Nervous System Diseases

Symptoms and General Pathology

All Conditions

Neoplasms

Ataxia

Cerebellar Ataxia

Infratentorial Neoplasms

Dyskinesias

Neurologic Manifestations

Cerebellar Diseases

Brain Diseases

Central Nervous System Diseases

Brain Neoplasms

Central Nervous System Neoplasms

Nervous System Neoplasms

Intervention therapist at 2 centres providing 8 week intervention programme 50% embedded qualitative study

Participants continue with usual care and existing therapy recorded in study diary

Therapist

Helen Hartley

Research Alder Hey

The overall aim of the study is to determine the feasibility of conducting a randomised controlled trial (RCT) studying the effectiveness of physiotherapy intervention (virtual training) in children with ataxia following surgical resection of posterior fossa tumour

Brain tumours are the most common group of solid tumours in children accounting for nearly a quarter of all childhood cancers. There are approximately 500 new cases of central nervous system (CNS) tumours in children/adolescents reported in the UK per year. Although prognosis has improved over the last 30 years, brain tumours remain the leading cause of tumour-associated death in children. Surgical resection is a mainstay of management of children with brain tumours, as for several tumour types there is strong evidence that survival and progression free survival are influenced by the degree of resection. Therefore extensive tumour removal is an operative goal, but the morbidity of surgery along with any subsequent oncological treatment should also be considered. The NICE guidelines for improving outcomes in children and young people with cancer identify that 'Survivors of CNS malignancy are among the neediest of all cancer survivors, because of the effects of the tumour and multimodality therapy, all of which affect neurological, psychological, endocrine and academic function and become more evident with increasing age'.

Children with posterior fossa tumours (PFT), which account for approximately 50% of all childhood brain tumours, have a distinctive set of issues e.g. potential for gross change pre/post operatively, rapid onset of ataxia, hydrocephalus and increased intra-cranial pressure adding deficits distinct from ataxia, in addition to potential problems from any subsequent oncological management such as radiotherapy. Of these issues, ataxia is the predominant motor problem in children with posterior fossa tumours. Ataxia can describe a related number of impairments including upper limb control, balance, gait difficulties, eye movement issues and speech problems. It is a presenting sign in 58-90% of children with posterior fossa tumours. Ataxia and balance problems also persist long term following surgery, Piscione et al found 70% of children with posterior fossa tumours will have long term post-operative balance problems. Lannering et al specified that truncal ataxia was the most disabling motor impairment in children with brain tumours.

Access to neuro-rehabilitation is recognized as crucial in paediatric neuro-oncology with physiotherapy integral to this; yet there is no consensus as to the type, intensity or timing of interventions. A literature review of physical therapy/physiotherapy for children with ataxia (of any origin) was carried out in preparation for this project to confirm the literature gap, and consider interventions that have been of value in other pathologies causing ataxia. Ten papers in total were identified which included three review papers that covered adult and paediatric literature though the data were not separated in the studies to enable consideration of paediatrics as a distinct group. Overall the reviews concurred there was a suggestion of the benefit of physiotherapy (including treadmill training, rehabilitation, virtual training) though treatment modalities were often not consistently defined and evidence was typically of low quality (level III/IV). The recommendations from these reviews are that the next step would be to undertake RCTs (randomised controlled trials) in more homogenous patient groups. Looking at the paediatric literature, there were three case studies identified (and one case series with a population of three adolescents) again in differing diagnoses though none were examining children with brain tumours that is the most common acute cause for ataxia in the paediatric population. The largest paediatric study (n=10) identified through the literature review was a cohort study by Ilg et al evaluating the effect of virtual training. Virtual training refers to the use of computer technologies that provide an interactive environment that requires limb movement to react to on screen game play. Ilgs study found positive results and proposed that virtual training should then complement/supplement therapy treatment tailored according to the individual and continued in the home environment. Virtual training has also been explored in other paediatric populations e.g. cerebral palsy.

Overall the literature review highlighted that there is a gap in the literature regarding physiotherapy intervention for children with ataxia, in particular there is no literature on effectiveness of physiotherapy for children with brain tumours. However, there is an emerging evidence base in adults with ataxia and to a lesser extent paediatric populations as described above, from similar (though non acute) lesions in the cerebellum reporting the benefits of balance therapies including virtual training. This suggests the potential for recovery from ataxia in similar conditions i.e. children with damage to the cerebellum following surgical resection of posterior fossa tumour.

Participants will be randomised to one of 2 groups 1. Intervention arm 2. Usual standard of care.

