Gait in Young Children With NF1

NCT07564622 | Not Yet Recruiting

• 2 other identifiers: 25-010381K23NS146694
• Study Type: observational
• Target: 56
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Neurofibromatosis Type 1

Keywords

NF1; Neurofibromatosis type 1; Gait; Neurodevelopment; gross motor

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

Young Children with NF1

Ambulatory children less than 6 years old with NF1.

Eligibility Criteria

• Age: Up to 71 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• NYU Langone Health: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

MeSH Terms

Conditions

Neurofibromatosis 1

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

Study Officials

• Nicolas Abreu, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Danika Anganoo-Khan

CONTACT

929-455-5629; Danika.anganoo-khan@nyulangone.org

Nicolas Abreu, MD

CONTACT

212-263-7744; Nicolas.abreu@nyulangone.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2026
• First Posted: May 4, 2026
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): January 31, 2030
• Study Completion (Estimated): January 31, 2031
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Immediately following publication. No end date.
• 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).