Neurofibromatosis 2NCT03617276
Reliability of Functional Outcome Measures in Neurofibromatosis 2
An observational study of Neurofibromatosis 2, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Neurofibromatosis 2 (NF2) is a rare inherited condition. Those with NF2 usually present with tumours (schwannomas) in their inner ear, or vestibular organ, with common symptoms including problems with hearing, balance, dizziness and vision. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Neurofibromatosis 2
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 7 Sep 2016to 17 May 2017
- Phase
- Observational
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All adults (over the age of 16 years) with a clinical diagnosis of neurofibromatosis 2 who attend the neurofibromatosis clinic for appointments at Guys hospital, London and who fit the inclusion/exclusion criteria for the trial are eligable to participate in the study.
Who may be able to join
- Fulfill diagnostic criteria for NF2 Aged 16 years or older Attend the national NF service at Guys and St Thomas' NHS Foundation Trust Are able to provide informed consent Do not have any non-NF2 related comorbidities that affect mobility or balance (confirmed by the treating clinician) Are able to walk more than 10m without assistance of a person Do not have unstable vascular or orthopaedic pathology at the cervical spine (confirmed by consultant) Have not had a stroke within the last 3 months
Who cannot join
- Do not fulfill diagnostic criteria for NF2 Aged under 16 years or older Are unable to provide informed consent Have non-NF2 related comorbidities that affect mobility or balance (confirmed by the treating clinician) Are unable to walk more than 10m without assistance of a person Have unstable vascular or orthopaedic pathology at the cervical spine (confirmed by consultant) Have had a stroke within the last 3 months
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Neurofibromatosis Unit, Guys Hospital.
London, SE1 9RT
About this study
Official title: A Study Investigating the Inter- and Intra- Rater Reliability of Functional Outcome Measures in Neurofibromatosis 2
Neurofibromatosis 2 (NF2) is a rare inherited condition. Those with NF2 usually present with tumours (schwannomas) in their inner ear, or vestibular organ, with common symptoms including problems with hearing, balance, dizziness and vision. This study aims to evaluate the reliability (stability) of a range of functional standardised outcome measures in adults with NF2.
Read the detailed description
Neurofibromatosis 2 (NF2) is an inherited condition that occurs in 1 in 33,000 births; affecting hearing, balance, dizziness and vision primarily related to tumours (schwannomas) in the inner ear or other tumours which develop within the central nervous system. Neuropathy may also be an issue for people with NF2.
The complications of NF2 can lead to varied difficulties with activities of daily living and may impair quality of life. More recently, new pharmacological treatments for NF2 are being trialed which may have the potential to dramatically influence quality of life for people with NF2. Currently, evaluation of treatment effect in NF2 is based on a clinician's neurological assessment and imaging. However, imaging results do not always correlate with clinical and reported functional changes of the individual. Therefore, evaluating the effect of interventions or treatment should include the evaluation of a person's function using standardised outcome measures. The findings from this study will improve the care of people with NF2 by ensuring that we are able to measure functional changes in a person with NF2 with greater confidence, meaning that we can start, continue or cease treatments based on the holistic picture of an individual.
The world confederation of physiotherapists (WCPT) stipulates that standardised outcome measures (SOM) are used to provide a quantitative measure of capability, and to evaluate the impact of treatment on function. There are a wide range of SOM's that evaluate function so a SOM is chosen by the clinician based on the patient's presenting functional difficulty and the scientific credibility of the SOM. Scientific credibility (robustness) is based on scientific properties of the SOM i.e. how stable the SOM is (reliability) and whether it assesses what it is intending to assess (validity). It is important to evaluate each SOM within the target population as variability within that condition, strongly influences the SOM's reliability and validity (de Vel 2011).
At present, functional SOM's have limited scientific and subsequent clinical credibility when used in people with NF2 because their scientific properties have not yet been evaluated within this patient group which means that if they are used clinically or within research, there is an inherent element of doubt when interpreting the SOM's results. The objective of this research team is to create a core set of functional SOM's which have undergone rigorous scientific evaluation so that they can be used in the future for clinical practice and for research purposes in adults with NF2.
This study primarily aims to evaluate three commonly used SOM's for reliability in adults with NF2 (over 16 years of age). The measures chosen for evaluation are the nine hole peg test, the four square step test and the modified clinical test of sensory integration and balance (mCTSIB). Interrater reliability (the test result remains the same when reviewed by different people) and intrarater reliability (the test result remains stable when reviewed twice by the same tester) will be evaluated. From this data, we will be able to calculate the standard error of measurement an additional important scientific characteristics of SOM evaluation. A secondary objective is to correlate SOM objective markers with a subjective disease specific quality of life measure (NFTI-QOL) and self-reported falls rate and near misses. We will also collect data on dynamic visual acuity in this patient group.
