Neurofibromatosis 1, NF1NCT02479360
Reliability of Functional Outcome Measures in Neurofibromatosis 1
An observational study of Neurofibromatosis 1, NF1, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Neurofibromatosis 1 (NF1) is a common inherited condition that affects the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and their secondary complications. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Neurofibromatosis 1, NF1
- How many people
- 49 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jun 2015to Sep 2015
- 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 1 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
- All patients aged 16 years or older who fulfil the diagnostic criteria for NF1 who do not have a significant mobility or balance impairment that is unrelated to their NF1,
- Able to walk more than 10 metres without physical assistance (may use walking aids)
- Patients who attend the national NF1 service at GSTT
Who cannot join
- Patients who are unable to provide informed consent will be unable to participate in this study.
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 a Battery of Functional Standardised Outcome Measures in Neurofibromatosis 1
Neurofibromatosis 1 (NF1) is a common inherited condition that affects the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and their secondary complications. At present evaluation of the effect of treatments in NF1 is based on a clinician's neurological assessment, magnetic resonance imaging and positron emission tomography but it is apparent that imaging results do not always link with clinical and functional changes in an individual. This study aims to evaluate the reliability (stability) of a range of functional standardised outcome measures in adults with NF1.
Read the detailed description
Neurofibromatosis 1 (NF1) is an inherited condition that occurs in 1 in 2,500 births; affecting the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and their secondary complications. One person with NF1 may have minimal issues whereas another may be profoundly disabled; one may have difficulties using their hands and arms and another may have difficulties standing from a chair. New pharmacological and nonpharmacological treatments for NF1 are currently being developed and they have potential to dramatically influence a person with NF1's life. At present evaluation of treatment effect in NF1 is based on a clinician's neurological assessment, magnetic resonance imaging and positron emission tomography and it is apparent that imaging results do not always link with clinical and functional changes in an individual. Therefore clinicians and researchers agree that the analysis of treatment effect needs to include the evaluation of a person's function. The findings from this study will improve the care of people with NF1 by ensuring that we are able to measure functional changes in a person with NF1 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 (e.g. walking, standing from a chair, use of hands) 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 NF1 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 NF1.
This study aims to evaluate four commonly used SOM's for reliability in adults with NF1 (over 16 years of age). The measures chosen for evaluation are the 10 metre walk test, the timed up and go test, the functional reach test and the nine hole peg test. 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.
- Conditions
- Neurofibromatosis 1, NF1
- Medical subject headings
- Neurofibromatosis 1
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 10 metre walk test, the timed up and go test, the functional reach test and the nine-hole peg test. A physiotherapist 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 NF1 consultants and one NF1 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
10 metre walk test
Participant is asked to stand at a point 2.5m before the start of a 10 metre walk way. They are advised to walk at their 'normal speed' from here to (a point 2.5m beyond the 10m walk way). The assessor times from the moment the participant's leading foot crosses the start point for the 10 metre walk way until the toes of the participant's leading foot crosses the end of the 10m walk way. Measurements are recorded in seconds and milliseconds. Participants can use their normal aids and orthotics if needed.
Measured: Between 5 and 30 seconds per test on average.
Functional Reach test
Participant stands with arms straight out in front of him/herself at shoulder level and hands forming fists. The assessor sticks a ruler to the wall at the end of their hands. Participant is advised to lean forwards as far as they can without falling over and the assessor measures the distance they can reach against the metre ruler in centimetres and millimetres.
Measured: 30 seconds per test
9 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 60 seconds per hand.
Timed up and go test
Participant sits in a chair at the end of the 3m walkway. S/he is asked to stand up from the chair walk to the line, turn around once they reach the line and return to the chair. If they do not touch the line during the test, the test is invalid. The participant can use any aids/ orthotics that they normally use. The assessor will time from the moment the participants back leaves the backrest until it touches it again at the end of the test. Time will be recorded in seconds and milliseconds.
