PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children
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ClinicalTrials.gov Identifier: NCT01734512 |
Recruitment Status :
Active, not recruiting
First Posted : November 27, 2012
Results First Posted : February 18, 2021
Last Update Posted : December 27, 2023
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Tracking Information | |||||||
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First Submitted Date ICMJE | November 21, 2012 | ||||||
First Posted Date ICMJE | November 27, 2012 | ||||||
Results First Submitted Date ICMJE | January 14, 2021 | ||||||
Results First Posted Date ICMJE | February 18, 2021 | ||||||
Last Update Posted Date | December 27, 2023 | ||||||
Actual Study Start Date ICMJE | December 13, 2012 | ||||||
Actual Primary Completion Date | January 31, 2020 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
Percentage of Participants With Progression Free Survival at 6 Months [ Time Frame: Up to 6 months ] Response was be determined by bi-dimensional diameters. RECIST criteria will be collected and used for secondary evaluation. Patients will have brain MRI scans with and without gadolinium performed prior to therapy, after every second course in the first year, after every third course in the second year, and at the End of Study visit (if not done within prior 3 months). Spine MRIs should be performed prior to therapy and at the same time points as standard brain MRIs if clinically indicated.
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Original Primary Outcome Measures ICMJE |
Evaluation of efficacy by Progression Free Survival associated with everolimus therapy [ Time Frame: up to 6 months ] Determined by 6-month progression free survival. Response will be determined by bi-dimensional diameters. RECIST criteria will be collected and used for secondary evaluation. Patients will have brain MRI scans with and without gadolinium performed prior to therapy, after every second course in the first year, after every third course in the second year, and at the End of Study visit (if not done within prior 3 months). Spine MRIs should be performed prior to therapy and at the same time points as standard brain MRIs if clinically indicated.
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Change History | |||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children | ||||||
Official Title ICMJE | PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children | ||||||
Brief Summary | This is an open label study of everolimus in children with recurrent or progressive low-grade glioma. | ||||||
Detailed Description | This is an open label study of everolimus in children with recurrent or progressive low-grade glioma. All patients will receive everolimus at a dose of 5 mg/m2/dose daily. An adaptive Simon two-stage design for phase 2 studies of targeted therapies will be used to assess the efficacy primary objective. The proposed treatment with everolimus will be deemed not worthy of further investigation in this patient population if the true PFS at 6-months (PFS6) is less than 50%. If in the first stage, with a combined sample size of 25, there is preliminary evidence to suggest efficacy of everolimus is restricted to patients with PI3K/AKT/mTOR activation as measured by p-S6 positivity, a total of 45 patients will be enrolled and the design will have 81% statistical power to detect a true disease stabilization rate ≥70%. If in the first stage there is preliminary evidence to suggest efficacy of everolimus is independent of PI3K/AKT/mTOR activation, a total of 65 patients will be enrolled and the design will have >95% statistical power to detect a true disease stabilization rate ≥70%. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE | Drug: Everolimus
Everolimus tablet will be taken daily by mouth with water. All patients will be given a dose of 5 mg/m2/dose daily.
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Study Arms ICMJE | Experimental: Everolimus
Everolimus tablet will be taken daily by mouth with water. Twenty-eight days will constitute one course and subsequent courses will immediately follow with no break in the administration of the drug. Dosing is based on the body surface area (BSA) calculated at the beginning of each course of therapy. Patients will also be provided with a drug diary for everolimus. The maximum time on study is 24-months, but if there is no disease progression or adverse events, the patient may speak with a doctor about continuing the treatment off-study.
Intervention: Drug: Everolimus
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||
Actual Enrollment ICMJE |
65 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | July 31, 2024 | ||||||
Actual Primary Completion Date | January 31, 2020 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria: --Patients must have radiographic progressive or recurrent confirmed world health organization (WHO) grade I or II astrocytomas, that was confirmed histologically. Progressive or recurrent disease should be based on MRI according to the definition below. Eligible histologies:
For agents that have known adverse events occurring beyond 7 days after administration, this period should be extended beyond the time during which adverse events are known to occur. This should be discussed with the study chair.
Exclusion Criteria:
Note: A detailed assessment of Hepatitis B/C medical history and risk factors must be done at screening for all patients. Hepatitis B Virus (HBV) DNA and Hepatitis C Virus (HCV) RNA Polymerase chain reaction (PCR) testing are required at screening for all patients with a positive medical history based on risk factors and/or confirmation of prior HBV/HCV infection. |
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Sex/Gender ICMJE |
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Ages ICMJE | 3 Years to 21 Years (Child, Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | United States | ||||||
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Administrative Information | |||||||
NCT Number ICMJE | NCT01734512 | ||||||
Other Study ID Numbers ICMJE | 120817 NCI-2012-02774 ( Registry Identifier: NCI Clinical Trials Reporting Program (CTRP) ) |
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Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | University of California, San Francisco | ||||||
Original Responsible Party | Daphne Haas-Kogan, M.D., University of California, San Francisco, Professor of Radiation Oncology and Neurological Surgery; Program Director and Vice Chair, Department of Radiation Oncology | ||||||
Current Study Sponsor ICMJE | University of California, San Francisco | ||||||
Original Study Sponsor ICMJE | Daphne Haas-Kogan, M.D. | ||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | University of California, San Francisco | ||||||
Verification Date | December 2023 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |