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| ID | Type | Description | Link |
|---|---|---|---|
| 1K23NS146694 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Neurological Disorders and Stroke (NINDS) | NIH |
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The objective of this study is to develop office-based tools to quantify gait in young children with NF1 that reflect overall gross motor impairment and predict future gross motor difficulties.
In this prospective observational study of ambulatory children less than six years old with NF1, investigators will evaluate gait speed as the fastest time to walk 10 meters and interlimb coordination derived from artificial intelligence-based pose estimates from video. Investigators will conduct office-based gait assessments with video, clinical exams, and neurodevelopmental evaluations during routine medical visits at baseline, 12 months, and 24 months at one of the largest NF1 centers in the U.S., the NYU Comprehensive Neurofibromatosis Center.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Young Children with NF1 | Ambulatory children less than 6 years old with NF1. |
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| Measure | Description | Time Frame |
|---|---|---|
| Developmental Assessment of Young Children (DAYC-2) Score | Assessment of child neurodevelopmental function in which novel tasks are performed; each task is pass/fail and scored 0 or 1 point. A raw score is calculated and converted to a standard score as follows: <70 (Very Poor); 70-79 (Poor); 80-89 (Below Average); 90-110 (Average); 111-120 (Above Average); 121-130 (Superior); >130 (Very Superior); higher scores indicate greater function. | Baseline, 12 Months, 24 Months |
| Vineland Adaptive Behavior Scale Score | Comprehensive, standardized measure of an individual's personal and social sufficiency to assist in diagnosing intellectual and developmental disabilities, autism spectrum disorder, and ADHD. The raw score is converted to a standardized score with a mean of 100 and standard deviation of 15; higher scores indicate greater daily functioning. | Baseline, 12 Months, 24 Months |
| 10-Meter Walk Test Score | Measure of gait; how quickly a participant can complete a 10-meter walk. | Baseline, 12 Months, 24 Months |
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Inclusion Criteria:
Exclusion Criteria:
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Children who are independently ambulatory who meet revised diagnostic criteria for NF1
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Danika Anganoo-Khan | Contact | 929-455-5629 | Danika.anganoo-khan@nyulangone.org | |
| Nicolas Abreu, MD | Contact | 212-263-7744 | Nicolas.abreu@nyulangone.org |
| Name | Affiliation | Role |
|---|---|---|
| Nicolas Abreu, MD | NYU Langone Health | Principal Investigator |
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The de-identified participant data from the final research dataset will be shared upon reasonable request immediately following publication, with no end date, or as required by a condition of awards or supporting agreements. The protocol and statistical analysis plan will be posted on Clinicaltrials.gov only as required by federal regulation or supporting awards and agreements.
Immediately following publication. No end date.
The investigator who proposed to use the data will be granted access upon reasonable request. Data are available for 5 years at a third party website (Link to be included).
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| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
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| D009370 |
| Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |