Tuberous Sclerosis ComplexACT in TSCNCT05867576
Acceptance and Commitment Therapy in Tuberous Sclerosis Complex
A study testing Acceptance and Commitment Therapy (ACT) in people with Tuberous Sclerosis Complex, run by University Hospitals Bristol and Weston NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 11 to 24 years
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- Acceptance and Commitment Therapy (ACT)
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 12 Aug 2021to 30 Mar 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 11 to 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
University Hospitals Bristol and Weston NHS Trust
Bristol
About this study
Official title: 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.
Read the 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.
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- Acceptance and Commitment Therapy (ACT)
- Keywords
- Tuberous Sclerosis Complex, Acceptance and Commitment Therapy, Mental Health, Psychology, Paediatrics, Neurology, Remote therapy, TAND
- Medical subject headings
- Tuberous Sclerosis, Psychological Well-Being
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acceptance and Commitment Therapy (ACT)Behavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Allocated to treatment | Active comparator | 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. Receives: Behavioral: Acceptance and Commitment Therapy (ACT) |
| Allocated to waitlist control | No intervention | 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 being measured
The primary outcome is the main question the study is designed to answer.
Treatment completion rate
The proportion of patients showing interest who then consent to the trial and complete the intervention.
Measured: 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.
Measured: Assessed at 6-month follow-up
Session attendance rate
The session attendance rate compared to feasibility benchmarks.
Measured: Assessed at 3-month follow-up
Session attendance rate
The session attendance rate compared to feasibility benchmarks.
Measured: Assessed at 6-month follow-up
The credibility/expectancy questionnaire
Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.
Measured: Assessed at baseline
The credibility/expectancy questionnaire
Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.
Measured: Assessed at session 2
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.
Measured: 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.
Measured: Assessed at 6-month follow-up
Secondary outcomes (9)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Assessed at 3, 6 and 12-month follow-up.
TAND - TSC Associated Neuropsychiatric Disorders
Assesses behavioural, intellectual, learning and psychiatric impact of TSC.
Measured: 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
Measured: Assessed at 3, 6 and 12-month follow-up.
Experiential interviews
Participant experiences of treatment as described in semi-structured qualitative interviews
Measured: Assessed at 3- and 6-month follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2023
- Study start12 Aug 2021
- Primary completion30 Mar 2024 (estimated)
- Study completion30 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospitals Bristol and Weston NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- The Tuberous Sclerosis Association, University College, London
- Responsible party
- Sponsor
- Investigators
- Sam Amin, MBCHB MSc PhD, Principal investigator, University Hospitals Bristol & Weston NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05867576
- Sponsor's protocol ID
- CH/2020/7042
- Acronym
- ACT in TSC
- Status last verified
- Feb 2024
- First submitted
- 21 May 2021
- Last updated
- 14 Feb 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomised Controlled Trial of Acceptance and Commitment Therapy in Tuberous Sclerosis Complex
- ClinicalTrials.gov ID
- NCT05867576
- Acronym / sponsor ID
- ACT in TSC / CH/2020/7042
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- Tuberous Sclerosis Complex, Acceptance and Commitment Therapy, Mental Health, Psychology, Paediatrics, Neurology, Remote therapy, TAND
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- University Hospitals Bristol and Weston NHS Foundation TrustUniversity, hospital or other
- Collaborators
- The Tuberous Sclerosis Association, University College, London
- Study start
- 12 Aug 2021 (actual)
- Primary completion
- 30 Mar 2024 (estimated)
- Study completion
- 30 Mar 2024 (estimated)
- First posted
- 22 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Allocated to treatment | Active comparator | Behavioral: Acceptance and Commitment Therapy (ACT)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. |
| Allocated to waitlist control | No intervention | –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. |
Who may qualify
- Ages
- 11 to 24 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Behavioural | Allocated to treatment |
Primary outcome
Treatment completion rate
Time frame: Assessed at 3-month follow-up.
The proportion of patients showing interest who then consent to the trial and complete the intervention.
Treatment completion rate
Time frame: Assessed at 6-month follow-up
The proportion of patients showing interest who then consent to the trial and complete the intervention.
Session attendance rate
Time frame: Assessed at 3-month follow-up
The session attendance rate compared to feasibility benchmarks.
Session attendance rate
Time frame: Assessed at 6-month follow-up
The session attendance rate compared to feasibility benchmarks.
The credibility/expectancy questionnaire
Time frame: Assessed at baseline
Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.
The credibility/expectancy questionnaire
Time frame: Assessed at session 2
Assessing participant ratings of treatment credibility. Minimum score = 5; maximum score = 45. Higher scores indicate better outcome.
The experience of service questionnaire
Time frame: Assessed at 3-month follow-up
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.
The experience of service questionnaire
Time frame: Assessed at 6-month follow-up
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.
9 secondary outcomes
Acceptance and Action Questionnaire II
Time frame: Assessed at 3, 6 and 12-month follow-up.
Avoidance and Fusion Questionnaire for Youth 8-items
Time frame: Assessed at 3, 6 and 12-month follow-up.
World Health Organisation wellbeing index 5-items
Time frame: Assessed at 3, 6 and 12-month follow-up.
Generalised Anxiety Disorder assessment 7-items
Time frame: Assessed at 3, 6 and 12-month follow-up.
Patient Health Questionnaire 9-items
Time frame: Assessed at 3, 6 and 12-month follow-up.
Client Service Receipt Inventory
Time frame: Assessed at 3, 6 and 12-month follow-up.
TAND - TSC Associated Neuropsychiatric Disorders
Time frame: Assessed at 3, 6 and 12-month follow-up.
Pediatric Quality of Life Inventory (PedsQL)
Time frame: Assessed at 3, 6 and 12-month follow-up.
Experiential interviews
Time frame: Assessed at 3- and 6-month follow-up
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sam Amin, MBCHB MSc PhD principal investigator
University Hospitals Bristol & Weston NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Acceptance and Commitment Therapy (ACT) in people with Tuberous Sclerosis Complex, run by University Hospitals Bristol and Weston NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 14 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment completion rate (time frame: Assessed at 3-month follow-up.). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2024.