Symptoms and General Pathology

All Conditions

Behaviors and Mental Disorders

Neoplasms

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Problem Behavior

Tuberous Sclerosis

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Behavioral Symptoms

Tuberous Sclerosis Complex

Participants randomized to the waitlist control (or delayed treatment) condition will receive written and verbal feedback after the baseline assessment visit. These families will participate in follow-up visits at 3 months, 6 months, and 12 months after the baseline visit. They will receive the same treatment (PCIT) after the 6-month assessment.

Participants randomized to the immediate treatment condition will receive written and verbal feedback after the baseline assessment visit. These families will begin PCIT shortly after this visit. These families will participate in follow-up visits at 3 and 6 months after the baseline visit.

PCIT is a dyadic therapy that focuses on enhancing the parent-child relationship and improving child behavior through positive parenting and limit setting skills. All PCIT sessions are delivered remotely via Zoom once per week for up to 20 weeks. Session length is about 1 hour. There are two distinct phases of PCIT, the Child Directed Interaction and the Parent Directed Interaction. Each phase begins with a Teach session, where parents learn about the skills they will practice during that portion of PCIT, and is followed by Coach sessions, in which the PCIT therapist provides direct feedback and live coaching on the use of the skills. An additional component is Daily Play practice. Parents are instructed to spend five minutes per day playing with their child, during which they practice skills learned during sessions. Adaptations have been made to the standard approach to increase suitability for the TSC population.

Parent-Child Interaction Therapy (PCIT)

Viviana Rodriguez

Nicole McDonald, PhD

Nicole McDonald

The RAINBOW study is a fully remote study focused on understanding and treating behavior problems in young children with tuberous sclerosis complex (TSC). The first goal is to enroll a group of 100 children with TSC between the ages of 3 and 6 years old, with and without problem behaviors, to learn about how best to measure behavioral challenges in TSC and how common these behaviors are during this age period. All families will get feedback from the clinical assessments collected. Eligible children who are experiencing behavior problems will be invited to enroll in a pilot clinical trial of internet-based Parent-Child Interaction Therapy (or PCIT), which is meant to help parents better manage their children's challenging behaviors. Adaptations to PCIT are incorporated to suit the needs of TSC children and families. Some participants will be randomly selected to receive the intervention immediately and others after a 6-month delay, but all families will receive the same intervention. Intervention sessions occur on a weekly basis for up to 20 weeks. Follow-up assessments, which include play interactions, parent interviews, and questionnaires occur about 3 and 6 months after the first visit. The delayed treatment group has an extra brief assessment about 12 months from the initial visit. This study will increase what is known about the types of behavior problems that come up during preschool age in TSC and how best to help children and families with TSC who are experiencing these problems.

The proposed study uses a telehealth approach to remotely assess and treat behavior problems in preschool-aged children with TSC. This study aims to 1) measure the prevalence and quality of externalizing behavior problems and their relation to family functioning in preschool-aged children with TSC; 2) examine the feasibility and acceptability of internet-based PCIT (I-PCIT) in children with TSC; 3) investigate the effects of I-PCIT on child and parent behavior in preschool-aged children with TSC and behavior problems; and 4) examine predictors of treatment response.

For Aim 1, 100 children with TSC ages 3-6 years old will be enrolled. Developmental abilities and behavior problems will be assessed using norm-referenced parent report measures and video recordings of parent-child interactions. The prevalence of behavior problems in young children with TSC and relation to family functioning will be assessed. A subset of the children from Aim 1 who exhibit elevated externalizing behavior will be recruited for the pilot intervention, with a target sample size of 32. Children will be randomized to an immediate treatment or waitlist control group. Assessments will be performed at baseline (using Aim 1 data), mid-point (3 months later), and post-treatment (6 months later). Families randomized to the waitlist group will have a brief, additional post-treatment assessment 12 months post-baseline). Sessions occur on a weekly basis for up to 20 weeks. Preliminary efficacy will be examined by comparing trajectories from baseline to post-treatment between the immediate treatment and waitlist control groups. Data on feasibility, acceptability, and predictors of treatment response will be studied across all intervention participants.

