Gait in Young Children With NF1

Quantitative Gait Metrics to Assess and Predict Gross Motor Impairment in Young Children With Neurofibromatosis Type 1

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Neurofibromatosis Type 1

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 56

Contacts and Locations

• Study Contact: Name: Danika Anganoo-Khan; Phone Number: 929-455-5629; Email: Danika.anganoo-khan@nyulangone.org
• Study Contact Backup: Name: Nicolas Abreu, MD; Phone Number: 212-263-7744; Email: Nicolas.abreu@nyulangone.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children who are independently ambulatory who meet revised diagnostic criteria for NF1

Description

Inclusion Criteria:

• Children who are independently ambulatory who meet revised diagnostic criteria for NF1
• Children less than or equal to 71 months of age
• Parent/Legal Guardian willing and able to provide parental consent.

Exclusion Criteria:

• Age ≥ 6 years old, inability to walk 10 meters independently, non-NF1 medical illness that alters their physical or neurological abilities that could significantly impact their performance.
• Parent/Legal Guardian not willing and able to provide parental consent

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Young Children with NF1; Ambulatory children less than 6 years old with NF1.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: NYU Langone Health
• Collaborators: National Institute of Neurological Disorders and Stroke (NINDS)
• Investigators: Principal Investigator: Nicolas Abreu, MD, NYU Langone Health

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): January 31, 2030
• Study Completion (Estimated): January 31, 2031

Study Registration Dates

• First Submitted: April 27, 2026
• First Submitted That Met QC Criteria: April 27, 2026
• First Posted (Actual): May 4, 2026

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Gait; Neurodevelopment; NF1; Neurofibromatosis type 1; gross motor
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms; Neuromuscular Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neoplasms by Histologic Type; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Neurofibroma; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurofibromatoses; Neurofibromatosis 1
• Other Study ID Numbers: 25-01038; 1K23NS146694 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Time Frame: Immediately following publication. No end date.
• IPD Sharing Access Criteria: 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).
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

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