Neurofibromatosis 1 and 2 (NF1 and NF2)NCT03672838
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 study testing hand-held dynamometer in people with Neurofibromatosis 1 and 2 (NF1 and NF2), run by NYU Langone Health at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Neurofibromatosis 1 and 2 (NF1 and NF2)
- What is being tested?
- hand-held dynamometer
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Sep 2018to 2 Dec 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Show all 10Show fewer
- 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
Who cannot join
- No orthopedic procedure or other major surgery that could influence extremity strength in past 6 months
- No tibial dysplasia
Trial locations and contacts
1 site in 1 country.
United States1 site
New York School of Medicine
New York, New York, 10016
About this study
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).
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)
- Being tested
- hand-held dynamometer
- Medical subject headings
- Neurofibromatosis 1, Neurofibromatosis 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hand-held dynamometerBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Active comparator | Patients with NF1 Receives: Behavioral: hand-held dynamometer |
| Cohort 2 | Active comparator | Patients with NF2 Receives: Behavioral: hand-held dynamometer |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 day
Secondary outcomes (3)
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.
Measured: 1 day
standard error of measurement of measuring strength in the weak muscles
Measured: 1 day
standard error of measurement of measuring strength in the dominant biceps muscle
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2018
- Study start24 Sep 2018
- Primary completion2 Dec 2019
- Study completion2 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1 and 2 (NF1 and NF2)Studies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kaleb H Yohay, MD, Principal investigator, New York School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03672838
- Sponsor's protocol ID
- 18-00831
- Status last verified
- Mar 2021
- First submitted
- 13 Sep 2018
- Last updated
- 18 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT03672838
- Acronym / sponsor ID
- 18-00831
- Conditions
- Neurofibromatosis 1 and 2 (NF1 and NF2)
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- 24 Sep 2018 (actual)
- Primary completion
- 2 Dec 2019 (actual)
- Study completion
- 2 Dec 2019 (actual)
- First posted
- 17 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Active comparator | Behavioral: hand-held dynamometerPatients with NF1 |
| Cohort 2 | Active comparator | Behavioral: hand-held dynamometerPatients with NF2 |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- For cohort 1:
- For Cohort 2
Key exclusion criteria (2 listed)
- No orthopedic procedure or other major surgery that could influence extremity strength in past 6 months
- No tibial dysplasia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hand-held dynamometerStrength 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. | Behavioural | Cohort 1, Cohort 2 |
Primary outcome
Intra-class correlation coefficient of strength measurement in the clinically identified weak muscle
Time frame: 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.
3 secondary outcomes
Intra-class correlation coefficient of strength measurement in the dominant biceps
Time frame: 1 day
standard error of measurement of measuring strength in the weak muscles
Time frame: 1 day
standard error of measurement of measuring strength in the dominant biceps muscle
Time frame: 1 day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2021.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Neurofibromatosis 1 and 2 (NF1 and NF2)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kaleb H Yohay, MD principal investigator
New York School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing hand-held dynamometer in people with Neurofibromatosis 1 and 2 (NF1 and NF2), run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Intra-class correlation coefficient of strength measurement in the clinically identified weak muscle (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2021.