Neurofibromatosis 1 and 2 (NF1 and NF2) Clinical Trial
Official title:
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).
| Verified date | March 2021 |
| Source | NYU Langone Health |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 33 |
| Est. completion date | December 2, 2019 |
| Est. primary completion date | December 2, 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 5 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| United States | New York School of Medicine | New York | New York |
| Lead Sponsor | Collaborator |
|---|---|
| NYU Langone Health |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | 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 | |
| Secondary | standard error of measurement of measuring strength in the weak muscles | 1 day | ||
| Secondary | standard error of measurement of measuring strength in the dominant biceps muscle | 1 day |