The clinical trial portion of this study uses a waitlist control design with random assignment to group. Randomization is stratified by age (3-4 years old, 5-6 years old).

Outcome assessments will be performed by assessors who are blind to group status. Investigators will be kept blind to study outcomes until data collection for the primary outcome is complete.

Remote Assessment and Intervention for Behavior Problems in Kids With TSC

Assessment and Treatment of Behavior Problems in TSC at Preschool Age: A Telehealth Approach

The ECBI is a norm-referenced caregiver report measure of disruptive behavior in children.

The BASC-3 is a norm-referenced caregiver report measure of broad behavior problems and adaptive skills in children.

The Vineland-3 is a norm-referenced caregiver report measure of adaptive skills for infancy through adulthood. Communication, daily living, socialization, and motor skills are measured. The parent interview version is used for this study.

The PSI-4-SF is a norm-referenced self-report measure of parenting-related stress.

The DPICS measures parent-child interactions across 3 situations: child led play, parent led play, and clean up. Language and behavior are coded according to the DPICS-IV coding system.

The TAI measures treatment acceptability in PCIT.

The TUQ measures comfort with the use of telehealth methods pre- and post-treatment.

The ABC is a caregiver report questionnaire commonly used to measure problem behaviors and behavior change in children and adults with disabilities.

University of California, Los Angeles

Assistant Clinical Professor

Central Nervous System Depressants

All Drugs and Chemicals

Other Dietary Supplements

Benzocaine

Tannic Acid

Patients randomly allocated to this arm will receive treatment immediately. They will receive 6-12 sessions of ACT while patients allocated to the waitlist control arm do no receive any treatment.

Patients randomly allocated to this arm will receive a delay in the treatment they receive. After a 12-week wait they will receive 6-12 sessions of ACT, while patients allocated to the immediate treatment arm receive no treatment for an equal duration.

Acceptance and commitment therapy (ACT) is an evidence-based psychological therapy that has been successfully used to improve physical and mental health among children and adults with chronic conditions. It is a "third wave" cognitive behavioural therapy that encourages openness to and awareness of the present moment in order to help participants maintain behaviours consistent with their life goals. ACT fosters engagement with, rather than avoidance of, painful experiences to move towards acceptance of unchangeable difficulties alongside building a rich and meaningful life despite the presence of ongoing difficulties. This gives ACT strong face validity for application to TCS patients where there can be permanent cognitive impairment and unavoidable ongoing physical symptoms and functional limitations.

Acceptance and Commitment Therapy (ACT)

Sam Amin, MBCHB MSc PhD

The study will assess the acceptability and feasibility of a randomised controlled trial of 6-12 sessions of remotely-delivered Acceptance and Commitment Therapy (ACT) versus waitlist control. Waitlist control will involve a delay in the offer of ACT sessions for 12 weeks. Participants may access all services as usual in this time. Follow-up assessments will be conducted at 12-, 24 and 48 weeks post-randomisation to measure effectiveness.

Tuberous Sclerosis Complex (TSC) is a genetic disease caused by mutations in the tumour suppressor genes TSC1 and TSC2. The clinical hallmarks of the disease are the growth of benign tumours in various organs such as the brain, kidneys and skin. Seizures are present in around 60% of the population and neurodevelopmental problems such as attention deficit disorder and autism are common. Anxiety and depressive disorders are similarly linked and at the psychosocial level, there is increasing evidence of the effect of TSC on self-esteem, family functioning and peer relationships resulting in poorer quality of life. Despite these difficulties, no unique treatments, and almost no effective evidenced psychological treatments for TSC are available.

