Assessing reliability of functional tests in adults with neurofibromatosis type 1
A Study Investigating the Test- and Retest Reliability of a Battery of Functional Standardised Outcome Measures in Adults With Neurofibromatosis 1
This study is testing how reliable certain physical tests are for adults with neurofibromatosis type 1 by having them repeat the tests two weeks apart to see if the results stay the same.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Guy's and St Thomas' NHS Foundation Trust Academic / other |
| Locations | 1 site (London) |
| Trial ID | NCT05186870 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate the test-retest reliability of various functional outcome measures in adults diagnosed with neurofibromatosis type 1 (NF1). Participants will undergo assessments using the 10 meter walk test, timed up and go test, functional reach test, and grip dynamometry test on two separate occasions, 14 days apart. The study will recruit participants from the National Centre for Neurofibromatosis at Guy's Hospital, ensuring that their symptoms remain stable between assessments. Written consent will be obtained, and the results will help determine the consistency of these functional measures in this patient population.
Who should consider this trial
Good fit: Ideal candidates include adults aged 16 and over who have been diagnosed with neurofibromatosis type 1 and can walk independently for more than 10 meters.
Not a fit: Patients with significant non-neurofibromatosis related co-morbidities affecting mobility or balance may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide reliable outcome measures that enhance the assessment and management of patients with neurofibromatosis type 1.
How similar studies have performed: While the reliability of functional measures has been studied in other conditions, this specific focus on neurofibromatosis type 1 is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
London
- Guy's Hospital — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Amelia Khan, MSc
- Email: amelia.khan@gstt.nhs.uk
- Phone: 020 7188 5088
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.