Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest

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Brief Title

Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest

Official Title

A Study Investigating the Test- and Retest Reliability of a Battery of Functional Standardised Outcome Measures in Adults With Neurofibromatosis 1

Brief Summary

      To examine the test-retest reliability (how stable the results are when the same
      participants, whose symptoms have remained stable, are assessed on 2 different occasions, 14
      days apart) of the 10 meter walk test, the timed up and go test, the functional reach test
      and the grip dynamometry test in adults with neurofibromatosis 1 (NF1).
    

Detailed Description

      Participants' will be recruited during their attendance to the National Centre for
      Neurofibromatosis at Guy's hospital between August 2019 and March 2020 then following a
      COVID-19 break, from March 2022 until October 2022. Guy's and St Thomas' Trust (GSTT) is a
      National Centre for the diagnosis, management and support of approximately 1600 people with
      neurofibromatosis 1. Currently patients' attend the clinic for their standard assessment
      appointment every 6-12 months. They will receive a participant information sheet before their
      appointment, and the treating clinician (specialist doctors and nurses) will confirm that the
      individual meets the inclusion criteria and identify whether they wish to participate. At
      each of these contact points as well as within the participant information sheet, the
      potential participant will be advised that their decision to participate/ not to participate
      within this study will have no impact on their clinical care. Each participant will be asked
      to complete each outcome measure at both study visits.

      Once written consent has been obtained each participant will be given a unique alphanumeric
      identification code for use within the trial. Written consent to participate in the study
      will be collected from the participant by the researcher (FD) on the day of their first
      outcome measure appointment. The researcher will then start the outcome measurement testing
      session. The participant will be expected to attend one additional appointments to their
      standard bi-yearly/yearly appointment where data on the outcome measure will only be
      collected, the additional appointment will be 14 days later. During each of these
      appointments the participants will be asked to complete 4 outcome measures (Timed-up and go,
      Timed 10m walk test, Functional reach test and Grip-A Dynamometry) 3 times (and a mean will
      be taken) alongside completion of the neurofibromatosis 1 quality of life questionnaire
      (INFQOL), and a basic neurological assessment (to establish a baseline of symptoms) During
      their second appointment, in addition to above, the participant will also be asked if they
      have noted any changes in their symptoms in the last 14 days. The testing procedure will take
      approximately 60 minutes per participant in total outside of the individual's clinic
      appointment time.

      Outcome measures The 10m walk test will time how long it takes the participant to walk for a
      distance of 10m (this will be on a marked walkway). The timed up and go test will time how
      long it takes the participant to stand up from a chair, walk 3m, turn around and return to
      sitting on the chair. For each of the above tests the participant will complete them
      independently but can use a walking aid if they require to. The functional reach test will
      require the participant to stand with there arm out in front of them and reach forward as far
      as they can without moving their feet, the distance will be recorded with a tape measure
      attached to the wall. The final test, the grip dynamometry with require the participant to
      squeeze the Grip-A Dynamometer as hard as they can. They will complete these 3 times and a
      mean score will be taken.

      Data Analysis and sample size Data will be collected and analyzed using Pearson and
      intra-class correlation models. These models are recommended when testing for reliability as
      repeated measurements cannot be regarded as randomized samples (Koo and Li, 2016). A sample
      of 20 (5 x the items to be tested) participants is recommended for use in test- retest
      studies (Park et al., 2017) to ensure that the sample is representative of the wider
      population. Once the data has been analyzed by the relevant clinician's the data will be
      destroyed in line with trust policy.

      The researcher will not be involved in the initial invitation of participants to the study
      and will have no influence on who can join the study other than providing the inclusion and
      exclusion criteria. The participants overall standard care will not be affected through
      participation in the study inclusive if the recruitment process. In accordance with Good
      Clinical Practice (GCP) we will ensure that participants understand fully the reason for the
      study and their involvement.

