A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)

Not Applicable

Completed

• Conditions: Neurofibromatosis 1 and 2 (NF1 and NF2)
• Interventions: Behavioral: hand-held dynamometer

• Registration Number: NCT03672838
• Lead Sponsor: NYU Langone Health

Brief Summary

Patients with clinically confirmed neurofibromatosis type 1 (NF1) or neurofibromatosis type 2 (NF2) or a known neurofibromatosis (NF) mutation aged 5 years and above will be eligible to participate and will be recruited from the neurofibromatosis clinic. Given the need for identifying measures that can reliably and sensitively measure focal muscle weakness and allow for measuring muscle strength as a functional outcome in therapeutic clinical trials in NF, this pilot study will assess the reliability of measuring muscle strength in NF1 and NF2 using a hand-held dynamometer.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-09-13
• Study First Submitted QC: 2018-09-14
• Study First Posted: 2018-09-17
• Study Start: 2018-09-24
• Primary Completion: 2019-12-02
• Study Completion: 2019-12-02
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-16
• Last Update Posted: 2021-03-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

• For cohort 1:

  • Patients must have clinically confirmed NF1 using the NIH consensus criteria or a known NF1 mutation
  • Ages 5 years and above
  • < 5/5 strength on Manual Muscle Testing (MMT) testing in at least one muscle group tested on the day of enrollment
  • Able to follow instructions and cooperate with exam to assess strength

• For Cohort 2

  • Patients must have clinically (NIH or Manchester criteria) or genetically confirmed diagnosis of NF2
  • Ages 5 years and above
  • < 5/5 strength on MMT testing in at least one muscle group tested on the day of enrollment
  • Able to follow instructions and cooperate with exam to assess strength

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Exclusion Criteria

• No orthopedic procedure or other major surgery that could influence extremity strength in past 6 months
• No tibial dysplasia

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 1	hand-held dynamometer	Patients with NF1
Cohort 2	hand-held dynamometer	Patients with NF2

Primary Outcome Measures

Name	Time	Method
Intra-class correlation coefficient of strength measurement in the clinically identified weak muscle	1 day	A sample size of 20 subjects with three observations per subject achieves 90% power to detect an intraclass correlation of 0.91 under the alternative hypothesis when the intraclass correlation under the null hypothesis is 0.75 using an F-test with a significance level of 0.05.

Secondary Outcome Measures

Name	Time	Method
standard error of measurement of measuring strength in the weak muscles	1 day
Intra-class correlation coefficient of strength measurement in the dominant biceps	1 day	A sample size of 20 subjects with three observations per subject achieves 90% power to detect an intraclass correlation of 0.91 under the alternative hypothesis when the intraclass correlation under the null hypothesis is 0.75 using an F-test with a significance level of 0.05.
standard error of measurement of measuring strength in the dominant biceps muscle	1 day

Trial Locations

Locations (1)

New York School of Medicine; 🇺🇸 New York, New York, United States