Pediatric Recurrent Progressive Low-grade GliomasPhase 2NCT01734512
PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children
A Phase 2 study testing Everolimus in people with Pediatric Recurrent Progressive Low-grade Gliomas and Pediatric Progressive Low-grade Gliomas, run by University of California, San Francisco at 18 sites in United States.
Trial summary
This is an open label study of everolimus in children with recurrent or progressive low-grade glioma. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Pediatric Recurrent Progressive Low-grade Gliomas, Pediatric Progressive Low-grade Gliomas
- What is being tested?
- Everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 18 sitesUnited States
- Study dates
- 13 Dec 2012to 31 Jul 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- --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:
- Pilocytic Astrocytoma - 90600112
- Astrocytoma, Low Grade (Fibrillary astrocytoma, WHO Grade 2) - 10065886
- Astrocytoma, Low Grade (Low-grade Astrocytoma, not otherwise specified (NOS), WHO Grade 2) - 10003571
- Tissue from the initial diagnosis or recurrence must be made available for correlative testing.
Show all 26Show fewer
- Patients must have measurable disease, defined as at least one lesion that can be accurately measured in at least two dimensions on MRI.
- Patients may have had treatment (chemotherapy and/or radiotherapy) for any number of relapses prior to this recurrence.
- Patients must have received their last dose of myelosuppressive anticancer chemotherapy at least three (3) weeks prior to study registration or at least six (6)weeks of nitrosourea.
- Patients must have received their last dose of other investigational or biological agent > 7 days prior to study entry.
- 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.
- If patients received prior monoclonal antibody treatment, at least three half-lives must be elapsed by the time of treatment initiation. These patients should also be discussed with the study chair.
- Patients must have received their last fraction of craniospinal or focal radiation to primary tumor or other sites >12 weeks (3 months) prior to registration.
- --Age ≥3 and ≤21 years.
- Because no dosing or adverse event data are currently available on the use of everolimus in patients <3 years of age, these young children are excluded from this study.
- Life expectancy of greater than 8 weeks.
- Patients must be able to swallow pills.
- Patient must have a Karnofsky (if ≥ 16 years of age) or Lansky Performance score (if ≤ 16 years of age) of ≥50 by the time of registration.
- Patients must have adequate bone marrow function (ANC ≥ 1,000/mm3, platelet count of ≥ 100,000/mm3, and hemoglobin ≥ 9 gm/dL) before starting therapy. Eligibility level for hemoglobin may be reached by transfusion.
- International Normalized Ratio (INR) ≤1.5. (Anticoagulation is allowed if target INR ≤ 1.5 on a stable dose of warfarin or on a stable dose of low molecular weight (LMW) heparin for >2 weeks at time of randomization).
- Patients must have adequate liver function (SGPT/alanine aminotransferase (ALT) ≤ 2.5 times ULN and bilirubin ≤ 1.5 times ULN) before starting therapy.
- Patients must have adequate renal function (serum creatinine ≤ 1.5 times institutional ULN for age or Glomerular filtration rate (GFR) ≥ 70 ml/min/1.73 m2) before starting therapy.
- Patients must have cholesterol level <350 mg/dL and triglycerides < 400 mg/dL before starting therapy. In case one or both of these are exceeded, the patient can only be included after initiation of appropriate lipid lowering medication and documentation of cholesterol < 350mg/dL and triglycerides < 400mg/dl before start of therapy.
- Patients must have normal pulmonary function testing for age based on pulse oximetry.
- The effects of everolimus on the developing human fetus at the recommended therapeutic dose are unknown. For this reason and because everolimus are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Female patients of child bearing potential must not be breastfeeding or pregnant as evidenced by a negative pregnancy test.
Who cannot join
- Patients with primary spinal cord tumors
- Patients receiving concomitant medication that may interfere with study outcome. For example, patients cannot be on enzyme inducing anticonvulsants like phenytoin.
