months of treatment. The PIC serves as both an actuarial pre-treatment diagnostic tool as well as a post-treatment repeated measurement indicating treatment associated change. A primary observer (mother) was trained to report symptom percentages present since previous observations utilizing the operationally defined and observer defined items of the systematic observation scale throughout the treatment process. Actigraphic measurements provide a basis by which to measure sleep and sleep onset latency as well as periods of mobility and immobility. When programmed to record activity every thirty seconds, thousands of measurements are quickly captured. Actigraphic measurements were made utilizing a watch-like actigraphic device with an 11 day baseline actigraphic measurement period and continued measurements that included the initiation of a Once-Daily, Bedtime (q.h.s.) pharmacological intervention for 6 days, followed by a planned adjustment to Twice a Day (b.i.d.) dosing that was measured for an additional 4 days. Repeated actigraphic studies were subsequently conducted after 22 months of risperidone treatment and were collected along with the repeated observational and psychometric data. This case study explores activity and sleep characteristics related to behavioral characteristics and treatment response and the utility of single subject repeated actigraphic, psychometric and observational measurements and monitoring of treatment response.
Interventions
DRUGrisperidone
Five treatment conditions: 1. Baseline no medication 2. Risperidone .25 mg at bedtime (q.h.s.) x 7 days 3. Risperidone .25 mg twice daily (b.i.d.) 4. Risperidone .25 mg three times a day (t.i.d.) and 5. Risperidone .5 mg three times a day (t.i.d.).
Sponsors
Child Psychopharmacology Institute
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
2 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of Kabuki Syndrome * Medication Naive * Single subject preparing to receive pharmacological intervention
Exclusion criteria
* Inability to wear actigraphy device
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Actigraphic Measurement of Treatment Conditions | May 12- July 14, 2010 | The child's impulsivity and inability to sleep represented a significant symptom and risk factor. Impulsivity and sleep will be actigraphically assessed by treatment conditions. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Systematic Observation Scale: Percentage of Irritability Observed | May-7-2008 to July -14-2010 | — |
| Systematic Observation Scale: Percentage of Distractibility Observed | May-7-2008 to July -14-2010 | — |
| Systematic Observation Scale: Percentage of Hyperactivity Observed | May-7-2008 to July -14-2010 | — |
| The Personality Inventory for Children: Adjustment Scale | May-7-2008 to July -14-2010 | This scale measures general personality adjustment reflecting a dimension associated with highly adaptive to maladaptive adjustment. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. The PIC was administered prior to treatment with risperidone and repeated after 22 months of treatment. The PIC serves as both an actuarial pre-treatment diagnostic tool as well as a post-treatment repeated measurement indicating treatment and developmentally associated change. |
| The Personality Inventory for Children: Achievement Scale | May-7-2008 to July -14-2010 | The factors that underlie this scale are academic ability, poor achievement, impulsivity, limited concentration and over or under-assertiveness with peers. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. |
| Systematic Observation Scale Item: Percentage of Impulsivity Observed | May-7-2008 to July -14-2010 | The Systematic Observation Scale utilizes single-subject repeated measurements. Symptoms and issues of interest are defined and a variety of frequency and sampling methods can be applied. The Systematic Observation Scale was designed so Primary Observers (parents, guardians, self observers or others) can make pre-treatment and subsequent observations to track, document and evaluate symptom variation over the course of an illness. The measurement utilized is the percentage of time the symptom is observed by the primary observer since the previous observation. |
| The Personality Inventory for Children: Delinquency Scale | May 7, 2008-July 14,2010 | The factors that underlie this scale are poor frustration tolerance, irritability, sadness, lack of interest, hostility,limited social participation and resistance to authority. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. |
| The Personality Inventory for Children: Withdrawal Scale | May-7-2008 to July -14-2010 | The factors that underlie this scale are physical isolation, Shyness, isolation from peers, emotional distance and isolated intellectual interests. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function |
| The Personality Inventory for Children: Social Skills Scale | May-7-2008 to July -14-2010 | The factors that underlie this scale are social success, social rejection, adults as only social contacts, and the ability to lead and follow. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. |
| The Personality Inventory for Children: Family Relations Scale | May-7-2008 to July -14-2010 | The factors that underlie this scale are family stability, inter-parent communication, presence of love and happiness in the home and appropriateness of discipline. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. |
| The Personality Inventory for Children: Somatic-Physiological Scale | May-7-2008 to July -14-2010 | The factors that underlie this scale are fatigue, aches and pains, insomnia, somatic response to stress and malingering. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. |
Countries
United States
Participant flow
Recruitment details
A six year old child with Autism Spectrum Disorder (pervasive developmental disorder) and Kabuki Syndrome anticipating a pharmacological intervention was evaluated for sleep and behavioral disturbances. She was invited to participate, her parents provided consent and the child acknowledged her assent.
