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Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

Recruiting
3-120 years
All
Phase N/A

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Overview

The goal of this study is to evaluate the efficacy of using sensorimotor multi-axis automated rotational therapy (SMART) to help treat post concussion syndrome (PCS) in adults.

The investigators hypothesize that patients who include SMART therapy as part of their treatment regimen will improve faster than patients who do not include SMART treatment.

The investigators hypothesize that patients whose treatment approach includes SMART will improve to a greater extent in their primary outcome measures than patients whose treatment approach did not include SMART.

The primary study endpoints are Post Concussion Symptom Scale (PCSS), Headache Impact Severity (HIT-6), Neck Disability Index (NDI), Dizziness Handicap Inventory (DHI), Functional Gait Assessment (FGA), Modified Clinical Test of Sensory Interaction in Balance (CTSIB-m), Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), and the Rivermeade Post-Concussion Symptom Questionnaire. These assessments will be performed before, midway, and after intervention.

Researchers will compare study endpoints to subjects that proceed with standard of care treatments in physical therapy (PT) and speech therapy/cognitive rehabilitation therapy (ST/CRT).

All participants will complete baseline testing in both PT and ST/CRT, and will continue with treatment in each therapy once a week. Intervention subjects will also complete 10 SMART session utilizing GyroStim, at a frequency of 2, 3 or 4 times a week. Follow up testing will happen in PT and ST/CRT after completion of 10 SMART sessions, or during their 6th PT and ST/CRT visit.

Statistical analysis will look compare groups to evaluate efficacy of SMART intervention, as well as evaluate efficacy of therapeutic frequency.

Eligibility

  • INCLUSION CRITERIA:

High-Risk and Low-Risk NF1 Cohorts

  • Age \>= 3 years old
  • Participants with clinical or genetic diagnosis of NF1.
  • Participants with a diagnosis of mosaic or segmental NF1 are also eligible.
  • Individuals may have (High-Risk Cohort) or not have (Low-Risk Cohort) at least one of the following characteristics:
    • Microdeletion or 844-848 missense variants or other variants associated with increased risk of malignant peripheral nervous sheath tumor (MPNST)
    • Family history of MPNST / atypical neurofibromatous neoplasm of unknown biologic potential (ANNUBP) / atypical neurofibromas (ANF)
    • Personal history of MPNST/ANNUBP/ANF or neurofibroma with CDKN2A loss
    • Prior radiation therapy at any site
    • Large plexiform neurofibroma (PN) burden (\>= 350 mL)
    • Presence \>= 1 DNL at baseline
  • The ability of the individual, parent/guardian or Legally Authorized Representative (LAR) to understand and the willingness to sign a written consent document for participation.

EXCLUSION CRITERIA:

High-Risk and Low-Risk NF1 Cohorts

\- Inability or unwillingness to undergo MRI imaging

INCLUSION CRITERIA:

Parent Cohort

  • Parent or guardian of pediatric individuals (8-17 years old) in High-Risk or Low-Risk Cohorts.
  • The ability of the parent/guardian or LAR to understand and the willingness to sign a written consent document for parent/guardian participation in this study.

EXCLUSION CRITERIA:

Parent Cohort

\- None.

Study details
    Neurofibromatosis 1
    Nerve Sheath Neoplasms

NCT06222203

National Cancer Institute (NCI)

13 May 2026

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