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 : February 18, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pediatric Recurrent Progressive Low-grade Gliomas Pediatric Progressive Low-grade Gliomas||Drug: Everolimus||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||65 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children|
|Actual Study Start Date :||December 13, 2012|
|Actual Primary Completion Date :||January 31, 2020|
|Estimated Study Completion Date :||April 30, 2024|
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.
Everolimus tablet will be taken daily by mouth with water. All patients will be given a dose of 5 mg/m2/dose daily.
- 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.
- Proportion of Participants With Objective Response [ Time Frame: Up to 6 months ]The proportion of participants who demonstrated a complete or partial response as defined by Response Evaluation Criteria in Solid Tumors (RECIST) v1 Criteria where complete Response (CR) is defined as the complete disappearance of all known disease for >=8 weeks. Complete response is dated from time all lesions have disappeared on a stable or decreasing dose of corticosteroids. A Partial Response (PR) is a reduction of at least 50% in the size of all measurable tumor as quantitated by sum of the products of the largest diameters (SLD) of measurable lesions and maintained for >=8 weeks on a stable or decreasing dose of corticosteroids. Partial response is dated from the time of first observation. Overall response also takes into account the response in both the target and non-target lesion, and the appearance of new lesions, where applicable and depend on the achievement of both measurement and confirmation criteria.
- Median Progression Free Survival in Recurrent Pediatric Low-grade Glioma (LGGs) [ Time Frame: Up to 5 years ]Progression free survival will be calculated from date of first treatment to the date of first observation of progressive disease, non-reversible neurological progression or increasing steroid requirements (applies to stable disease only), death due to any cause, or early discontinuation of treatment
- Median Overall Survival in Recurrent Pediatric LGGs [ Time Frame: Up to 5 years ]Overall survival will be calculated from date of original diagnoses to death and also from the date of study registration to death. The latter will be an endpoint for assessment of benefit of this therapy.
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): NCT01734512
|Study Chair:||Daphne Haas-Kogan, MD||Dana-Farber Cancer Institute|
|Principal Investigator:||Sabine Mueller, MD||University of California, San Francisco|