Ignite Creation Date:
2024-05-06 @ 6:54 PM
Last Modification Date:
2024-10-26 @ 2:57 PM
Study NCT ID:
NCT05829668
Status:
RECRUITING
Last Update Posted:
2023-08-21
First Post:
2023-02-14
Brief Title:
Behavioral Assessment and Treatment of Problem Behavior in Children With Cornelia de Lange Syndrome
Sponsor:
Hugo W Moser Research Institute at Kennedy Krieger Inc
Organization:
Hugo W Moser Research Institute at Kennedy Krieger Inc
Study Overview
Official Title:
Behavioral Assessment and Treatment of Problem Behavior in Children With Cornelia de Lange Syndrome
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goals of this clinical trial are to identify factors associated with the development of problem behavior in Cornelia de Lange syndrome CdLS and to develop an effective behavioral assessment and treatment model for problem behavior in children with CdLS
The hypotheses are as follows
1 Based on pilot data the investigators hypothesize that individuals with CdLS will exhibit preferences for auditory stimuli relative to other categories eg visual tactile of stimuli
2 Based on pilot data the investigators hypothesize that individuals with CdLS will exhibit problem behavior to obtain adult attention or to escape task demands relative to tangible and control conditions as measured by functional analysis results
3 Function-based behavioral treatments will reduce problem behavior in individuals with CdLS by 80 or greater relative to baseline rates
4 Individuals with CdLS and problem behavior will exhibit more impaired communication demonstrate increased emotion dysregulation and exhibit more severe symptoms of Autism Spectrum Disorder ASD relative to those with CdLS and no problem behavior
Participants in the intervention group families of children with CdLS and problem behaviors will be asked to complete study measures and attend 2 full days and one half-day of clinic services at Kennedy Krieger Institute so that the study team can provide assessment and treatment of child problem behaviors and then train parents to apply the intervention Participants in the control group families of children with CdLS and no problem behavior will be asked to complete study measures once every 3 months for a 2-year period to monitor the children
This study will improve the ability to effectively treat problem behavior is CdLS as well as identify key variables associated with problem behavior in CdLS which may be examined in future studies and clinical practice to foster early intervention and prevention efforts
The hypotheses are as follows
1 Based on pilot data the investigators hypothesize that individuals with CdLS will exhibit preferences for auditory stimuli relative to other categories eg visual tactile of stimuli
2 Based on pilot data the investigators hypothesize that individuals with CdLS will exhibit problem behavior to obtain adult attention or to escape task demands relative to tangible and control conditions as measured by functional analysis results
3 Function-based behavioral treatments will reduce problem behavior in individuals with CdLS by 80 or greater relative to baseline rates
4 Individuals with CdLS and problem behavior will exhibit more impaired communication demonstrate increased emotion dysregulation and exhibit more severe symptoms of Autism Spectrum Disorder ASD relative to those with CdLS and no problem behavior
Participants in the intervention group families of children with CdLS and problem behaviors will be asked to complete study measures and attend 2 full days and one half-day of clinic services at Kennedy Krieger Institute so that the study team can provide assessment and treatment of child problem behaviors and then train parents to apply the intervention Participants in the control group families of children with CdLS and no problem behavior will be asked to complete study measures once every 3 months for a 2-year period to monitor the children
This study will improve the ability to effectively treat problem behavior is CdLS as well as identify key variables associated with problem behavior in CdLS which may be examined in future studies and clinical practice to foster early intervention and prevention efforts
Detailed Description:
The presence of problem behavior such as self-injury and aggression is a prominent and challenging feature of Cornelia de Lange syndrome CdLS Despite the growing body of research on behavior problems associated with behavioral phenotypes in genetic disorders problem behavior in this population is not well understood For example individuals with CdLS are at high risk for developing self-jury however it is not known why some do not develop this problem behavior Additionally few studies have examined environmental influences on problem behavior in CdLS The field of applied behavior analysis ABA offers precise assessment methods that have led to an increased understanding of problem behavior and improved treatment outcomes for individuals with intellectual and developmental disabilities IDD In particular functional analysis procedures are considered the most rigorous method for determining what variables serve to maintain problem behavior thereby leading to the prescription of effective individualized treatments However there are only a few cases reported in the literature of functional analysis of problem behavior in CdLS Preliminary findings from functional analyses and other assessments in CdLS indicate differences in forms of problem behavior functions for problem behavior and responsiveness to certain classes of stimuli relative to other genetic disorders Previous literature and preliminary data suggest that variables associated with certain genetic conditions may impact form function and responsiveness to treatment for problem behavior Thus the goals of this clinical trial are to 1 to identify key variables associated with problem behavior in CdLS by comparing children with and without problem behavior across multiple variables including language ability stimulus preferences emotion dysregulation and presence of autism and 2 evaluate the effectiveness of an outpatient model of ABA-based assessment and treatment for children with CdLS exhibiting problem behavior and 3 follow children with CDLS without problem behavior over a period of 2 years to begin to obtain information about what variables lead to the emergence of problem behavior and to apply early intervention strategies