- Conditions
- Neurofibromatosis 2
- Medical subject headings
- Neurofibromatosis 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Outcome measurement | Each participant will be asked to complete each standardised outcome measure (SOM) three times and each trial will be videotaped by the researcher. The selected SOM's are the modified nine-hole peg test, the four square step test and the modified clinical test of sensory integration and balance. A doctor will watch the video on 2 separate occasions to evaluate intra-rater reliability. Inter-rater reliability will be assessed through asking three other neurofibromatosis specialist professionals (two NF2 consultants and one NF2 specialist nurse) to review the video and to score each measure completed. Once the filmed sessions have been analysed by the relevant clinician's the data will be destroyed in line with Trust policy. Each participant will also be required to complete the INFI-QOL questionnaire, the dynamic visual acuity test and provide information about the number of falls/near misses they have had over the past 12 months |
What is being measured
The primary outcome is the main question the study is designed to answer.
Modified nine hole peg test
Participant is sitting comfortably in a chair in front of a desk. The 9 hole peg test equipment is set up so that the bowl containing pegs is directly behind the pegboard and in front of the participant. Participants can rest the forearms on the desk but must use only 1 hand for each test. If not the test is invalid. The assessor measures from the time the fingers pick up the first peg from the bowl until the fingers place the last peg in the hole of the pegboard. Time will be recorded in seconds and milliseconds.
Measured: Up to 3 minutes
Four square step test
This is a test of balance when moving. Participants are asked to step forwards, sideways and backwards in a specific pattern. The rater times how long it takes to complete the test
Measured: Up to 30 seconds
Modified Clinical Test of Sensory Integration and Balance
This is a test of standing balance. Participants are asked to stand upright with the feet together, first on a hard surface (the floor) and then on a soft surface (some foam). The rater will time the participant standing in each position with the eyes open and shut.
Measured: Up to 10 minutes
Secondary outcomes (2)
Dynamic Visual Acuity test
This is a test of vision when the head is turning. The participant is asked to read letters from a computer screen when the head is still then when it is being turned by a physiotherapist. The rater measures the accuracy of vision
Measured: Up to 3 minutes
The NFTI-QOL: A Disease-Specific Quality of Life Questionnaire for Neurofibromatosis 2
An eight question, self-report, disease specific quality of life questionnaire. Participants rate problems experienced within eight domains (balance, hearing, facial weakness, sight, walking, outlook on life, pain, mood) as no issues, slight problems, some difficulties, severe difficulties)
Measured: Up to 2 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2018
- Study start7 Sep 2016
- Primary completion17 May 2017
- Study completion17 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 2Studies of the same condition, open and under way.
- More trials run by Guy's and St Thomas' NHS Foundation TrustThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03617276
- Sponsor's protocol ID
- 16/NW/0504
- Status last verified
- Aug 2018
- First submitted
- 1 Aug 2018
- Last updated
- 6 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to share individual participant data with other researchers- this was not agreed in our ethical plan
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study Investigating the Inter- and Intra- Rater Reliability of Functional Outcome Measures in Neurofibromatosis 2
- ClinicalTrials.gov ID
- NCT03617276
- Acronym / sponsor ID
- 16/NW/0504
- Conditions
- Neurofibromatosis 2
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Guy's and St Thomas' NHS Foundation TrustUniversity, hospital or other
- Study start
- 7 Sep 2016 (actual)
- Primary completion
- 17 May 2017 (actual)
- Study completion
- 17 May 2017 (actual)
- First posted
- 6 Aug 2018 (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.
Neurofibromatosis 2 (NF2) is a rare inherited condition. Those with NF2 usually present with tumours (schwannomas) in their inner ear, or vestibular organ, with common symptoms including problems with hearing, balance, dizziness and vision. This study aims to evaluate the reliability (stability) of a range of functional standardised outcome measures in adults with NF2.
Detailed description
Neurofibromatosis 2 (NF2) is an inherited condition that occurs in 1 in 33,000 births; affecting hearing, balance, dizziness and vision primarily related to tumours (schwannomas) in the inner ear or other tumours which develop within the central nervous system. Neuropathy may also be an issue for people with NF2.
The complications of NF2 can lead to varied difficulties with activities of daily living and may impair quality of life. More recently, new pharmacological treatments for NF2 are being trialed which may have the potential to dramatically influence quality of life for people with NF2. Currently, evaluation of treatment effect in NF2 is based on a clinician's neurological assessment and imaging. However, imaging results do not always correlate with clinical and reported functional changes of the individual. Therefore, evaluating the effect of interventions or treatment should include the evaluation of a person's function using standardised outcome measures. The findings from this study will improve the care of people with NF2 by ensuring that we are able to measure functional changes in a person with NF2 with greater confidence, meaning that we can start, continue or cease treatments based on the holistic picture of an individual.
