Behavioral Therapy for Tuberous Sclerosis

(RT Trial)

Overseen By Jamie Capal, MD

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: University of Rochester

Disqualifiers: Substance use, Psychotic disorders, Schizophrenia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 12

105 Participants Needed

Trial Summary

What is the purpose of this trial?

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).

Will I have to stop taking my current medications?

The trial requires that participants be on a stable medication regimen for at least 4 weeks before joining and not start any new behavior medications during the study.

How is behavioral therapy for tuberous sclerosis different from other treatments?

Behavioral therapy for tuberous sclerosis is unique because it focuses on addressing emotional and behavioral issues, which are common in patients with this condition, rather than just the physical symptoms. This approach is different from standard medical treatments that primarily target the physical manifestations of the disease. ^{1 2 3 4 5}

Research Team

Jamie Capal, MD

Principal Investigator

University of North Carollina at Chapel Hill

Eligibility Criteria

This trial is for children aged 8-17 with Tuberous Sclerosis Complex (TSC) showing emotional issues like tantrums or impulsivity. They need stable internet for remote sessions, speak English, and have an IQ over 65. Kids on steady medication without plans to start new therapies or drugs soon can join.

Inclusion Criteria

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).

I can speak and understand English well enough to participate in treatment sessions.

Child must have a minimum IQ>65 on the WASI-II at the screening/baseline visit).

Exclusion Criteria

My medication has not changed in the last 4 weeks.

I am not planning to begin any new mental health treatments soon.

You have behaviors that could be dangerous or very disruptive to the group, or have serious mental health issues that need other kinds of treatment, or have major trouble seeing or hearing.

Timeline

Screening/Baseline

Participants are screened for eligibility to participate in the trial

1 visit

1 visit (in-person)

RT Control Phase

5-week observational period to establish baseline measures

5 weeks

Remote monitoring

RT Intensive Phase

Remote group treatment intervention twice per week

5 weeks

10 sessions (remote)

RT Individualization Phase

Individualized consultation with participant and caregiver

1 week

1 session (remote)

RT Generalization Phase

Participants utilize learned skills at home, followed by a final interview

10 weeks

Home practice and final interview

Treatment Details

Interventions

• Regulating Together

Trial Overview The study tests 'Regulating Together', a non-drug program delivered remotely to help manage emotional problems in kids with TSC. It's a group intervention aiming to improve behavior without using medications.

Participant Groups

Group I: Parents Experimental Treatment 1 Intervention

In this 22-week almost completely remote study, following a 5-week control phase, parents will receive intensive treatment twice per week for 5 weeks followed by a one-time, 1-on-1 counseling session with an RT psychologist (week 11). Then, parents will use skill practice and application in the home environment for 10 weeks. Following this, a final interview is conducted (week 22).

Group II: Children Experimental Treatment 1 Intervention

In this 22-week almost completely remote study, following a 5-week control phase, children will receive intensive group treatment, twice a week for 5 weeks delivered remotely. During the children's sessions, they will use child structured videos, the PlayPosit curriculum and learning rewards. The following assessments will be completed by the parents, regarding their children, at each phase; EDI, ABC-2, FS, BRIEF-2, CRS, PSI-4SF and CGI-I. Following the completion of group treatment, there will be a one-time, 1-on-1 counseling session with an RT psychologist.

Who Is Running the Clinical Trial?

University of Rochester

Lead Sponsor

Trials

883

Recruited

555,000+

University of North Carolina, Chapel Hill

Lead Sponsor

Trials

1,588

Recruited

4,364,000+

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+

Findings from Research

A significant 63% of adult patients with brain tumors reported elevated stress levels, highlighting the need for effective stress reduction interventions.

While 86% of patients expressed a desire to learn about stress reduction techniques, only a small percentage (25%) were interested in participating in the programs offered, indicating a gap between interest and willingness to engage in stress management activities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stress and intervention preferences of patients with brain tumors. Keir, ST., Guill, AB., Carter, KE., et al.[2022]

A survey of 29 health professionals revealed that 93% found emotional and behavioral disorders in pediatric brain tumor survivors difficult to assess, primarily due to symptom overlap and a lack of appropriate diagnostic frameworks.

All participants expressed a strong interest in developing a new evaluative tool to aid in the diagnosis and treatment of these disorders, highlighting the need for systematic assessment strategies for this vulnerable population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Difficulties encountered by physicians and mental health professionals in evaluating and caring for affective and behavioral problems in pediatric brain tumor survivors. Lopez, C., Sultan, S., Lamore, K., et al.[2021]

A study involving 54 brain tumor patients (28 in inpatient and 26 in outpatient rehabilitation) demonstrated that multidisciplinary rehabilitation significantly improved physical functioning and daily activities after 12 weeks (p < 0.0001).

The rehabilitation also led to significant reductions in cognitive complaints, indicating that early intervention can effectively reduce symptoms and disability associated with brain tumors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Can Multidisciplinary Inpatient and Outpatient Rehabilitation Provide Sufficient Prevention of Disability in Patients with a Brain Tumor?-A Case-Series Report of Two Programs and A Prospective, Observational Clinical Trial. Hojan, K., Gerreth, K.[2020]

Eurythmy therapy (EYT) is a feasible complementary treatment for pediatric survivors of posterior fossa brain tumors, showing good adherence and initial improvements in cognitive and neuromotor functioning after 6 months of therapy.

While improvements were noted in cognitive and neuromotor skills, some benefits in visuomotor integration diminished after 12 months, suggesting the need for ongoing support to maintain these gains.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eurythmy therapy in the aftercare of pediatric posterior fossa tumour survivors--a pilot study. Kanitz, JL., Pretzer, K., Calaminus, G., et al.[2019]

Interventions to manage obesity in survivors of childhood brain tumors (SCBT) show some effectiveness in altering obesity markers and reducing cardiometabolic risk, but the overall evidence is limited and of low quality, based on 11 publications reviewed.

There is an urgent need for well-designed, adequately powered trials to explore both existing and new therapies for obesity management in SCBT to improve their health outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effectiveness of interventions to treat hypothalamic obesity in survivors of childhood brain tumours: a systematic review. Wang, KW., Chau, R., Fleming, A., et al.[2018]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Stress and intervention preferences of patients with brain tumors. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

Difficulties encountered by physicians and mental health professionals in evaluating and caring for affective and behavioral problems in pediatric brain tumor survivors. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Can Multidisciplinary Inpatient and Outpatient Rehabilitation Provide Sufficient Prevention of Disability in Patients with a Brain Tumor?-A Case-Series Report of Two Programs and A Prospective, Observational Clinical Trial. [2020]

4. Scotland pubmed.ncbi.nlm.nih.gov

Eurythmy therapy in the aftercare of pediatric posterior fossa tumour survivors--a pilot study. [2019]

5. England pubmed.ncbi.nlm.nih.gov

The effectiveness of interventions to treat hypothalamic obesity in survivors of childhood brain tumours: a systematic review. [2018]