Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

NCT02584413 | Terminated DMC

• 1 other identifier: 201303074
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

Currently, optic pathway gliomas (OPG) are detected based on abnormal findings made during annual ophthalmologic exams. However, because these exams are annual, it is possible for healthcare providers to miss the point at which a child's vision begins to decline (potentially indicating an OPG). If at-risk children are screened for hypotonia early in life, those children who are hypotonic may undergo magnetic resonance imaging (MRI) to evaluate for OPG before they are showing ophthalmologic symptoms. This would enable healthcare providers to discover vision loss earlier and treat symptomatic OPGs earlier, thereby allowing us a better chance of preventing further vision loss in children with OPGs.

Conditions

Neurofibromatosis Type 1

Outcome Measures

Primary Outcomes (1)

• Predictive accuracy of clinical diagnosis of hypotonia as an indicator of OPG in children with NF1

  * A pediatric nurse practitioner (PNP) or a pediatric physician specializing in NF1, and physical therapist will screen the children for hypotonia. * The MRI scan will show hypotonia if the children have thickening or enlargement of any portion of the optic nerve, optic chiasm, or optic tracts. * The data analysis for this will be descriptive in nature.

  At the time of MRI (1 day)

Secondary Outcomes (2)

• Other features that may be indicatory of OPG in children with NF1

  At the time of MRI (1 day)

• Determine if a physical therapist (PT) can train another clinical professional to accurately diagnose hypotonia

  1 day

Study Arms (1)

Arm 1: MRI of brain with gadolinium contrast

EXPERIMENTAL

-Eligible children whose guardians have consented to their participation will undergo routine clinical brain MRI with gadolinium contrast. The MRI scan will last no more than 45 minutes

Device: Magnetic resonance imaging; Drug: Gadolinium contrast

Interventions

Magnetic resonance imaging DEVICE

-Standard of care

Also known as: MRI

Arm 1: MRI of brain with gadolinium contrast

Gadolinium contrast DRUG

-Standard of care

Arm 1: MRI of brain with gadolinium contrast

Eligibility Criteria

• Age: 1 Year - 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patient must be seen at the St. Louis Children's Hospital NF Clinic
• Diagnosis of NF1
• Between 1 and 7 years of age, inclusive
• Diagnosed with hypotonia
• Legally authorized representative/guardian must be able to understand and willing to sign an IRB-approved informed consent document
• Must have an MRI scan ordered by a treating physician

You may not qualify if:

• Normal tone on clinical exam
• Known allergy to gadolinium or the sedative, propofol, used during MRI
• Poor kidney function defined as a known renal disease or elevated BUN and creatine
• Requiring intubation for anesthesia

Sponsors & Collaborators

• Washington University School of Medicine: lead
• St. Louis Children's Hospital: collaborator

Study Sites (1)

Washington University School of Medicine (St. Louis Children's Hospital)

St Louis, Missouri, 63110, United States

Location

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Magnetic Resonance Imaging; gadolinium compound P760

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

Intervention Hierarchy (Ancestors)

Tomography; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis

Study Officials

• David Gutmann, M.D., Ph.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SCREENING
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2015
• First Posted: October 22, 2015
• Study Start: April 16, 2013
• Primary Completion: February 7, 2019
• Study Completion: February 7, 2019
• Last Updated: September 3, 2019

Record last verified: 2019-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)