The world confederation of physiotherapists (WCPT) stipulates that standardised outcome measures (SOM) are used to provide a quantitative measure of capability, and to evaluate the impact of treatment on function. There are a wide range of SOM's that evaluate function so a SOM is chosen by the clinician based on the patient's presenting functional difficulty and the scientific credibility of the SOM. Scientific credibility (robustness) is based on scientific properties of the SOM i.e. how stable the SOM is (reliability) and whether it assesses what it is intending to assess (validity). It is important to evaluate each SOM within the target population as variability within that condition, strongly influences the SOM's reliability and validity (de Vel 2011).
At present, functional SOM's have limited scientific and subsequent clinical credibility when used in people with NF2 because their scientific properties have not yet been evaluated within this patient group which means that if they are used clinically or within research, there is an inherent element of doubt when interpreting the SOM's results. The objective of this research team is to create a core set of functional SOM's which have undergone rigorous scientific evaluation so that they can be used in the future for clinical practice and for research purposes in adults with NF2.
This study primarily aims to evaluate three commonly used SOM's for reliability in adults with NF2 (over 16 years of age). The measures chosen for evaluation are the nine hole peg test, the four square step test and the modified clinical test of sensory integration and balance (mCTSIB). Interrater reliability (the test result remains the same when reviewed by different people) and intrarater reliability (the test result remains stable when reviewed twice by the same tester) will be evaluated. From this data, we will be able to calculate the standard error of measurement an additional important scientific characteristics of SOM evaluation. A secondary objective is to correlate SOM objective markers with a subjective disease specific quality of life measure (NFTI-QOL) and self-reported falls rate and near misses. We will also collect data on dynamic visual acuity in this patient group.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Outcome measurement | – | –Each participant will be asked to complete each standardised outcome measure (SOM) three times and each trial will be videotaped by the researcher. The selected SOM's are the modified nine-hole peg test, the four square step test and the modified clinical test of sensory integration and balance. A doctor will watch the video on 2 separate occasions to evaluate intra-rater reliability. Inter-rater reliability will be assessed through asking three other neurofibromatosis specialist professionals (two NF2 consultants and one NF2 specialist nurse) to review the video and to score each measure completed. Once the filmed sessions have been analysed by the relevant clinician's the data will be destroyed in line with Trust policy. Each participant will also be required to complete the INFI-QOL questionnaire, the dynamic visual acuity test and provide information about the number of falls/near misses they have had over the past 12 months |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All adults (over the age of 16 years) with a clinical diagnosis of neurofibromatosis 2 who attend the neurofibromatosis clinic for appointments at Guys hospital, London and who fit the inclusion/exclusion criteria for the trial are eligable to participate in the study.
Key inclusion criteria (1 listed)
- Fulfill diagnostic criteria for NF2 Aged 16 years or older Attend the national NF service at Guys and St Thomas' NHS Foundation Trust Are able to provide informed consent Do not have any non-NF2 related comorbidities that affect mobility or balance (confirmed by the treating clinician) Are able to walk more than 10m without assistance of a person Do not have unstable vascular or orthopaedic pathology at the cervical spine (confirmed by consultant) Have not had a stroke within the last 3 months
Key exclusion criteria (1 listed)
- Do not fulfill diagnostic criteria for NF2 Aged under 16 years or older Are unable to provide informed consent Have non-NF2 related comorbidities that affect mobility or balance (confirmed by the treating clinician) Are unable to walk more than 10m without assistance of a person Have unstable vascular or orthopaedic pathology at the cervical spine (confirmed by consultant) Have had a stroke within the last 3 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Modified nine hole peg test
Time frame: Up to 3 minutes
Participant is sitting comfortably in a chair in front of a desk. The 9 hole peg test equipment is set up so that the bowl containing pegs is directly behind the pegboard and in front of the participant. Participants can rest the forearms on the desk but must use only 1 hand for each test. If not the test is invalid. The assessor measures from the time the fingers pick up the first peg from the bowl until the fingers place the last peg in the hole of the pegboard. Time will be recorded in seconds and milliseconds.
Four square step test
Time frame: Up to 30 seconds
This is a test of balance when moving. Participants are asked to step forwards, sideways and backwards in a specific pattern. The rater times how long it takes to complete the test
Modified Clinical Test of Sensory Integration and Balance
Time frame: Up to 10 minutes
This is a test of standing balance. Participants are asked to stand upright with the feet together, first on a hard surface (the floor) and then on a soft surface (some foam). The rater will time the participant standing in each position with the eyes open and shut.
2 secondary outcomes
Dynamic Visual Acuity test
Time frame: Up to 3 minutes
The NFTI-QOL: A Disease-Specific Quality of Life Questionnaire for Neurofibromatosis 2
Time frame: Up to 2 minutes
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2018.
- Individual participant dataSharing planned: No. No plan to share individual participant data with other researchers- this was not agreed in our ethical plan
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis 2, run by Guy's and St Thomas' 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 1 inclusion and 1 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: finished, as of the sponsor's last update on 6 Aug 2018. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Modified nine hole peg test (time frame: Up to 3 minutes). The study lists 3 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 6 Aug 2018.