Measured: Up to 60 seconds
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2015 (estimated)
- Study startJun 2015
- Primary completionSep 2015
- Study completionSep 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1, NF1Studies 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
- 49 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
- NCT02479360
- Sponsor's protocol ID
- RJ115/N174
- Status last verified
- Jun 2015
- First submitted
- 19 Jun 2015
- Last updated
- 6 Oct 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2015. 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 a Battery of Functional Standardised Outcome Measures in Neurofibromatosis 1
- ClinicalTrials.gov ID
- NCT02479360
- Acronym / sponsor ID
- RJ115/N174
- Conditions
- Neurofibromatosis 1, NF1
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Guy's and St Thomas' NHS Foundation TrustUniversity, hospital or other
- Study start
- Jun 2015
- Primary completion
- Sep 2015 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 24 Jun 2015 (estimated)
- 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 1 (NF1) is a common inherited condition that affects the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and their secondary complications. At present evaluation of the effect of treatments in NF1 is based on a clinician's neurological assessment, magnetic resonance imaging and positron emission tomography but it is apparent that imaging results do not always link with clinical and functional changes in an individual. This study aims to evaluate the reliability (stability) of a range of functional standardised outcome measures in adults with NF1.
Detailed description
Neurofibromatosis 1 (NF1) is an inherited condition that occurs in 1 in 2,500 births; affecting the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and their secondary complications. One person with NF1 may have minimal issues whereas another may be profoundly disabled; one may have difficulties using their hands and arms and another may have difficulties standing from a chair. New pharmacological and nonpharmacological treatments for NF1 are currently being developed and they have potential to dramatically influence a person with NF1's life. At present evaluation of treatment effect in NF1 is based on a clinician's neurological assessment, magnetic resonance imaging and positron emission tomography and it is apparent that imaging results do not always link with clinical and functional changes in an individual. Therefore clinicians and researchers agree that the analysis of treatment effect needs to include the evaluation of a person's function. The findings from this study will improve the care of people with NF1 by ensuring that we are able to measure functional changes in a person with NF1 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 (e.g. walking, standing from a chair, use of hands) 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 NF1 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 NF1.
This study aims to evaluate four commonly used SOM's for reliability in adults with NF1 (over 16 years of age). The measures chosen for evaluation are the 10 metre walk test, the timed up and go test, the functional reach test and the nine hole peg test. 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.
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 10 metre walk test, the timed up and go test, the functional reach test and the nine-hole peg test. A physiotherapist 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 NF1 consultants and one NF1 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. |
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 1 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 (3 listed)
- All patients aged 16 years or older who fulfil the diagnostic criteria for NF1 who do not have a significant mobility or balance impairment that is unrelated to their NF1,
- Able to walk more than 10 metres without physical assistance (may use walking aids)
- Patients who attend the national NF1 service at GSTT
Key exclusion criteria (1 listed)
- Patients who are unable to provide informed consent will be unable to participate in this study.
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
10 metre walk test
Time frame: Between 5 and 30 seconds per test on average.
Participant is asked to stand at a point 2.5m before the start of a 10 metre walk way. They are advised to walk at their 'normal speed' from here to (a point 2.5m beyond the 10m walk way). The assessor times from the moment the participant's leading foot crosses the start point for the 10 metre walk way until the toes of the participant's leading foot crosses the end of the 10m walk way. Measurements are recorded in seconds and milliseconds. Participants can use their normal aids and orthotics if needed.
Functional Reach test
Time frame: 30 seconds per test
Participant stands with arms straight out in front of him/herself at shoulder level and hands forming fists. The assessor sticks a ruler to the wall at the end of their hands. Participant is advised to lean forwards as far as they can without falling over and the assessor measures the distance they can reach against the metre ruler in centimetres and millimetres.
9 hole peg test
Time frame: Up to 60 seconds per hand.
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.
Timed up and go test
Time frame: Up to 60 seconds
Participant sits in a chair at the end of the 3m walkway. S/he is asked to stand up from the chair walk to the line, turn around once they reach the line and return to the chair. If they do not touch the line during the test, the test is invalid. The participant can use any aids/ orthotics that they normally use. The assessor will time from the moment the participants back leaves the backrest until it touches it again at the end of the test. Time will be recorded in seconds and milliseconds.
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 Oct 2015.
Related trials for Neurofibromatosis 1, NF1
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms
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 1, NF1, 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 3 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 Oct 2015. 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?
10 metre walk test (time frame: Between 5 and 30 seconds per test on average.). The study lists 4 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 Oct 2015.