This trial aims to assess the feasibility and acceptability of Acceptance and Commitment Therapy (ACT) as a psychological treatment to improve quality of life among adolescents and young adults with TSC. ACT is a cognitive behavioural therapy that helps participants accept difficulties that they are unable to change. There is strong evidence for ACT's clinical effectiveness amongst patients with chronic diseases. The intervention will be delivered to participants aged 11-24 with TSC and sufficient cognitive and speech capabilities to take part. This will be delivered remotely via secure video-conferencing software. Our primary hypothesis is that ACT will be acceptable and feasible delivered remotely and may yield clinical improvements in health and quality of life.

The study will be a 12-week, waitlist controlled randomised clinical trial. Participants will be randomised to receive 12-weeks treatment either immediately or following a 12-week wait. The treatment will be ACT adapted for 11-24-year olds who have TSC. Treatment will involve 6 to 12 weekly sessions of ACT of up to one hour each in length. Clinical outcomes will be assessed unblinded at baseline, 12, 24 and 48 weeks from randomisation. As a feasibility and acceptability study a range of physical and mental health outcomes are assessed. All clinical outcomes focus on health, wellbeing and quality of life from baseline to 12 week (3-month) follow-up amongst those offered ACT immediately versus waitlist controls.

Randomised controlled trial of 6-12 sessions of ACT versus waitlist control. Waitlist control will involve a delay in the offer of ACT sessions for 12 weeks.

The study design is an unblinded, waitlist-controlled, randomised controlled trial (RCT). Randomisation will be conducted using established randomisation software RedCap. Researchers will not be blind to trial arm allocation.

Acceptance and Commitment Therapy in Tuberous Sclerosis Complex

Randomised Controlled Trial of Acceptance and Commitment Therapy in Tuberous Sclerosis Complex

The proportion of patients showing interest who then consent to the trial and complete the intervention.

The session attendance rate compared to feasibility benchmarks.

Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.

A questionnaire used nationally in child and adolescent mental health services completed by patients and parents/carers (if under 16) to assess participants' experience of the intervention. An additional item has been added to the experience of service questionnaire to assess video-conferencing treatment satisfaction. Minimum score = 0; maximum score = 20. Higher scores indicate better outcome.

A brief 7-item self-report measure assessing psychological inflexibility, which is the central treatment target for ACT. Validated for use among patients aged 16 and over. Minimum score = 7; maximum score = 49. Higher scores indicate worse outcome.

A brief self-report measure of psychological inflexibility, for children (11 to 15 year old participants). Minimum score = 0; maximum score = 32. Higher scores indicate worse outcome.

A brief self-reported assessment of wellbeing and mental health. Minimum score = 7 score; maximum = 49. Higher scores indicate worse outcome. Minimum score = 0; maximum score = 49. Higher scores indicate worse outcome.

A brief self-reported measure of generalised anxiety symptoms using 4-point Likert scales based on diagnostic criteria. Minimum score = 0; maximum score = 21. Higher scores indicate worse outcome.

Assesses symptoms of depression using self-reported 4-point Likert scales based on diagnostic criteria for major depression. Minimum score = 0; maximum score = 27. Higher scores indicate worse outcome.

A research instrument developed to collect information on service receipt, service-related issues and income. In addition, the Client Service Receipt Inventory collects information on school attendance in the 3-months prior to assessment.

Assesses behavioural, intellectual, learning and psychiatric impact of TSC.

A 23-item generic health status instrument with parent and child forms that assesses five domains of health (physical functioning, emotional functioning, psychosocial functioning, social functioning, and school functioning) in children and adolescents

Participant experiences of treatment as described in semi-structured qualitative interviews

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Remote Assessment and Intervention for Behavior Problems in Kids With TSC	NCT06311474	Tuberous Sclero...	Parent-Child In...	3 Years - 6 Years	University of California, Los Angeles
Acceptance and Commitment Therapy in Tuberous Sclerosis Complex	NCT05867576	Tuberous Sclero...	Acceptance and ...	11 Years - 24 Years	University Hospitals Bristol and Weston NHS Foundation Trust

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Spots Global Cancer Trial Database for tand

Every month we try and update this database with for tand cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for tand cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.