Individualized function-based treatments will be evaluated with each participant using single-case experimental designs Assessment and treatment data from all participants will also be combined to evaluate program outcomes within a consecutive controlled case series design This type of study design minimizes potential selection bias favoring positive outcomes by including all participants encountered regardless of outcome All participants will complete standardized measures of preference adaptive skills problem behavior ASD and emotion dysregulation Program outcomes will also be evaluated using statistical analysis methods with consultation from the Intellectual and Developmental Disabilities Research Center IDDRC Behavioral Phenotyping Core at the Kennedy Krieger InstituteJohns Hopkins University Parent responses on study measures will be combined to evaluate satisfaction with treatment and with services
Treatment Group
Pre-Visit Assessments
1 Parent will be asked to complete research measures at home including Vineland Adaptive Behavior Scales VABS-3 caregiver rating form which is a standardized measure that provides developmental scores for communication skills daily living skills and socialization skills Emotional Dysregulation Inventory EDI which is a caregiver-report questionnaire designed to capture emotional distress and problems with emotion regulation in both minimally verbal and verbal individuals the Behavior Problem Inventory BPI which is a 49-item parent-completed instrument designed to measure the frequency and severity of a childs self-injurious aggressivedestructive and stereotypic behaviors the Repetitive Behavior Scale-Revised RBS-R a 44-item inventory which measures the presence of repetitive and stereotypic behaviors in individual with ASD the Aberrant Behavior Checklist ABC a 58-item inventory designed to measure a variety of emotional and behavior difficulties in individuals with IDD and the Gilliam Autism Rating Scale 2nd Edition GARS Completed measures will be mailed back to the study team
2 Parent will be asked to provide any records or reports related to problem behavior including functional behavioral assessment behavioral intervention plan or behavioral data regarding previous or current behavioral services
3 After parent returns measures by mail a study team member will conduct a parent interview by phone to complete the Reinforcer Assessment Inventory for the Severely Disabled RAISD and the Functional Analysis Interview
Study Visit at Kennedy Krieger Institute KKIDay 1
1 For any child with self-injurious behavior SIB the team will administer the Self-Injury Trauma scale SIT which is designed to quantify the amount of tissue damage associated with SIB
2 Conduct paired choice preference assessment to identify rank order of preferred stimuli that may be used in treatment
3 Conduct a functional analysis to identify environmental variables maintaining child problem behavior Sessions will be conducted in accordance with a standard protocol in use by the Neurobehavioral Unit Outpatient clinic NBU OPC including conditions that test for social functions of problem behavior The study team will video-record these sessions
Study Visit at KKIDay 2
1 Study team and parent meet to discuss assessment findings and plan for treatment evaluation
2 Conduct treatment evaluation of function-based intervention Treatment options are within the standard of care in the NBU OPC The study team will video-record these sessions
3 Parent completes the Intervention Rating Profile-15 a 15-item measure of treatment acceptability
4 Train parent to implement behavioral treatment using standard procedures within the NBU OPC
5 Parent completes the Intervention Rating Profile-15 and the Family Satisfaction Survey
6 Follow-up phone call 1 week post treatment to assess child progress The study team will also provide a brief summary of the childs preferred items activities and a summary of the results from the questionnaires the parent completed
Study visit at KKIFollow Up 2 weeks post treatment During a 3-hour study visit the continued effectiveness of the behavior plan and parent implementation of such will be assessed via direct observation of parent and child The study team will video-record these sessions Results will be reviewed with the family Parents will also complete the ABC and BPI
Follow-up phone calls at 4 8 and 12 weeks post treatment Refer for additional services if necessary
Control Group
Once ages and adaptive functioning levels of treatment group participants are established recruitment for matched controls will be initiated Investigators will seek to identify children with CdLS between the ages of 3 and 15 years who do not exhibit self-injurious behavior aggression or disruptive behavior Parents of potential participants will be asked to participate in 2 phone interviews In the first phone interview a parent will be asked a series of questions to rule out the presence of child problem behavior Once it is determined that the child does not exhibit problem behavior parents will be asked if they would like to participate in the study After the family has consented the study team will mail the VABS-3 for completion If VABS-3 results match that of a treatment group participant a second phone interview will be conducted to complete the RAISD The BPI ABC RBS-R GARS and EDI will be mailed Local families will be invited to attend a 2-hr clinic visit for preference assessments To monitor for emergence of problem behavior in the control group the BPI and EDI will be repeated every 3 months for up to 2 yrs If such behavior does emerge clinical services will be offered to the participant and family at no cost
