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

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test a program called Regulating Together (RT), a remote therapy designed to help children and teens with Tuberous Sclerosis Complex (TSC) manage emotional challenges and related behavioral issues. Over 22 weeks, participants will engage in group sessions and receive guidance from a psychologist. The program suits kids aged 8-17 who have TSC and struggle with mood swings or aggressive behavior. Participants need a device with internet access and must be fluent in English. As an unphased trial, this study offers a unique opportunity to explore innovative therapy methods for managing TSC-related challenges.

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.

What prior data suggests that the Regulating Together program is safe for children and adolescents with Tuberous Sclerosis Complex?

Research has shown that the treatment, Regulating Together (RT), is a non-drug method designed to help manage emotional challenges in children and teens with Tuberous Sclerosis Complex (TSC). As a type of behavioral therapy, RT doesn't use medications, eliminating concerns about drug-related side effects.

Although specific safety data for RT isn't available, its non-drug nature suggests it is likely safe. This therapy involves exercises and activities, making it a safe choice. Participants in similar programs often report positive results without major issues.

The trial is labeled as "Not Applicable" in terms of phases, indicating it tests a behavioral method rather than a new drug. This suggests the treatment is already considered safe for use. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it explores a novel approach to managing Tuberous Sclerosis through behavioral therapy. Unlike traditional treatments that often focus on medication or surgery, this method emphasizes remote, structured group sessions for both children and parents. The use of interactive tools like PlayPosit and personalized, one-on-one sessions with psychologists are designed to enhance learning and behavior management. This approach not only aims to improve symptoms but also empowers families by integrating therapy into their home environment, potentially leading to more lasting benefits.

What evidence suggests that the Regulating Together program is effective for emotion dysregulation in Tuberous Sclerosis?

Research has shown that the "Regulating Together" program is a promising non-drug treatment designed to help children and teens with Tuberous Sclerosis Complex (TSC) manage their emotions better. In this trial, children will participate in intensive group treatment sessions, while parents will attend separate intensive sessions. Although detailed results about the program's success aren't widely available yet, similar programs have shown promise in helping with emotional control in other conditions. The program teaches skills to both children and their parents, creating a supportive environment for handling emotional challenges. Early findings suggest that structured, skill-based approaches can significantly aid in managing symptoms. While more solid evidence is still needed, the program is based on methods that have proven helpful in similar areas. ^{1 2 6 7 8}

Who Is on the Research Team?

Jamie Capal, MD

Principal Investigator

University of North Carollina at Chapel Hill

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06105736?term=AREA%5BBasicSearch%5D(AREA%5BConditionSearch%5D(%22Behavioral%20Symptoms%22))&rank=1

Regulating Together in Tuberous Sclerosis Complex (RT) The purpose of this study is to use a program called Regulating Together (RT), a remote, non-pharmacologic intervention to treat symptoms of emotion ...

2. tscalliance.org tscalliance.org/tsc-matters/tsc-and-emotion-dysregulation-study-for-children-8-to-18-years-old-and-their-caregivers/

TSC and Emotion Dysregulation Study for Children 8 to 18 ... A study to learn more about the effectiveness of a Tuberous Sclerosis Complex (TSC) treatment program called “Regulating Together.” This study will be ...

3. trialx.com trialx.com/clinical-trials/listings/237901/regulating-together-in-tuberous-sclerosis-complex/

Regulating Together in Tuberous Sclerosis Complex The purpose of this study is to use a program called Regulating Together (RT), a remote, non-pharmacologic intervention to treat symptoms of ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7431762/

Autism and Epilepsy in Patients With Tuberous Sclerosis ... Data coming from the EXIST-3 trial confirm that adjunctive everolimus might reduce seizure frequency in pediatric patients with treatment- ...

5. answers.childrenshospital.org answers.childrenshospital.org/rapamycin-autism-tuberous-sclerosis/

Earlier treatment may help reverse autism-like behavior in ... But when a similar drug, everolimus, was tested in children with TSC, neurocognitive functioning and behavior didn't significantly improve.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3715752/

Targeted treatment trials for tuberous sclerosis and autism This review summarizes the advantages of using TSC as model of ASD and the recent advances in the translational and clinical treatment trials in TSC.

7. cdmrp.health.mil cdmrp.health.mil/tscrp/pbks/TSCRP%20Program%20Book_web%20version_8Jan24.pdf

Tuberous Sclerosis Complex Research Program (FY22). Regulating Together in Tuberous Sclerosis Complex: A Pilot. Feasibility Study in Children and Adolescents with TSC-Associated.

8. sciencedirect.com sciencedirect.com/science/article/pii/S1878747923016045

Tuberous Sclerosis: A New Frontier in Targeted Treatment ... A study of early developmental trajectories in children with TSC and ASD found a trend towards more severe epilepsy in patients with TSC with ASD, as well as a ...