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)

NCT03672838 | Completed

• 1 other identifier: 18-00831
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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.

Conditions

Neurofibromatosis 1 and 2 (NF1 and NF2)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Intra-class correlation coefficient of strength measurement in the dominant biceps

  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

Study Arms (2)

Cohort 1

ACTIVE COMPARATOR

Patients with NF1

Behavioral: hand-held dynamometer

Cohort 2

ACTIVE COMPARATOR

Patients with NF2

Behavioral: hand-held dynamometer

Interventions

hand-held dynamometer BEHAVIORAL

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.

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

New York School of Medicine

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibromatosis 2

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuroma, Acoustic; Neurilemmoma; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neuroma; Vestibulocochlear Nerve Diseases; Retrocochlear Diseases; Ear Diseases; Otorhinolaryngologic Diseases; Otorhinolaryngologic Neoplasms; Cranial Nerve Neoplasms; Cranial Nerve Diseases

Study Officials

• Kaleb H Yohay, MD

  New York School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2018
• First Posted: September 17, 2018
• Study Start: September 24, 2018
• Primary Completion: December 2, 2019
• Study Completion: December 2, 2019
• Last Updated: March 18, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: Beginning 3 months and ending 5 years following article publication
• 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.

Locations

US (1)