The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour

The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour (ASPECT)

Number of patients identified who meet the inclusion criteria and are then subsequently recruited will be recorded.

Number of patients who complete all sessions of the intervention will be recorded and the patient's adherence to treatment in the home setting will be noted.

Scale for the Assessment and Rating of Ataxia

Paediatric Evaluation of Disability Index

Inventory of Non Ataxia Signs. Assessment of compliance (diary)

Alder Hey Children's NHS Foundation Trust

Diseases and Abnormalities at or Before Birth

Musculoskeletal Diseases

Heart and Blood Diseases

Skin and Connective Tissue Diseases

Behaviors and Mental Disorders

Rare Diseases

Syndrome

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Noonan Syndrome

Costello Syndrome

Nerve Sheath Neoplasms

Neoplasms, Nerve Tissue

Neoplasms by Histologic Type

Neoplastic Syndromes, Hereditary

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Peripheral Nervous System Diseases

Neuromuscular Diseases

Congenital Abnormalities

Craniofacial Abnormalities

Musculoskeletal Abnormalities

Heart Defects, Congenital

Cardiovascular Abnormalities

Heart Diseases

Connective Tissue Diseases

Abnormalities, Multiple

Quality of Life

Neurofibromatosis

Neurofibromatosis Type 1

Cardiofaciocutaneous Syndrome

Legius Syndrome

Participants will begin their 1-week baseline EMA data collection period and then take part in intervention procedures immediately after enrollment to assess feasibility and acceptability.

Participants will begin their 1-week baseline EMA data collection period immediately after randomization and will begin receiving the 8-week intervention at the end of this week/beginning of the next week (depending on participant schedule).

After an initial 1-week baseline period for EMA data collection and 8 weeks of maintaining their usual routine (wait list period), participants will begin receiving the 8-week intervention.

8 weeks of maintaining usual routine followed by 8-week intervention that emphasizes mindfulness, acceptance, perspective taking, and values-based actions.

Waitlist

8-week intervention that emphasizes mindfulness, acceptance, perspective taking, and values-based actions.

ACT Intervention

Staci M Peron, Ph.D.

Staci Peron, Ph.D.

Background:

RASopathies are a group of genetic diseases that affect a child s development. They cause physical, cognitive, and behavioral symptoms. Caring for a child with a RASopathy can be stressful. Acceptance and Commitment Therapy (ACT) is a therapy that helps people become more aware and accepting of difficult thoughts and feelings. ACT has been found to be helpful for parents with high parenting stress.

Objective:

To find out if Acceptance and Commitment Therapy (ACT) can help caregivers of children with a RASopathy better cope with parenting stress.

Eligibility:

People aged 18 years or older who care for a child (younger than 18 years) with a RASopathy. The child must live with the caregiver at least 50% of the time.

Design:

The study is fully remote. Participants need a mobile device that can play audio and video and connect to the internet. They can borrow an iPod if needed.

Participants will download a free app called MetricWire. They will use this app to watch videos and answer questions.

The first 8 participants will be in a pilot study. They will receive the ACT intervention starting the first week after they begin the study.

After the pilot study, we will start a new phase called the randomized trial. In this phase, participants will have a 50-50 chance of being in the group that will start the intervention right away or the group that will start the intervention after about 2 months.

Participants will fill out surveys on 5 random days each week. These surveys have 7 questions and take about 2 minutes. They will also fill out 3 longer questionnaires: once before ACT begins, once just after the 8-week study period, and once about 3 months later. Questions will cover topics including:

Parenting stress

Life satisfaction

Self-compassion

Uncomfortable feelings and thoughts

Mindfulness

Participants will take part in an 8-week ACT intervention. They will have one 75-minute session with an ACT coach in the first week.

Participants will watch 9- to 17-minute videos each week. The videos talk about how to practice ACT techniques to cope with parenting stress.

Participants will have 20- to 30-minute coaching sessions in weeks 3 and 6. The coach will help them practice exercises and work through any problems.

Background:

* RASopathies are a group of neurodevelopmental genetic conditions caused by mutations affecting components within the RAS-map kinase (RAS-MAPK) cellular signaling pathway.
* Caregivers of children with a RASopathy are faced with numerous challenges related to the physical, cognitive, and behavioral symptoms associated with their child s condition.
* The use of virtual interventions has been increasing over the past decade, which increase accessibility and are especially critical during times of physical distancing, such as due to COVID-19.
* Similarly, Ecological Momentary Assessments (EMA) are being utilized more frequently in order to maximize ecological validity of results and minimize recall bias.
* To our knowledge, no psychological interventions have been investigated targeting parenting stress among caregivers of a child with a RASopathy.

