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Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

This study is currently recruiting participants.
Verified March 2017 by Washington University School of Medicine
Sponsor:
ClinicalTrials.gov Identifier:
NCT02584413
First Posted: October 22, 2015
Last Update Posted: March 20, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
St. Louis Children's Hospital
Information provided by (Responsible Party):
Washington University School of Medicine
  Purpose
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.

Condition Intervention
Neurofibromatosis Type 1 Device: Magnetic resonance imaging Drug: Gadolinium contrast

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1

Resource links provided by NLM:


Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Predictive accuracy of clinical diagnosis of hypotonia as an indicator of OPG in children with NF1 [ Time Frame: At the time of MRI (1 day) ]
    • 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 [ Time Frame: At the time of MRI (1 day) ]
    • 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 [ Time Frame: 1 day ]
    -PTs use subjective muscle tone, a pull-to-sit test, and the presence or absence of head lag to determine hypotonia.


Estimated Enrollment: 80
Actual Study Start Date: April 16, 2013
Estimated Study Completion Date: April 30, 2023
Estimated Primary Completion Date: April 30, 2023 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Arm 1: MRI of brain with gadolinium contrast
-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
Other Name: MRI
Drug: Gadolinium contrast

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The guardians of children who are deemed hypotonic will be approached by the Clinical Research Assistant in a private room at the St. Louis Children's Hospital NF Clinic and informed about the current study. The guardians will be given the opportunity to discuss the risks and benefits of the study, and the Clinical Research Assistant will ensure that the families feel comfortable with all potential procedures.
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02584413


Contacts
Contact: David Gutmann, M.D., Ph.D. 314-362-7379 gutmannd@wustl.edu

Locations
United States, Missouri
Washington University School of Medicine (St. Louis Children's Hospital) Recruiting
St. Louis, Missouri, United States, 63110
Contact: David Gutmann, M.D., Ph.D.    314-362-7379    gutmannd@wustl.edu   
Principal Investigator: David Gutmann, M.D., Ph.D.         
Sub-Investigator: Robert McKinstry, M.D., Ph.D.         
Sub-Investigator: Joshua Rubin, M.D., Ph.D.         
Sub-Investigator: Joshua Shimony, M.D., Ph.D.         
Sub-Investigator: Stephanie Morris, M.D.         
Sub-Investigator: Courtney Dunn, P.T., D.P.T.         
Sub-Investigator: Courtney Monroe, B.S., CRA         
Sub-Investigator: Feng Gao, Ph.D.         
Sponsors and Collaborators
Washington University School of Medicine
St. Louis Children's Hospital
Investigators
Principal Investigator: David Gutmann, M.D., Ph.D. Washington University School of Medicine
  More Information

Additional Information:
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT02584413     History of Changes
Other Study ID Numbers: 201303074
First Submitted: October 20, 2015
First Posted: October 22, 2015
Last Update Posted: March 20, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibroma
Neurofibromatosis 1
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
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases