Acceptance and Commitment Therapy in Tuberous Sclerosis Complex (ACT in TSC)

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Tuberous Sclerosis Complex
• Intervention / Treatment: Behavioral: Acceptance and Commitment Therapy (ACT)

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Bristol, United Kingdom
    • University Hospitals Bristol and Weston NHS Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Aged 11 to 24 years.
• Diagnosis of Tuberous Sclerosis Complex.
• Sufficient understanding of English to engage with the intervention (spoken and written), as judged by the assessing clinician.
• Sufficient cognitive, sensory and speech capabilities to take part in the intervention.
• Participants (or their parents if under 16) give verbal or written informed consent to participate in the study.
• Participants give verbal or written assent if under 16.
• Receiving treatment over video-conferencing will be the default modality for delivery, so access to the internet is a requirement.

Exclusion Criteria:

• Previous structured behavioural intervention within last 6 months.
• Previous or current alcohol/substance dependence, psychosis, suicidality, or anorexia nervosa.
• Moderate or severe intellectual disability.
• Immediate risk to self or others.
• Parent or child not able to speak, read or write English.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Allocated to treatment; 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.	Behavioral: Acceptance and Commitment Therapy (ACT); 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.
No Intervention: Allocated to waitlist control; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The credibility/expectancy questionnaire	Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.	Assessed at baseline
The credibility/expectancy questionnaire	Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.	Assessed at session 2
Treatment completion rate	The proportion of patients showing interest who then consent to the trial and complete the intervention.	Assessed at 3-month follow-up.
Treatment completion rate	The proportion of patients showing interest who then consent to the trial and complete the intervention.	Assessed at 6-month follow-up
Session attendance rate	The session attendance rate compared to feasibility benchmarks.	Assessed at 3-month follow-up
Session attendance rate	The session attendance rate compared to feasibility benchmarks.	Assessed at 6-month follow-up
The experience of service questionnaire	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.	Assessed at 3-month follow-up
The experience of service questionnaire	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.	Assessed at 6-month follow-up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acceptance and Action Questionnaire II	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.	Assessed at 3, 6 and 12-month follow-up.
Avoidance and Fusion Questionnaire for Youth 8-items	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.	Assessed at 3, 6 and 12-month follow-up.
World Health Organisation wellbeing index 5-items	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.	Assessed at 3, 6 and 12-month follow-up.
Generalised Anxiety Disorder assessment 7-items	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.	Assessed at 3, 6 and 12-month follow-up.
Patient Health Questionnaire 9-items	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.	Assessed at 3, 6 and 12-month follow-up.
Client Service Receipt Inventory	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.	Assessed at 3, 6 and 12-month follow-up.
TAND - TSC Associated Neuropsychiatric Disorders	Assesses behavioural, intellectual, learning and psychiatric impact of TSC.	Assessed at 3, 6 and 12-month follow-up.
Pediatric Quality of Life Inventory (PedsQL)	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	Assessed at 3, 6 and 12-month follow-up.
Experiential interviews	Participant experiences of treatment as described in semi-structured qualitative interviews	Assessed at 3- and 6-month follow-up

Collaborators and Investigators

• Sponsor: University Hospitals Bristol and Weston NHS Foundation Trust
• Collaborators: University College, London; The Tuberous Sclerosis Association
• Investigators: Principal Investigator: Sam Amin, MBCHB MSc PhD, University Hospitals Bristol & Weston NHS Trust

Study record dates

Study Major Dates

• Study Start (Actual): August 12, 2021
• Primary Completion (Estimated): March 30, 2024
• Study Completion (Estimated): March 30, 2024

Study Registration Dates

• First Submitted: May 21, 2021
• First Submitted That Met QC Criteria: May 10, 2023
• First Posted (Actual): May 22, 2023

Study Record Updates

• Last Update Posted (Actual): February 14, 2024
• Last Update Submitted That Met QC Criteria: February 13, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Acceptance and Commitment Therapy; Mental Health; Tuberous Sclerosis Complex; Paediatrics; Neurology; Psychology; Remote therapy; TAND
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neoplasms; Congenital Abnormalities; Genetic Diseases, Inborn; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Neurocutaneous Syndromes; Hamartoma; Neoplasms, Multiple Primary; Sclerosis; Tuberous Sclerosis
• Other Study ID Numbers: CH/2020/7042

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No