Clinical trial NCT06105736: Regulating Together in Tuberous Sclerosis Complex
Official title: Regulating Together in Tuberous Sclerosis Complex: A Pilot Feasibility Study in Children and Adolescents With TSC-Associated Neuropsychiatric Disorder (TAND)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: TSC
Behavioral Symptoms
Lead sponsor: University of Rochester
Sponsor class: OTHER
Collaborators: name: United States Department of Defense
First posted: 2023-10-30
Enrollment: 105
Sex: ALL
Minimum age (years): 8
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: DIAGNOSTIC

Brief summary: The purpose of this study is to use a program called Regulating Together (RT), a remote, non-pharmacologic intervention to treat symptoms of emotion dysregulation in children and adolescents with Tuberous Sclerosis Complex (TSC) and TSC-Associated Neuropsychiatric Disorder (TAND).

Detailed description: This is a non-pharmacological intervention that will utilize small group-based therapy with participants, followed by a separate caregiver session. The clinical trial will consist of four phases; 1) single in person screening/baseline visit; 2) RT control phase which is a 5-week observational period (Week 1-5); 3) RT intensive phase which is a remote group treatment intervention twice per week over 5 weeks (Week 6-10); 4) RT individualization phase, which is an individualized consultation with the participant and caregiver (Week 11); and 5) the RT generalization phase which is a 10 week follow up period consisting of utilizing the learned skills at home. At the end of the 10-week generalization phase, a semi-structured interview for thematic analysis will occur at the end of the study to capture facilitators and barriers to the intervention (Week 12-22).

Eligibility criteria: Inclusion Criteria:

* Children ages 8-17 years inclusive with a documented clinical and/or genetic diagnosis of TSC and exhibiting symptoms of behavioral dysregulation (identified on the TAND Checklist having temper tantrums, aggressive outbursts, self-injury, and/or impulsivity) are eligible for inclusion.

Additional inclusion/eligibility criteria include:

* Participants and their caregivers have access to a computer, phone, or tablet with video capability and stable internet connection. If lack of a device or internet service is the sole barrier to eligibility/participation, interested individuals will be connected to a TSC Clinic social worker to link them to existing government and charity programs specifically addressing this disparity in underserved communities and households in need (e.g., the Affordable Connectivity Program).
* Participants must also be willing to participate in treatment sessions and have minimal levels of functional verbal communication (child and their caregiver must be fluent in English
* Child must have a minimum IQ\>65 on the WASI-II at the screening/baseline visit).

Exclusion Criteria:

* Participants be on a stable medication regimen at least 4 weeks prior to enrollment
* Do not have a plan to start a new psychosocial intervention (e.g., individual psychotherapy, family psychotherapy, group psychotherapy or social skills training) or behavior medication within 30 days prior to enrollment or at any point during the study. ----Participants with significant disruptive, aggressive, self-injurious or sexually inappropriate behaviors deemed potentially dangerous or overly disruptive to the group dynamic/session, having significant co-occurring neuropsychiatric illness warranting other treatment approaches as determined by study clinician (e.g., substance use disorders, psychotic disorders, schizophrenia), or having significant sensory impairment that would limit participation in intervention curriculum/materials (e.g., blindness or uncorrected hearing loss) also will be excluded.

Primary outcomes:
measure: Prevalence of ED in TSC Cohort
timeFrame: Baseline
measure: Change in EDI-R Score from Control Phase to RT Intensive and RT Generalization Phases Combined
timeFrame: Intensive Phase (Week 6-10) and Generalization Phase (Week 12-22)
measure: Change in CP-CTI score from pre-treatment to post-treatment
timeFrame: Administered after completion of RT (Week 22)
Secondary outcomes:
Other outcomes:
Locations:
city: Carrboro
country: United States
facility: University of North Carolina Chapel Hill-Carolina Institute for Developmental Disabilities
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center (CCHMC)
state: Ohio
Study officials:
affiliation: University of North Carollina at Chapel Hill
name: Jamie Capal, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Shaffer RC, Schmitt LM, Reisinger DL, Coffman M, Horn P, Goodwin MS, Mazefsky C, Randall S, Erickson C. Regulating Together: Emotion Dysregulation Group Treatment for ASD Youth and Their Caregivers. J Autism Dev Disord. 2023 May;53(5):1942-1962. doi: 10.1007/s10803-022-05461-x. Epub 2022 Feb 9.
citation: Shaffer RC, Wink LK, Ruberg J, Pittenger A, Adams R, Sorter M, Manning P, Erickson CA. Emotion Regulation Intensive Outpatient Programming: Development, Feasibility, and Acceptability. J Autism Dev Disord. 2019 Feb;49(2):495-508. doi: 10.1007/s10803-018-3727-2.