Regulating Together in Tuberous Sclerosis Complex

Behavioral Symptoms Clinical Trial

— RT  

Official title:

Regulating Together in Tuberous Sclerosis Complex: A Pilot Feasibility Study in Children and Adolescents With TSC-Associated Neuropsychiatric Disorder (TAND)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06105736
• Other study ID #: 23-0274
• Secondary ID: HT9425-23-1-0344
• Status: Recruiting
• Phase: N/A
• Start date: December 20, 2023
• Est. completion date: December 20, 2026

Study information

• Verified date: January 2024
• Source: University of North Carolina, Chapel Hill
• Contact: Jamie Capal, MD
• Phone: (919) 966-9343
• Email: jamie.capal[@]cidd.unc.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 105
• Est. completion date: December 20, 2026
• Est. primary completion date: December 20, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Behavioral Symptoms
• TSC
• Tuberous Sclerosis

Intervention

Behavioral:
• Behavioral Intervention in a small group platform
RT is small-group-based, intensive behavioral intervention session delivered twice weekly over a secure remote platform for 60-minutes over 5 weeks. Each session is led by a psychologist trained and certified in RT. Caregiver groups meet at the same frequency and duration but at different times. Each session has a specific focus for teaching participants to identify emotions and incorporation of cognitive behavioral therapy (CBT) and mindfulness skills that are reinforced through repeated practice. Each session will include a review, relaxation, new material didactic, activities to reinforce material, mindfulness, and homework.

Locations

Country	Name	City	State
United States	University of North Carolina Chapel Hill-Carolina Institute for Developmental Disabilities	Carrboro	North Carolina
United States	Cincinnati Children's Hospital Medical Center (CCHMC)	Cincinnati	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
University of North Carolina, Chapel Hill	United States Department of Defense

Country where clinical trial is conducted

United States, 

References & Publications (2)

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: 10.1 — View 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/s10803-018-3 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence of ED in TSC Cohort	Prevalence of emotion dysregulation is highly prevalent in TSC patients who exhibit problematic behaviors. The EDI-R score at the screening study visit will determine the EDI-R and determine the presence or absence of emotion dysregulation in the cohort.	Baseline
Primary	Change in EDI-R Score from Control Phase to RT Intensive and RT Generalization Phases Combined	EDI-R score will be compared from pre-treatment (control phase) to post-treatment (RT Intensive Phase and RT Generalization Phases combined). EDI-R is a caregiver administered, emotion dysregulation inventory with construct, range, and directionality that includes questions that the rater is asked to answer based on behaviors that are observed to cause problems. These questions include, but are not limited to, creating a dangerous situation, interference with daily activities, increasing the stress of those around the individual, and hurting relationships. There are 13 questions on the EDI-R; they are rated at, not at all, mild, moderate, severe, and very severe.	Intensive Phase (Week 6-10) and Generalization Phase (Week 12-22)
Primary	Change in CP-CTI score from pre-treatment to post-treatment	The primary endpoint will be CP-CTI responses from caregivers upon completion of RT Generalization Phase using a thematic analysis. This will identify obstacles to treatment access. Scores will be compared pre-treatment to post-treatment. CP-CTI is a staff administered, caregiver perspective clinical trial interview. This will occur through a telephone call from the study team to the caregiver.	Administered after completion of RT (Week 22)