Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

Neurofibromatosis Type 1 Clinical Trial

Official title:

Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02584413
• Other study ID #: 201303074
• Status: Terminated
• Phase: N/A
• Start date: April 16, 2013
• Est. completion date: February 7, 2019

Study information

• Verified date: August 2019
• Source: Washington University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 29
• Est. completion date: February 7, 2019
• Est. primary completion date: February 7, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 7 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1
• Neurofibromatosis Type 1

Intervention

Device:
• Magnetic resonance imaging
-Standard of care

Drug:
• Gadolinium contrast
-Standard of care

Locations

Country	Name	City	State
United States	Washington University School of Medicine (St. Louis Children's Hospital)	Saint Louis	Missouri

Sponsors (2)

Lead Sponsor	Collaborator
Washington University School of Medicine	St. Louis Children's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Other features that may be indicatory of OPG in children with NF1	MRI findings may include other brain tumors or T2 hyperintensities.; The data analysis will be descriptive in nature.	At the time of MRI (1 day)
Secondary	Determine if a physical therapist (PT) can train another clinical professional to accurately diagnose hypotonia	-PTs use subjective muscle tone, a pull-to-sit test, and the presence or absence of head lag to determine hypotonia.	1 day

External Links

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