CancerPhase 2AflacST1502NCT02574728
Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
A Phase 2 study testing Sirolimus, Celecoxib and Etoposide in people with Cancer, run by Emory University at 6 sites in United States.
Trial summary
This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors. Read more about this study
- Who is it for?
- Ages 12 months to 30 years
- Conditions the study is for
- Cancer
- What is being tested?
- Sirolimus, Celecoxib and Etoposide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 6 sitesUnited States
- Study dates
- Jun 2015to Jun 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 months to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants with any of the following tumors who have experienced relapse following front-line therapy, or who are refractory to front-line therapy, and participants with tumors that carry a poor prognosis and have no known standard curative therapy
- Brain tumors of all World Health Organization (WHO) grades, except diffuse intrinsic pontine glioma (DIPG) - enrollment in the brain tumor stratum is closed
- Extracranial solid tumors including histiocytoses
- Participants must have had a histologic verification of malignancy at original diagnosis or relapse, except in participants with optic pathway gliomas, or participants with pineal tumors and elevations of serum or cerebrospinal fluid (CSF) alpha-fetoprotein (AFP) or beta-human chorionic gonadotropin (beta-HCG)
- Tissue blocks or slides must be sent
- Participants must have radiographically measurable disease at the time of study enrollment to be eligible. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG+) evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as tumor that is measurable (≥ 10 mm) in two perpendicular diameters on MRI and visible on more than one slice. For all patients, tumors that are located in a previously irradiated area may be considered measurable if the lesion has shown tumor growth after radiation or has been biopsied and proven to have active disease.
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- Participant's current disease state must be one for which there is no known curative therapy
- Karnofsky performance level of greater than or equal to 50 percent for participants who are greater than 16 years of age at the time of screening
- Lansky performance level of greater than or equal to 50 percent for participants who are less than or equal to 16 years of age at the time of screening
- Fully recovered from acute toxic effects of all prior anti-cancer therapy
- Adequate bone marrow function as deemed by the protocol at the time of screening
- Adequate renal function as deemed by the study protocol at the time of screening
- Adequate liver function as deemed by the study protocol at the time of screening
- Serum triglyceride level ≤300 mg/dL and serum cholesterol ≤ 300 mg/dL
- Random or fasting blood glucose within the upper normal limits for age
- Adequate pulmonary function as deemed by the study protocol at the time of screening
Who cannot join
- Women who are currently pregnant or breastfeeding
- Receiving corticosteroids who have not been on a stable dose for at least 7 days
- Currently receiving enzyme inducing anticonvulsants
- Currently receiving receiving potent CYP3A4 (enzyme) inducers or inhibitors
- Currently receiving another investigational drug
- Currently receiving any other anti-cancer agents
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- The use of cannabis oil is prohibited during the first 2 cycles of this protocol. Patients must be off of cannabis oil for 3 days prior to enrollment.
- Uncontrolled infection
- Participants who in the opinion of the investigator may not be able to comply with the safety monitoring requirements
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Children's Healthcare of Atlanta-Egleston
Atlanta, Georgia, 30322
Children's Healthcare of Atlanta, Scottish Rite
Atlanta, Georgia, 30342
University of Virginia Health System
Charlottesville, Virginia, 22908
Children's Mercy Hospital
Kansas City, Missouri, 64108
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803
About this study
Official title: AflacST1502: A Phase II Study of Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors.
Read the detailed description
This study aims to learn if the combination of oral sirolimus once daily with celecoxib, and with oral etoposide alternating every 21 days with oral cyclophosphamide (metronomic chemotherapy) is effective in shrinking relapsed or refractory tumors in pediatric participants. In addition, this study seeks to learn the length of time this combination can keep the tumor from growing, learn more about the side effects of sirolimus when used in this combination, and to learn if the sirolimus is working by evaluating blood and tumor tissue.
- Conditions
- Cancer
- Being tested
- Sirolimus, Celecoxib, Etoposide, Cyclophosphamide
- Keywords
- Pediatrics, Brain Tumors, Medulloblastoma, Ependymoma, Atypical teratoid rhabdoid tumor (ATRT), Pineoblastoma, Germ cell tumors (CNS and non-CNS), Neuroblastoma, Osteosarcoma, Ewing's Sarcoma, Rhabdomyosarcoma, Wilms Tumors, Soft Tissue Sarcomas, Langerhans cell histiocytosis (LCH), Histiocytic disorders, Rare pediatric solid tumors, Carcinomas
- Medical subject headings
- Neoplasms, Brain Neoplasms, Medulloblastoma, Ependymoma, Rhabdoid Tumor, Pinealoma, Neoplasms, Germ Cell and Embryonal, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune, rapamycin
The starting dose for sirolimus is 2 mg/m2 once daily. The dose of sirolimus will be individually adjusted to achieve a target serum trough concentration in the range of 10-15 ng/ml. Sirolimus will be given by mouth every day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles.