Individualized function-based treatments will be evaluated with each participant using single-case experimental designs Assessment and treatment data from all participants will also be combined to evaluate program outcomes within a consecutive controlled case series design This type of study design minimizes potential selection bias favoring positive outcomes by including all participants encountered regardless of outcome All participants will complete standardized measures of preference adaptive skills problem behavior ASD and emotion dysregulation Program outcomes will also be evaluated using statistical analysis methods with consultation from the Intellectual and Developmental Disabilities Research Center IDDRC Behavioral Phenotyping Core at the Kennedy Krieger InstituteJohns Hopkins University Parent responses on study measures will be combined to evaluate satisfaction with treatment and with services
Treatment Group
Pre-Visit Assessments
1 Parent will be asked to complete research measures at home including Vineland Adaptive Behavior Scales VABS-3 caregiver rating form which is a standardized measure that provides developmental scores for communication skills daily living skills and socialization skills Emotional Dysregulation Inventory EDI which is a caregiver-report questionnaire designed to capture emotional distress and problems with emotion regulation in both minimally verbal and verbal individuals the Behavior Problem Inventory BPI which is a 49-item parent-completed instrument designed to measure the frequency and severity of a childs self-injurious aggressivedestructive and stereotypic behaviors the Repetitive Behavior Scale-Revised RBS-R a 44-item inventory which measures the presence of repetitive and stereotypic behaviors in individual with ASD the Aberrant Behavior Checklist ABC a 58-item inventory designed to measure a variety of emotional and behavior difficulties in individuals with IDD and the Gilliam Autism Rating Scale 2nd Edition GARS Completed measures will be mailed back to the study team
2 Parent will be asked to provide any records or reports related to problem behavior including functional behavioral assessment behavioral intervention plan or behavioral data regarding previous or current behavioral services
3 After parent returns measures by mail a study team member will conduct a parent interview by phone to complete the Reinforcer Assessment Inventory for the Severely Disabled RAISD and the Functional Analysis Interview
Study Visit at Kennedy Krieger Institute KKIDay 1
1 For any child with self-injurious behavior SIB the team will administer the Self-Injury Trauma scale SIT which is designed to quantify the amount of tissue damage associated with SIB
2 Conduct paired choice preference assessment to identify rank order of preferred stimuli that may be used in treatment
3 Conduct a functional analysis to identify environmental variables maintaining child problem behavior Sessions will be conducted in accordance with a standard protocol in use by the Neurobehavioral Unit Outpatient clinic NBU OPC including conditions that test for social functions of problem behavior The study team will video-record these sessions
Study Visit at KKIDay 2
1 Study team and parent meet to discuss assessment findings and plan for treatment evaluation
2 Conduct treatment evaluation of function-based intervention Treatment options are within the standard of care in the NBU OPC The study team will video-record these sessions
3 Parent completes the Intervention Rating Profile-15 a 15-item measure of treatment acceptability
4 Train parent to implement behavioral treatment using standard procedures within the NBU OPC
5 Parent completes the Intervention Rating Profile-15 and the Family Satisfaction Survey
6 Follow-up phone call 1 week post treatment to assess child progress The study team will also provide a brief summary of the childs preferred items activities and a summary of the results from the questionnaires the parent completed
Study visit at KKIFollow Up 2 weeks post treatment During a 3-hour study visit the continued effectiveness of the behavior plan and parent implementation of such will be assessed via direct observation of parent and child The study team will video-record these sessions Results will be reviewed with the family Parents will also complete the ABC and BPI
Follow-up phone calls at 4 8 and 12 weeks post treatment Refer for additional services if necessary
Control Group
Once ages and adaptive functioning levels of treatment group participants are established recruitment for matched controls will be initiated Investigators will seek to identify children with CdLS between the ages of 3 and 15 years who do not exhibit self-injurious behavior aggression or disruptive behavior Parents of potential participants will be asked to participate in 2 phone interviews In the first phone interview a parent will be asked a series of questions to rule out the presence of child problem behavior Once it is determined that the child does not exhibit problem behavior parents will be asked if they would like to participate in the study After the family has consented the study team will mail the VABS-3 for completion If VABS-3 results match that of a treatment group participant a second phone interview will be conducted to complete the RAISD The BPI ABC RBS-R GARS and EDI will be mailed Local families will be invited to attend a 2-hr clinic visit for preference assessments To monitor for emergence of problem behavior in the control group the BPI and EDI will be repeated every 3 months for up to 2 yrs If such behavior does emerge clinical services will be offered to the participant and family at no cost
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None