Neurofibromatosis Type 1NCT02584413
Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma
A study testing Magnetic resonance imaging and Gadolinium contrast in people with Neurofibromatosis Type 1, run by Washington University School of Medicine at 1 site in United States.
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). Read more about this study
- Who is it for?
- Ages 1 year to 7 years
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- Magnetic resonance imaging and Gadolinium contrast
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Apr 2013to 7 Feb 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine (St. Louis Children's Hospital)
St Louis, Missouri, 63110
About this study
Official title: Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1
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
- Being tested
- Magnetic resonance imaging, Gadolinium contrast
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Magnetic resonance imagingDevice
Also called MRI
-Standard of care
Gadolinium contrastDrug
-Standard of care
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Device: Magnetic resonance imaging; Drug: Gadolinium contrast |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At the time of MRI (1 day)
Secondary outcomes (2)
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.
Measured: At the time of MRI (1 day)
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.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2015 (estimated)
- Study start16 Apr 2013
- Primary completion7 Feb 2019
- Study completion7 Feb 2019
Why it stopped: Low accrual rate
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- St. Louis Children's Hospital
- Responsible party
- Sponsor
- Investigators
- David Gutmann, M.D., Ph.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02584413
- Sponsor's protocol ID
- 201303074
- Status last verified
- Aug 2019
- First submitted
- 20 Oct 2015
- Last updated
- 3 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hypotonia as a Clinical Predictor of Optic Pathway Glioma in Children With Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT02584413
- Acronym / sponsor ID
- 201303074
- Conditions
- Neurofibromatosis Type 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low accrual rate
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- St. Louis Children's Hospital
- Study start
- 16 Apr 2013 (actual)
- Primary completion
- 7 Feb 2019 (actual)
- Study completion
- 7 Feb 2019 (actual)
- First posted
- 22 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1: MRI of brain with gadolinium contrast | Experimental | Device: Magnetic resonance imaging, Drug: 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 |
Who may qualify
- Ages
- 1 year to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Magnetic resonance imagingAlso: MRI-Standard of care | Device | Arm 1: MRI of brain with gadolinium contrast |
| Gadolinium contrast-Standard of care | Drug | Arm 1: MRI of brain with gadolinium contrast |
Primary outcome
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.
2 secondary outcomes
Other features that may be indicatory of OPG in children with NF1
Time frame: At the time of MRI (1 day)
Determine if a physical therapist (PT) can train another clinical professional to accurately diagnose hypotonia
Time frame: 1 day
Results and safety
The study was stopped early (reason given: Low accrual rate). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2019.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Gutmann, M.D., Ph.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Magnetic resonance imaging and Gadolinium contrast in people with Neurofibromatosis Type 1, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Low accrual rate). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2019.