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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT01365468 |
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Date of registration:
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27/05/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
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Scientific title:
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A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1) |
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Date of first enrolment:
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April 2012 |
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Target sample size:
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9 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01365468 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 2
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Countries of recruitment
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Israel
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Contacts
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Name:
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Novartis Pharmaceuticals |
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Address:
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Telephone:
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Email:
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Affiliation:
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Novartis Pharmaceuticals |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Clinically definite diagnosis of NF1 according to the NIH consensus conference
criteria.
2. Patients must have PN that have the potential to cause significant morbidity, such as
lesions that could compromise the airway or the great vessels, lesions that could
cause nerve compression, lesions that could result in major deformity or significant
cosmetic problems
3. Measurable disease: patient must have at least one measurable PN amenable to
volumetric MRI analysis.
Exclusion Criteria:
1. Chronic treatment with systemic steroids or another immunosuppressive agent.
2. Evidence of an active optic glioma, malignant glioma, malignant peripheral nerve
sheath tumor, or other cancer requiring treatment with chemotherapy or radiation
therapy.
3. Clinical evidence of significantly impaired lung function
4. Pregnancy or breast feeding.
5. Prior therapy with mTOR inhibitors (e.g.sirolimus, temsirolimus, everolimus).
6. No contraindications for MRI assessments
Other protocol-defined inclusion/exclusion criteria may apply
Age minimum:
6 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
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Intervention(s)
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Drug: Everolimus (RAD001)
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Primary Outcome(s)
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Time to Disease Progression (TTP) Based on Change in Volumetric MRI Measurements in Children and Adults (In Stratum I Only)
[Time Frame: Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days)]
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Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only)
[Time Frame: Screening, after course #6, then every 6 months and end of treatment(1 course=28days)]
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Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04
[Time Frame: From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months)]
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Secondary ID(s)
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CRAD001MIL04T
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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