- Patients should not receive immunization with attenuated live vaccines within one week of study entry or during study period. Close contact with those who have received attenuated live vaccines should be avoided during treatment with everolimus. Examples of live vaccines include intranasal influenza, measles, mumps, rubella, oral polio, bacille Calmette-Guerin (BCG), yellow fever, varicella and TY21a typhoid vaccines
- Hepatitis B/C blood test must be done at screening for all patients. Patients who test positive for Hepatitis C antibodies and the Hepatitis B antigen are ineligible.
- A known history of HIV seropositivity. HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with everolimus. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
- Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
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- Patients may not have therapy for this recurrence (including radiation).
- Patients who do not have measurable disease on MRI.
- Patients who have been previously treated with an mTOR inhibitor.
- Patients with a known hypersensitivity to everolimus or other rapamycins (e.g. sirolimus, temsirolimus).
- Patients receiving any other concurrent anticancer or investigational therapy.
- Patients with any clinically significant unrelated systemic illness that would compromise the patient's ability to tolerate protocol therapy.
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection.
- Patients with inability to return for follow-up visits to assess toxicity to therapy.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Patients with a history of any other cancer (except non-melanoma skin cancer or carcinoma in situ of the cervix), unless in complete remission and off of all therapy for that disease for a minimum of 3 years.
- 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.
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
Johns Hopkins University
Baltimore, Maryland, 21218
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32611
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of California, Los Angeles
Los Angeles, California, 90027
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Children's Hospitals and Clinics of Minneapolis
Minneapolis, Minnesota, 55404
Children's Hospital Oakland
Oakland, California, 94609
The Children's Hospital Of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84112
University of California, San Diego Rady Children's Hospital
San Diego, California, 92123
University of California, San Francisco
San Francisco, California, 94158
University of Washington, Seattle
Seattle, Washington, 98195
St. Louis Children's Hospital, Washington University
St Louis, Missouri, 63130
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
This is an open label study of everolimus in children with recurrent or progressive low-grade glioma.
Read the 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%.
- Conditions
- Pediatric Recurrent Progressive Low-grade Gliomas, Pediatric Progressive Low-grade Gliomas
- Being tested
- Everolimus
- Keywords
- pediatric recurrent progressive low-grade gliomas, pediatric progressive low-grade gliomas, everolimus, mTOR inhibition
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EverolimusDrug
Also called Zortress, Afinitor
Everolimus tablet will be taken daily by mouth with water. All participants will be given a dose of 5 mg/m2/dose daily.
| Study group | Role | What they receive |
|---|---|---|
| Everolimus | Experimental | 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. Receives: Drug: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Progression Free Survival at 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.
Measured: Up to 6 months
Secondary outcomes (4)
Proportion of Participants With Objective Response
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.
Measured: Up to 6 months
Median Progression Free Survival in Recurrent Pediatric Low-grade Glioma (LGGs)
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
Measured: Up to 5 years
Median Overall Survival From Time of Diagnosis
Overall survival in participants with recurrent pediatric low-grade glioma (LGGs) will be calculated from date of original diagnoses to death using Kaplan Meier method. Median time to death and 95% confidence interval will be reported.
Measured: Up to 5 years
Median Overall Survival From Time of Enrollment
Overall survival in participants with recurrent pediatric low-grade glioma (LGGs) will be calculated from date of study registration to death using Kaplan Meier method. Median time to death and 95% confidence interval will be reported.
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2012 (estimated)
- Study start13 Dec 2012
- Primary completion31 Jan 2020
- Study completion31 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pediatric Recurrent Progressive Low-grade GliomasStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals, Pacific Pediatric Neuro-Oncology Consortium, The Pediatric Low Grade Astrocytoma (PLGA) Foundation
- Responsible party
- Sponsor
- Investigators
- Daphne Haas-Kogan, MD, Study chair, Dana-Farber Cancer InstituteSabine Mueller, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01734512
- Sponsor's protocol ID
- 120817
- Other IDs
- NCI-2012-02774
- Status last verified
- Sep 2025
- First submitted
- 21 Nov 2012
- Last updated
- 9 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Haas-Kogan DA, Aboian MS, Minturn JE, Leary SES, Abdelbaki MS, Goldman S, Elster JD, Kraya A, Lueder MR, Ramakrishnan D, von Reppert M, Liu KX, Rokita JL, Resnick AC, Solomon DA, Phillips JJ, Prados M, Molinaro AM, Waszak SM, Mueller S. Everolimus for Children With Recurrent or Progressive Low-Grade Glioma: Results From the Phase II PNOC001 Trial. J Clin Oncol. 2024 Feb 1;42(4):441-451. doi: 10.1200/JCO.23.01838. Epub 2023 Nov 17.
PubMed 37978951
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children
- ClinicalTrials.gov ID
- NCT01734512
- Acronym / sponsor ID
- 120817
- Conditions
- Pediatric Recurrent Progressive Low-grade Gliomas, Pediatric Progressive Low-grade Gliomas
- Keywords
- pediatric recurrent progressive low-grade gliomas, pediatric progressive low-grade gliomas, everolimus, mTOR inhibition
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals, Pacific Pediatric Neuro-Oncology Consortium, The Pediatric Low Grade Astrocytoma (PLGA) Foundation
- Study start
- 13 Dec 2012 (actual)
- Primary completion
- 31 Jan 2020 (actual)
- Study completion
- 31 Jul 2024 (actual)
- First posted
- 27 Nov 2012 (estimated)
- Results
- Posted 18 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus | Experimental | Drug: EverolimusEverolimus 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. |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (26 listed)
- --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.
- Pilocytic Astrocytoma - 90600112
- Astrocytoma, Low Grade (Fibrillary astrocytoma, WHO Grade 2) - 10065886
- Astrocytoma, Low Grade (Low-grade Astrocytoma, not otherwise specified (NOS), WHO Grade 2) - 10003571
- 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.
Key exclusion criteria (17 listed)
- Patients with primary spinal cord tumors
- Patients receiving concomitant medication that may interfere with study outcome. For example, patients cannot be on enzyme inducing anticonvulsants like phenytoin.
- Patients should not receive immunization with attenuated live vaccines within one week of study entry or during study period. Close contact with those who have received attenuated live vaccines should be avoided during treatment with everolimus. Examples of live vaccines include intranasal influenza, measles, mumps, rubella, oral polio, bacille Calmette-Guerin (BCG), yellow fever, varicella and TY21a typhoid vaccines
- Hepatitis B/C blood test must be done at screening for all patients. Patients who test positive for Hepatitis C antibodies and the Hepatitis B antigen are ineligible.
- A known history of HIV seropositivity. HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with everolimus. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
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 |
|---|---|---|
| EverolimusAlso: Zortress, AfinitorEverolimus tablet will be taken daily by mouth with water. All participants will be given a dose of 5 mg/m2/dose daily. | Drug | Everolimus |
Primary outcome
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.
4 secondary outcomes
Proportion of Participants With Objective Response
Time frame: Up to 6 months
Median Progression Free Survival in Recurrent Pediatric Low-grade Glioma (LGGs)
Time frame: Up to 5 years
Median Overall Survival From Time of Diagnosis
Time frame: Up to 5 years
Median Overall Survival From Time of Enrollment
Time frame: Up to 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37978951Haas-Kogan DA, Aboian MS, Minturn JE, Leary SES, Abdelbaki MS, Goldman S, Elster JD, Kraya A, Lueder MR, Ramakrishnan D, von Reppert M, Liu KX, Rokita JL, Resnick AC, Solomon DA, Phillips JJ, Prados M, Molinaro AM, Waszak SM, Mueller S. Everolimus for Children With Recurrent or Progressive Low-Grade Glioma: Results From the Phase II PNOC001 Trial. J Clin Oncol. 2024 Feb 1;42(4):441-451. doi: 10.1200/JCO.23.01838. Epub 2023 Nov 17.
- 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.
Daphne Haas-Kogan, MD study chair
Dana-Farber Cancer Institute
Sabine Mueller, MD principal investigator
University of California, San Francisco
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Everolimus in people with Pediatric Recurrent Progressive Low-grade Gliomas and Pediatric Progressive Low-grade Gliomas, run by University of California, San Francisco at 18 sites 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 26 inclusion and 17 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: finished, as of the sponsor's last update on 9 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Progression Free Survival at 6 Months (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01734512, which also lists 1 linked publication.
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 9 Sep 2025.