Fragile X SyndromeNCT03510156
Treatment of Disruptive Behaviors in Fragile X Syndrome
A study testing Behavior analytic treatment in people with Fragile X Syndrome and Disruptive Behavior, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 10 years, men only
- Conditions the study is for
- Fragile X Syndrome, Disruptive Behavior
- What is being tested?
- Behavior analytic treatment
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2016to Mar 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Department of Psychiatry and Behavioral Sciences
Stanford, California, 94305
About this study
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.
- Conditions
- Fragile X Syndrome, Disruptive Behavior
- Being tested
- Behavior analytic treatment
- Medical subject headings
- Fragile X Syndrome, Problem Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Behavior analytic treatmentBehavioural
Function-based treatment
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Not described Receives: Behavioral: Behavior analytic treatment |
| Observation | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks
Aberrant Behavior Checklist - Community (ABC-C)
Measured: 0, 4, 8, 12, 16 weeks
Secondary outcomes (1)
Change from baseline level of treatment acceptability at 4, 8, 12 and 16 weeks
Treatment Acceptability Rating Form - Revised (TARF-R)
Measured: 0, 4, 8, 12, 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2018
- Study startJul 2016
- Primary completionMar 2022
- Study completionMar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- The John Merck Fund
- Responsible party
- Principal investigator, Scott Hall, Stanford University
- Investigators
- Scott S Hall, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03510156
- Sponsor's protocol ID
- 36394
- Status last verified
- May 2022
- First submitted
- 17 Apr 2018
- Last updated
- 4 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 36180840Hall 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.
PubMed 33218305
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Disruptive Behaviors in Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT03510156
- Acronym / sponsor ID
- 36394
- Conditions
- Fragile X Syndrome, Disruptive Behavior
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- The John Merck Fund
- Study start
- Jul 2016 (actual)
- Primary completion
- Mar 2022 (actual)
- Study completion
- Mar 2022 (actual)
- First posted
- 27 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Behavioral: Behavior analytic treatment |
| Observation | No intervention | – |
Who may qualify
- Ages
- 3 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Behavior analytic treatmentFunction-based treatment | Behavioural | Treatment |
Primary outcome
Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks
Time frame: 0, 4, 8, 12, 16 weeks
Aberrant Behavior Checklist - Community (ABC-C)
1 secondary outcome
Change from baseline level of treatment acceptability at 4, 8, 12 and 16 weeks
Time frame: 0, 4, 8, 12, 16 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2022.
- Derived from this study: PubMed 36180840Hall 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.
- Derived from this study: PubMed 33218305Hall 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.
- Individual participant dataSharing planned: No
Related trials for Fragile X Syndrome
Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Scott S Hall, PhD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Behavior analytic treatment in people with Fragile X Syndrome and Disruptive Behavior, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline level of problem behavior at 4, 8, 12 and 16 weeks (time frame: 0, 4, 8, 12, 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03510156, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 May 2022.