Treatment of Disruptive Behaviors in Fragile X Syndrome
Primary Purpose
Fragile X Syndrome, Disruptive Behavior
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Behavior analytic treatment
Sponsored by
About this trial
This is an interventional treatment trial for Fragile X Syndrome
Eligibility Criteria
Inclusion Criteria:
- Child has a confirmed diagnosis of FXS (>200 CGG repeats on the FMR1 gene with evidence of aberrant methylation)
- Child is male, between the ages of 3-10 years old
- Child is reported to show self-injury, property destruction and/or aggression on at least a daily basis
- The caregiver agrees to keep any therapies that the child receives (i.e., medications or other treatments) as stable as possible throughout involvement in the study
- The family has a high-speed internet connection at home or lives in an area with 4G network coverage
- Availability for one-hour daily telemedicine treatment sessions
- Availability for in-home assessment totaling 8 hours across two consecutive days
Exclusion Criteria:
- The child or caregiver has significant sensory impairments (e.g., blindness or deafness)
- Non-English speaking
- The child receives Applied Behavior Analysis services in excess of five hours per week
- The child has a significant neurological condition (e.g., frequent seizures, brain injury, Tourette's syndrome) that would preclude participation
- The child or caregiver has significant mobility issues
- The child is currently participating in another research study that would preclude participation in the study
Sites / Locations
- Department of Psychiatry and Behavioral Sciences
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Treatment
Observation
Arm Description
Outcomes
Primary Outcome Measures
Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks
Aberrant Behavior Checklist - Community (ABC-C)
Secondary Outcome Measures
Change from baseline level of treatment acceptability at 4, 8, 12 and 16 weeks
Treatment Acceptability Rating Form - Revised (TARF-R)
Full Information
NCT ID
NCT03510156
First Posted
April 17, 2018
Last Updated
May 3, 2022
Sponsor
Stanford University
Collaborators
The John Merck Fund
1. Study Identification
Unique Protocol Identification Number
NCT03510156
Brief Title
Treatment of Disruptive Behaviors in Fragile X Syndrome
Official Title
Treatment of Disruptive Behaviors in Fragile X Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
July 2016 (Actual)
Primary Completion Date
March 2022 (Actual)
Study Completion Date
March 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
Collaborators
The John Merck Fund
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Disruptive behaviors such as self-injury, aggression, and property destruction pose significant health-related issues to children diagnosed with fragile X syndrome (FXS), impacting the child's quality of life and causing significant distress to families.
Access to appropriate treatment for families is severely limited by factors such as cost of care, shortages of qualified treatment providers, and geographic spread of children with FXS across the country. To address these potential issues, the effectiveness of administering a standardized function-based behavioral treatment for problem behaviors in FXS will be evaluated using telemedicine. The proposed study intervention therefore offers a tremendous step forward in clinical research both in the field of FXS and in the field of developmental disabilities more broadly, and thus will have a significant impact on public health.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome, Disruptive Behavior
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
60 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment
Arm Type
Experimental
Arm Title
Observation
Arm Type
No Intervention
Intervention Type
Behavioral
Intervention Name(s)
Behavior analytic treatment
Intervention Description
Function-based treatment
Primary Outcome Measure Information:
Title
Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks
Description
Aberrant Behavior Checklist - Community (ABC-C)
Time Frame
0, 4, 8, 12, 16 weeks
Secondary Outcome Measure Information:
Title
Change from baseline level of treatment acceptability at 4, 8, 12 and 16 weeks
Description
Treatment Acceptability Rating Form - Revised (TARF-R)
Time Frame
0, 4, 8, 12, 16 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Child has a confirmed diagnosis of FXS (>200 CGG repeats on the FMR1 gene with evidence of aberrant methylation)
Child is male, between the ages of 3-10 years old
Child is reported to show self-injury, property destruction and/or aggression on at least a daily basis
The caregiver agrees to keep any therapies that the child receives (i.e., medications or other treatments) as stable as possible throughout involvement in the study
The family has a high-speed internet connection at home or lives in an area with 4G network coverage
Availability for one-hour daily telemedicine treatment sessions
Availability for in-home assessment totaling 8 hours across two consecutive days
Exclusion Criteria:
The child or caregiver has significant sensory impairments (e.g., blindness or deafness)
Non-English speaking
The child receives Applied Behavior Analysis services in excess of five hours per week
The child has a significant neurological condition (e.g., frequent seizures, brain injury, Tourette's syndrome) that would preclude participation
The child or caregiver has significant mobility issues
The child is currently participating in another research study that would preclude participation in the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Scott S Hall, PhD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Psychiatry and Behavioral Sciences
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
36180840
Citation
Hall SS, Rodriguez AB, Jo B, Pollard JS. Long-term follow-up of telehealth-enabled behavioral treatment for challenging behaviors in boys with fragile X syndrome. J Neurodev Disord. 2022 Sep 30;14(1):53. doi: 10.1186/s11689-022-09463-9.
Results Reference
derived
PubMed Identifier
33218305
Citation
Hall SS, Monlux KD, Rodriguez AB, Jo B, Pollard JS. Telehealth-enabled behavioral treatment for problem behaviors in boys with fragile X syndrome: a randomized controlled trial. J Neurodev Disord. 2020 Nov 20;12(1):31. doi: 10.1186/s11689-020-09331-4.
Results Reference
derived
Learn more about this trial
Treatment of Disruptive Behaviors in Fragile X Syndrome
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