Primary Objectives:

* Internal Pilot Study: To assess the feasibility (e.g., enrollment and attrition) and acceptability (e.g., satisfaction) of an Acceptance and Commitment Therapy (ACT) coping intervention on parenting stress for caregivers of a child with a RASopathy
* Randomized Controlled Trial (RCT): To compare Parental Stress Scale (PSS) changes from baseline to 8 weeks between the Immediate Intervention Arm and the Waitlist Control Arm

Eligibility:

* Caregiver (parent or legal guardian) of a child (\< 18 years) residing with them at least 50% of the time who has a diagnosis of RASopathy syndrome including NF1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous (CFC) syndrome, and Costello Syndrome, or another RASopathy
* Access to necessary resources for participating in a technology-based intervention (i.e., computer, smartphone, internet access) or be willing to use an iPod provided by the study team
* Must score a 15 or higher total score on modified questions from the PSS to indicate at least a moderate level of parenting stress
* Ability to read and speak English

Design:

* This is an 8-week psychological intervention study that has two phases: the first, an internal pilot study, will enroll an initial, small cohort of caregivers (n = 8), all of whom will participate in the ACT intervention. (COMPLETE)
* Due to the success of the internal pilot study (with specific benchmarks met), the second phase of this study will be an RCT comparing participants who receive the intervention immediately (Immediate Intervention Arm) with those in a control group (Waitlist Control Arm). Participants randomized to the Waitlist Control Arm will cross over to receive the intervention after the waitlist period.
* The intervention for the RCT phase involves a baseline 90-minute coaching session and two follow-up coaching sessions with a therapist over video chat, followed by weekly prerecorded video modules that the participants can view at their convenience over the following 8 weeks.
* Participants will complete measures of parenting stress, mindfulness, psychological flexibility, self-compassion, and perceptions of child well-being at baseline, the 8-week post-intervention assessment, and a 3-month follow-up assessment. Additionally, the study will use EMA whereby questions will be sent electronically at random times and days (once a day at varying times on 5 days a week) during weeks 0-8 (the first week will serve as baseline data for comparison) for the Immediate Intervention Arm and weeks 0-18 for the Waitlist Control Arm using the Catalyst mobile applicatioon by MetricWire software.
* We plan to enroll 8 participants in the internal pilot phase and 56 participants in the RCT phase to account for the potential attrition of two caregivers from the intervention, for a total accrual target of 64 evaluable participants.

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Phase III Randomized Controlled Trial

.All IPD recorded in the medical record will be shared with intramural investigators upon request.

Feasibility defined by compliance with viewing \>50% of the weekly videos (proposed target rate of 5 out of 8 videos watched; must watch at least 80% of each video), and 70% of participants attending both coaching sessions 2 and 3. Acceptability defined by descriptive data per caregiver reports of satisfaction on the Study Satisfaction survey (mean of questions 1-7 and 9).

RCT: Mean scores on the Parental Stress Scale (PSS) will be compared from pre- to post-intervention (baseline to 8 weeks between the immediate intervention arm and the wait list control arm.

Feasibility defined by compliance with daily surveys (target rate of 30 out of 40 surveys (75%) completed on average by each participant). Acceptability defined by post-intervention participant reports of satisfaction with completing daily EMA ratings on the Study Satisfaction survey.

We will examine pre- to post-intervention changes in scores on measures of psychological flexibility (MPFI-SF), self-compassion (SCS-SF), experiential avoidance (BEAQ), depression (PROMIS Depression 8a) and child affect (PROMIS Positive Affect Scale).

We will look for a significant indirect association between treatment arm assignment and pre- to post-intervention PSS changes via psychological flexibility as measured by the BEAQ.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour	NCT03945682	Ataxia Posterior Fossa...	Therapist	4 Years - 18 Years	Alder Hey Children's NHS Foundation Trust
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial	NCT05361811	Neurofibromatos... Noonan Syndrome Legius Syndrome Cardiofaciocuta... Costello Syndro...	Waitlist ACT Interventio...	18 Years -	National Institutes of Health Clinical Center (CC)
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial	NCT05361811	Neurofibromatos... Noonan Syndrome Legius Syndrome Cardiofaciocuta... Costello Syndro...	Waitlist ACT Interventio...	18 Years -	National Institutes of Health Clinical Center (CC)

Spots Global Cancer Trial Database for therapist

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Spots Global Cancer Trial Database for therapist

Every month we try and update this database with for therapist 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 therapist 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.