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)

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).

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.

Study Overview

• Status: Completed
• Conditions: Neurofibromatosis 1 and 2 (NF1 and NF2)
• Intervention / Treatment: Behavioral: hand-held dynamometer

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10016
      • New York School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: Cohort 1; Patients with NF1	Behavioral: hand-held dynamometer; Strength of one weak muscle group and of the biceps of the dominant arm will be measured using a hand-held dynamometer. An average of three readings will be used as one observation. Three observations per patient will be obtained on one day with a minimum of 15 minute rest period between each observation.
ACTIVE_COMPARATOR: Cohort 2; Patients with NF2	Behavioral: hand-held dynamometer; Strength of one weak muscle group and of the biceps of the dominant arm will be measured using a hand-held dynamometer. An average of three readings will be used as one observation. Three observations per patient will be obtained on one day with a minimum of 15 minute rest period between each observation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Intra-class correlation coefficient of strength measurement in the clinically identified weak muscle	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.	1 day

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Intra-class correlation coefficient of strength measurement in the dominant biceps	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.	1 day
standard error of measurement of measuring strength in the weak muscles		1 day
standard error of measurement of measuring strength in the dominant biceps muscle		1 day

Collaborators and Investigators

• Sponsor: NYU Langone Health
• Investigators: Principal Investigator: Kaleb H Yohay, MD, New York School of Medicine

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 24, 2018
• Primary Completion (ACTUAL): December 2, 2019
• Study Completion (ACTUAL): December 2, 2019

Study Registration Dates

• First Submitted: September 13, 2018
• First Submitted That Met QC Criteria: September 14, 2018
• First Posted (ACTUAL): September 17, 2018

Study Record Updates

• Last Update Posted (ACTUAL): March 18, 2021
• Last Update Submitted That Met QC Criteria: March 16, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Peripheral Nervous System Diseases; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Neoplastic Syndromes, Hereditary; Nerve Sheath Neoplasms; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibromatoses; Neurofibromatosis 1; Neurofibroma
• Other Study ID Numbers: 18-00831

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

De-identified data for patients who consent for their data to be shared for future research.

The data will be made available: Requests should be directed to xxx@yyy. To gain access, data requestors will need to sign a data access agreement.

• IPD Sharing Time Frame: Beginning 3 months and ending 5 years following article publication
• IPD Sharing Access Criteria: Investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose will have access to the data.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No