      Inclusion/exclusion criteria All participants aged 16 years or older who fulfill the
      diagnostic criteria for neurofibromatosis 1, who do not have a significant mobility or
      balance impairment that is unrelated to their neurofibromatosis 1, are able to walk more than
      10 meters without physical assistance (may use walking aids), who are able to provide written
      consent and who attend the national Neurofibromatosis service at GSTT will be eligible to
      participate in the study.

      Consent Participants will be recruited by the researcher (chief investigator) will obtain
      written consent to participate in the study a from every individual after they have had at
      least 24 hours to read the information sheet and the opportunity to ask questions about the
      study. Consent will be obtained by the chief investigator who has attended Good Clinical
      Practice Training.

      Steps to reduce researcher bias To reduce the risk of selection bias, multiple clinicians
      will be included in recruitment however, the researcher will not be involved in this process.

      To reduce the risk of performance bias, the outcome measurements will all be collected by the
      same Physiotherapist with standardized instructions.

      To reduce the risk of detection bias, a statistician will review the statistical plan and the
      data collected to ensure appropriateness and accuracy.
    


Study Type

Observational


Primary Outcome

Timed 10 meter walk test.


Condition

Neurofibromatosis Type 1

Intervention

Outcome measures


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

20

Start Date

November 30, 2019

Completion Date

July 2023

Primary Completion Date

July 2022

Eligibility Criteria

        Inclusion Criteria:

          -  Patients who

               1. fulfill the diagnostic criteria for neurofibromatosis 1

               2. are aged 16 years or over

               3. attend the national neurofibromatosis service at GSTT

               4. are capable of providing informed consent

               5. do not have any non neurofibromatosis 1 related co-morbidities that affect
                  mobility or balance (confirmed with the treating clinician)

               6. are able to walk more than 10 metres without assistance of a person (may use
                  walking aid/orthotics)

               7. Symptoms that have remained stable between the 2 testing sessions (14 days). This
                  will be assessed with the use of a neurological examination (Power, Sensation,
                  Co-ordination, proprioception and reflexes), the NFQOL questionnaire and a
                  subjective question to the patient as to if they feel there have been any changes
                  to their symptoms in the last 14 days (completed on a percentage, 0= no change,
                  100% large change impacting their function). The assessing clinician will
                  determine if any change is a significant change based on a reduction in power of
                  more than 1 point (Oxford scale 0-5) and if there is a significant change in
                  scores in comparison with the published norms.

        Exclusion Criteria:

          -  Patients who

               1. do not fulfill the diagnostic criteria for neurofibromatosis 1

               2. are under the age of 16 years

               3. are unable to provide informed consent

               4. have non neurofibromatosis 1 related co-morbidities that affect mobility or
                  balance (confirmed with the treating clinician)

               5. are unable to walk more than 10 metres without assistance of a person (may use
                  walking aid/orthotics)

               6. Patients whose symptoms have not remained stable between the 2 testing sessions
                  (14 days). This will be assessed with the use of a neurological examination
                  (Power, Sensation, Co-ordination, proprioception and reflexes), the NFQOL
                  questionnaire and a subjective question to the patient as to if they feel there
                  have been any changes to their symptoms in the last 14 days (completed on a
                  percentage, 0= no change, 100% large change impacting their function). The
                  assessing clinician will determine if any change is a significant change based on
                  The assessing clinician will determine if any change is a significant change
                  based on a reduction in power of more than 1 point (Oxford scale 0-5) and if
                  there is a significant change in scores in comparison with the published norms.

               7. If the participant has been involved in research which may impact the testing
                  (e.g. changes in treatment which may impact their symptoms during the
                  trial/currently involved in a study completing the same outcome measures and
                  adding a level of learned bias) then they will not be invited to join the study.
      

Gender

All

Ages

16 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

, , 

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT05186870

Organization ID

253954


Responsible Party

Sponsor

Study Sponsor

Guy's and St Thomas' NHS Foundation Trust


Study Sponsor

, , 


Verification Date

December 2021