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Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

Primary Purpose

Neurofibromatosis Type 1

Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Magnetic resonance imaging
Gadolinium contrast
Sponsored by
Washington University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional screening trial for Neurofibromatosis Type 1

Eligibility Criteria

1 Year - 7 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm 1: MRI of brain with gadolinium contrast

Arm Description

-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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.

Full Information

First Posted
October 20, 2015
Last Updated
August 29, 2019
Sponsor
Washington University School of Medicine
Collaborators
St. Louis Children's Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT02584413
Brief Title
Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma
Official Title
Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1
Study Type
Interventional

2. Study Status

Record Verification Date
August 2019
Overall Recruitment Status
Terminated
Why Stopped
Low accrual rate
Study Start Date
April 16, 2013 (Actual)
Primary Completion Date
February 7, 2019 (Actual)
Study Completion Date
February 7, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Washington University School of Medicine
Collaborators
St. Louis Children's Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis Type 1

7. Study Design

Primary Purpose
Screening
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm 1: MRI of brain with gadolinium contrast
Arm Type
Experimental
Arm Description
-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
Intervention Type
Device
Intervention Name(s)
Magnetic resonance imaging
Other Intervention Name(s)
MRI
Intervention Description
-Standard of care
Intervention Type
Drug
Intervention Name(s)
Gadolinium contrast
Intervention Description
-Standard of care
Primary Outcome Measure Information:
Title
Predictive accuracy of clinical diagnosis of hypotonia as an indicator of OPG in children with NF1
Description
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.
Time Frame
At the time of MRI (1 day)
Secondary Outcome Measure Information:
Title
Other features that may be indicatory of OPG in children with NF1
Description
MRI findings may include other brain tumors or T2 hyperintensities. The data analysis will be descriptive in nature.
Time Frame
At the time of MRI (1 day)
Title
Determine if a physical therapist (PT) can train another clinical professional to accurately diagnose hypotonia
Description
-PTs use subjective muscle tone, a pull-to-sit test, and the presence or absence of head lag to determine hypotonia.
Time Frame
1 day

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Gutmann, M.D., Ph.D.
Organizational Affiliation
Washington University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Washington University School of Medicine (St. Louis Children's Hospital)
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
http://www.siteman.wustl.edu
Description
Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

Learn more about this trial

Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

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