CelecoxibDrug
Celecoxib 100 mg will be given by mouth twice a day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles.
EtoposideDrug
Also called Etopophos, Toposar
Etoposide 50 mg/m2 (maximum dose 100 mg) will be given daily by mouth for the first 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles.
CyclophosphamideDrug
Also called Cytoxan
Cyclophosphamide 2.5 mg/Kg (maximum dose 100 mg) will be given daily by mouth for the second 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles.
| Study group | Role | What they receive |
|---|---|---|
| Oral sirolimus, celecoxib, etoposide, and cyclophosphamide | Experimental | Participants in this group will receive oral sirolimus and celecoxib in addition to cycles of oral etoposide and cyclophosphamide for up to two years. Receives: Drug: Sirolimus; Drug: Celecoxib; Drug: Etoposide; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Radiographic response to treatment for solid tumors
Radiographic response to treatment will be assessed by Response Evaluation Criteria in Solid Tumors (RECIST) for participants with solid tumors via CT or MRI scan. A complete response (CR) is a disappearance of all target and non-target lesions. Partial response (PR) is at least a 30% decrease in the disease measurement compared to the baseline measurement. Stable disease (SD) is neither sufficient shrinkage to qualify for partial response (PR) nor sufficient increase to qualify for progressive disease (PD) taking as reference the smallest disease measurement since baseline. Progressive disease (PD) is at least a 20% increase in the disease measurement compared to baseline, or the appearance of one or more new lesions, or evidence of laboratory or clinical progression.
Measured: Baseline, End of Treatment (Up to 2 years)
Radiographic response to treatment for central nervous system (CNS) tumors
Radiographic response to treatment will be assessed for participants with CNS tumors via CT or MRI scan. Response criteria are assessed based on the lesion of the longest diameter and its longest perpendicular diameter. Development of new disease or progression in any established lesions is considered progressive disease, regardless of response in other lesions. Complete response (CR) is the the disappearance of all target lesions. Partial response (PR) is greater than or equal to 50% decrease decrease in the sum of the products of the two perpendicular diameters of all target lesions, taking into reference the baseline measurement. Stable disease (SD) is neither a sufficient decrease in the sum of the products of the two perpendicular diameters of all target lesions to qualify for PR, nor sufficient increase in a single target lesion to qualify for progressive disease (PD).
Measured: Baseline, End of Treatment (Up to 2 years)
Secondary outcomes (1)
Number of adverse events
The adverse events associated with sirolimus in combination with metronomic chemotherapy administered on this schedule will be defined and evaluated throughout the treatment period.
Measured: Baseline, End of Treatment (Up to 2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2015 (estimated)
- Study startJun 2015
- Primary completionJun 2026 (estimated)
- Study completionJun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CancerStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Cannonball Kids' Cancer Foundation, Hyundai Hope On Wheels
- Responsible party
- Principal investigator, William T Cash, Emory University
- Investigators
- Thomas Cash, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02574728
- Sponsor's protocol ID
- IRB00082488
- Acronym
- AflacST1502
- Status last verified
- Feb 2026
- First submitted
- 2 Oct 2015
- Last updated
- 2 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AflacST1502: A Phase II Study of Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
- ClinicalTrials.gov ID
- NCT02574728
- Acronym / sponsor ID
- AflacST1502 / IRB00082488
- Conditions
- Cancer
- Keywords
- Pediatrics, Brain Tumors, Medulloblastoma, Ependymoma, Atypical teratoid rhabdoid tumor (ATRT), Pineoblastoma, Germ cell tumors (CNS and non-CNS), Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Cannonball Kids' Cancer Foundation, Hyundai Hope On Wheels
- Study start
- Jun 2015
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Jun 2026 (estimated)
- First posted
- 14 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.
This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors.
