Clinical trial NCT03510156: Treatment of Disruptive Behaviors in Fragile X Syndrome
Official title: Treatment of Disruptive Behaviors in Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Fragile X Syndrome
Disruptive Behavior
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: The John Merck Fund
First posted: 2018-04-27
Enrollment: 60
Sex: MALE
Minimum age (years): 3
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. Child has a confirmed diagnosis of FXS (\>200 CGG repeats on the FMR1 gene with evidence of aberrant methylation)
2. Child is male, between the ages of 3-10 years old
3. Child is reported to show self-injury, property destruction and/or aggression on at least a daily basis
4. 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
5. The family has a high-speed internet connection at home or lives in an area with 4G network coverage
6. Availability for one-hour daily telemedicine treatment sessions
7. Availability for in-home assessment totaling 8 hours across two consecutive days

Exclusion Criteria:

1. The child or caregiver has significant sensory impairments (e.g., blindness or deafness)
2. Non-English speaking
3. The child receives Applied Behavior Analysis services in excess of five hours per week
4. The child has a significant neurological condition (e.g., frequent seizures, brain injury, Tourette's syndrome) that would preclude participation
5. The child or caregiver has significant mobility issues
6. The child is currently participating in another research study that would preclude participation in the study

Primary outcomes:
measure: Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks
timeFrame: 0, 4, 8, 12, 16 weeks
Secondary outcomes:
measure: Change from baseline level of treatment acceptability at 4, 8, 12 and 16 weeks
timeFrame: 0, 4, 8, 12, 16 weeks
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Department of Psychiatry and Behavioral Sciences
state: California
Study officials:
affiliation: Stanford University
name: Scott S Hall, PhD
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.