Pre-assignment details
The child had previously not responded to multiple medication trials, was stimulated by diphenylhydramine, over-sedated on clonidine and had mood destabilization when tried on mirtazapine. The child received no medication for the month preceding the initiation of the study.
Participants by arm
| Arm | Count |
|---|---|
| Actigraphic (Activity Count) Measurement of Sleep and Activity Actigraphic measurements obtained by attaching an actigraphic watch device to the child's non-dominant wrist obtain measurements that occur every thirty seconds for a duration of approximately 21 days. These measurements reflect treatment condition sleep characteristics where sleep is indicated by activity counts below 40. Three 21 day actigraphic measurement periods occurred with the initial measurement occurring through baseline period and the period in which the child's pharmacological treatment was initiated. The second and third, 21 day periods of Actigraphic measurement occurred at 22 and 23 months from baseline. Five treatment conditions during the measurement periods including 1. baseline no medication 2. risperidone .25 mg at bedtime for 7 days (q.h.s.) 3. risperidone twice daily (b.i.d.) 4. risperidone .25 mg three times a day (t.i.d.) and 5. risperidone .50 mg three times a day (t.i.d.) | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Actigraphic (Activity Count) Measurement of Sleep and Activity |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 6 years STANDARD_DEVIATION 0 |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
Actigraphic Measurement of Treatment Conditions
The child's impulsivity and inability to sleep represented a significant symptom and risk factor. Impulsivity and sleep will be actigraphically assessed by treatment conditions.
Time frame: May 12- July 14, 2010
Population: Single subject repeated Actigraphic(at thirty second intervals), observational (every two months) and psychometric (baseline and at 23 months) measurements of a child anticipating pharmacological treatment. Actigraphic measurements (three 21 day periods) over five treatment conditions that included a non-medication baseline and spanned 23 months.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Actigraphic (Activity Count) Measurement of Sleep and Activity | Actigraphic Measurement of Treatment Conditions | Baseline No Medication Midnight to 1:00 AM | 55.02 activity | Standard Deviation 206.432 |
| Actigraphic (Activity Count) Measurement of Sleep and Activity | Actigraphic Measurement of Treatment Conditions | risperidone .5 mg t.i.d. Midnight to 1:00 AM | 9.94 activity | Standard Deviation 51.651 |
Secondary
Systematic Observation Scale Item: Percentage of Impulsivity Observed
The Systematic Observation Scale utilizes single-subject repeated measurements. Symptoms and issues of interest are defined and a variety of frequency and sampling methods can be applied. The Systematic Observation Scale was designed so Primary Observers (parents, guardians, self observers or others) can make pre-treatment and subsequent observations to track, document and evaluate symptom variation over the course of an illness. The measurement utilized is the percentage of time the symptom is observed by the primary observer since the previous observation.
Time frame: May-7-2008 to July -14-2010
Secondary
Systematic Observation Scale: Percentage of Distractibility Observed
Time frame: May-7-2008 to July -14-2010
Secondary
Systematic Observation Scale: Percentage of Hyperactivity Observed
Time frame: May-7-2008 to July -14-2010
Secondary
Systematic Observation Scale: Percentage of Irritability Observed
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Achievement Scale
The factors that underlie this scale are academic ability, poor achievement, impulsivity, limited concentration and over or under-assertiveness with peers. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function.
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Adjustment Scale
This scale measures general personality adjustment reflecting a dimension associated with highly adaptive to maladaptive adjustment. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function. The PIC was administered prior to treatment with risperidone and repeated after 22 months of treatment. The PIC serves as both an actuarial pre-treatment diagnostic tool as well as a post-treatment repeated measurement indicating treatment and developmentally associated change.
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Delinquency Scale
The factors that underlie this scale are poor frustration tolerance, irritability, sadness, lack of interest, hostility,limited social participation and resistance to authority. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function.
Time frame: May 7, 2008-July 14,2010
Secondary
The Personality Inventory for Children: Family Relations Scale
The factors that underlie this scale are family stability, inter-parent communication, presence of love and happiness in the home and appropriateness of discipline. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function.
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Social Skills Scale
The factors that underlie this scale are social success, social rejection, adults as only social contacts, and the ability to lead and follow. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function.
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Somatic-Physiological Scale
The factors that underlie this scale are fatigue, aches and pains, insomnia, somatic response to stress and malingering. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function.
Time frame: May-7-2008 to July -14-2010
Secondary
The Personality Inventory for Children: Withdrawal Scale
The factors that underlie this scale are physical isolation, Shyness, isolation from peers, emotional distance and isolated intellectual interests. The Personality Inventory for Children (PIC) is an age and gender referenced objective multidimensional measurement of affect, behavior, ability and family function
Time frame: May-7-2008 to July -14-2010