Detailed description
This study aims to learn if the combination of oral sirolimus once daily with celecoxib, and with oral etoposide alternating every 21 days with oral cyclophosphamide (metronomic chemotherapy) is effective in shrinking relapsed or refractory tumors in pediatric participants. In addition, this study seeks to learn the length of time this combination can keep the tumor from growing, learn more about the side effects of sirolimus when used in this combination, and to learn if the sirolimus is working by evaluating blood and tumor tissue.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oral sirolimus, celecoxib, etoposide, and cyclophosphamide | Experimental | Drug: Sirolimus, Drug: Celecoxib, Drug: Etoposide, Drug: CyclophosphamideParticipants in this group will receive oral sirolimus and celecoxib in addition to cycles of oral etoposide and cyclophosphamide for up to two years. |
Who may qualify
- Ages
- 12 months to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Participants with any of the following tumors who have experienced relapse following front-line therapy, or who are refractory to front-line therapy, and participants with tumors that carry a poor prognosis and have no known standard curative therapy
- Participants must have had a histologic verification of malignancy at original diagnosis or relapse, except in participants with optic pathway gliomas, or participants with pineal tumors and elevations of serum or cerebrospinal fluid (CSF) alpha-fetoprotein (AFP) or beta-human chorionic gonadotropin (beta-HCG)
- Tissue blocks or slides must be sent
- Participants must have radiographically measurable disease at the time of study enrollment to be eligible. Patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG+) evaluable disease are eligible. Measurable disease in patients with CNS involvement is defined as tumor that is measurable (≥ 10 mm) in two perpendicular diameters on MRI and visible on more than one slice. For all patients, tumors that are located in a previously irradiated area may be considered measurable if the lesion has shown tumor growth after radiation or has been biopsied and proven to have active disease.
- Participant's current disease state must be one for which there is no known curative therapy
Key exclusion criteria (9 listed)
- Women who are currently pregnant or breastfeeding
- Receiving corticosteroids who have not been on a stable dose for at least 7 days
- Currently receiving enzyme inducing anticonvulsants
- Currently receiving receiving potent CYP3A4 (enzyme) inducers or inhibitors
- Currently receiving another investigational drug
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 |
|---|---|---|
| SirolimusAlso: Rapamune, rapamycinThe starting dose for sirolimus is 2 mg/m2 once daily. The dose of sirolimus will be individually adjusted to achieve a target serum trough concentration in the range of 10-15 ng/ml. Sirolimus will be given by mouth every day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles. | Drug | Oral sirolimus, celecoxib, etoposide, and cyclophosphamide |
| CelecoxibCelecoxib 100 mg will be given by mouth twice a day for six weeks (every six weeks is called one cycle) for up to two years or 16 cycles. | Drug | Oral sirolimus, celecoxib, etoposide, and cyclophosphamide |
| EtoposideAlso: Etopophos, ToposarEtoposide 50 mg/m2 (maximum dose 100 mg) will be given daily by mouth for the first 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles. | Drug | Oral sirolimus, celecoxib, etoposide, and cyclophosphamide |
| CyclophosphamideAlso: CytoxanCyclophosphamide 2.5 mg/Kg (maximum dose 100 mg) will be given daily by mouth for the second 3 weeks of a 6 week cycle. Six week cycles will be repeated for up to two years or 16 cycles. | Drug | Oral sirolimus, celecoxib, etoposide, and cyclophosphamide |
Primary outcome
Radiographic response to treatment for solid tumors
Time frame: Baseline, End of Treatment (Up to 2 years)
Radiographic response to treatment will be assessed by Response Evaluation Criteria in Solid Tumors (RECIST) for participants with solid tumors via CT or MRI scan. A complete response (CR) is a disappearance of all target and non-target lesions. Partial response (PR) is at least a 30% decrease in the disease measurement compared to the baseline measurement. Stable disease (SD) is neither sufficient shrinkage to qualify for partial response (PR) nor sufficient increase to qualify for progressive disease (PD) taking as reference the smallest disease measurement since baseline. Progressive disease (PD) is at least a 20% increase in the disease measurement compared to baseline, or the appearance of one or more new lesions, or evidence of laboratory or clinical progression.
Radiographic response to treatment for central nervous system (CNS) tumors
Time frame: Baseline, End of Treatment (Up to 2 years)
Radiographic response to treatment will be assessed for participants with CNS tumors via CT or MRI scan. Response criteria are assessed based on the lesion of the longest diameter and its longest perpendicular diameter. Development of new disease or progression in any established lesions is considered progressive disease, regardless of response in other lesions. Complete response (CR) is the the disappearance of all target lesions. Partial response (PR) is greater than or equal to 50% decrease decrease in the sum of the products of the two perpendicular diameters of all target lesions, taking into reference the baseline measurement. Stable disease (SD) is neither a sufficient decrease in the sum of the products of the two perpendicular diameters of all target lesions to qualify for PR, nor sufficient increase in a single target lesion to qualify for progressive disease (PD).
1 secondary outcome
Number of adverse events
Time frame: Baseline, End of Treatment (Up to 2 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
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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.
Thomas Cash, MD principal investigator
Emory University
6 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 Sirolimus, Celecoxib and Etoposide in people with Cancer, run by Emory University at 6 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 16 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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?
This study is still under way. Results are not available in the source record yet.
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?
Radiographic response to treatment for solid tumors (time frame: Baseline, End of Treatment (Up to 2 years)). The study lists 2 primary outcomes in all. 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 2 Mar 2026.