The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour (ASPECT)
Primary Purpose
Ataxia, Posterior Fossa Tumor
Status
Unknown status
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
Therapist
Sponsored by
About this trial
This is an interventional treatment trial for Ataxia focused on measuring physiotherapy, surgical resection
Eligibility Criteria
Inclusion Criteria:
- Child or young person (4-<18 years) demonstrating ataxia. (For this study, ataxia is currently defined as SARA greater than 2. This definition is taken from data from the CARS study to date, from the cut off threshold value distinguishing no ataxia from mild ataxia)
- 12 months to 3 years following surgical resection of posterior fossa tumour. (This time frame has been selected as this covers the time when the children will typically have completed any adjunct oncology treatment and therefore more emphasis is dedicated to rehabilitation).
Exclusion Criteria:
- Medically unstable/currently undergoing adjunctive treatment e.g. radiotherapy or chemotherapy
- Less than 4 years of age (due to inability to complete standardized assessments)
- Aged 18 or over
- Unable to stand independently for less than one minute or SARA gait item score over 4, either of which would inhibit capacity to complete the training session.
- Presence of co-morbities with the potential to affect the safety of training, such as congenital or acquired disorders causing high risk of falls or lack of comprehension of training tasks, or current musculoskeletal impairments (such as a non-union fracture or fracture currently healing limiting weight-bearing)
Sites / Locations
- Alder Hey Children's NHS Foundation TrustRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Therapist
Usual Standard of Care
Arm Description
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
Outcomes
Primary Outcome Measures
Process Measures
Number of patients identified who meet the inclusion criteria and are then subsequently recruited will be recorded.
Intervention Process Measures
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.
Secondary Outcome Measures
SARA
Scale for the Assessment and Rating of Ataxia
BARS
Brief Ataxia Rating Scale
9HPT
Nine hole peg test
PEDI
Paediatric Evaluation of Disability Index
PedsQL
Brain Tumour Module
BBS
Berg Balance Scale
INAS
Inventory of Non Ataxia Signs. Assessment of compliance (diary)
Subjective Impact
Subjective Impact Likert Scale
Full Information
NCT ID
NCT03945682
First Posted
May 8, 2019
Last Updated
May 9, 2019
Sponsor
Alder Hey Children's NHS Foundation Trust
1. Study Identification
Unique Protocol Identification Number
NCT03945682
Brief Title
The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour
Acronym
ASPECT
Official Title
The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour (ASPECT)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2019
Overall Recruitment Status
Unknown status
Study Start Date
March 5, 2018 (Actual)
Primary Completion Date
December 31, 2020 (Anticipated)
Study Completion Date
December 31, 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alder Hey Children's NHS Foundation Trust
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ataxia, Posterior Fossa Tumor
Keywords
physiotherapy, surgical resection
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Participants will be randomised to one of 2 groups 1. Intervention arm 2. Usual standard of care.
Masking
None (Open Label)
Masking Description
No study drug as part of this RCT
Allocation
Randomized
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Therapist
Arm Type
Experimental
Arm Description
Intervention therapist at 2 centres providing 8 week intervention programme 50% embedded qualitative study
Arm Title
Usual Standard of Care
Arm Type
No Intervention
Arm Description
Participants continue with usual care and existing therapy recorded in study diary
Intervention Type
Other
Intervention Name(s)
Therapist
Intervention Description
Intervention therapist at 2 centres providing 8 week intervention programme 50% embedded qualitative study
Primary Outcome Measure Information:
Title
Process Measures
Description
Number of patients identified who meet the inclusion criteria and are then subsequently recruited will be recorded.
Time Frame
32 months
Title
Intervention Process Measures
Description
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.
Time Frame
32 months
Secondary Outcome Measure Information:
Title
SARA
Description
Scale for the Assessment and Rating of Ataxia
Time Frame
32 months
Title
BARS
Description
Brief Ataxia Rating Scale
Time Frame
32 months
Title
9HPT
Description
Nine hole peg test
Time Frame
32 months
Title
PEDI
Description
Paediatric Evaluation of Disability Index
Time Frame
32 months
Title
PedsQL
Description
Brain Tumour Module
Time Frame
32 months
Title
BBS
Description
Berg Balance Scale
Time Frame
32 months
Title
INAS
Description
Inventory of Non Ataxia Signs. Assessment of compliance (diary)
Time Frame
32 months
Title
Subjective Impact
Description
Subjective Impact Likert Scale
Time Frame
32 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Child or young person (4-<18 years) demonstrating ataxia. (For this study, ataxia is currently defined as SARA greater than 2. This definition is taken from data from the CARS study to date, from the cut off threshold value distinguishing no ataxia from mild ataxia)
12 months to 3 years following surgical resection of posterior fossa tumour. (This time frame has been selected as this covers the time when the children will typically have completed any adjunct oncology treatment and therefore more emphasis is dedicated to rehabilitation).
Exclusion Criteria:
Medically unstable/currently undergoing adjunctive treatment e.g. radiotherapy or chemotherapy
Less than 4 years of age (due to inability to complete standardized assessments)
Aged 18 or over
Unable to stand independently for less than one minute or SARA gait item score over 4, either of which would inhibit capacity to complete the training session.
Presence of co-morbities with the potential to affect the safety of training, such as congenital or acquired disorders causing high risk of falls or lack of comprehension of training tasks, or current musculoskeletal impairments (such as a non-union fracture or fracture currently healing limiting weight-bearing)
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Helen Hartley
Phone
0151 252 5660
Email
helen.hartley@alderhey.nhs.uk
First Name & Middle Initial & Last Name or Official Title & Degree
Research Alder Hey
Phone
0151 252 5570
Email
research@alderhey.nhs.uk
Facility Information:
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
State/Province
Merseyside
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Research Alder Hey
Phone
0151 252 5570
Email
research@alderhey.nhs.uk
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour
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