Sirolimus + Chemotherapy for High-Risk Pediatric Cancers
(AflacST1903 Trial)
Trial Summary
What is the purpose of this trial?
The primary objective of this study is to improve the 2-year progression-free survival in children with high-risk solid tumors who are administered a maintenance regimen with continuous sirolimus administered on a backbone of metronomic chemotherapy following the completion of "standard" therapy, as compared to high-risk solid tumor patients treated with observation alone following completion of "standard" therapy.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications. You cannot be on enzyme-inducing anticonvulsants, potent CYP3A4 inducers or inhibitors, other investigational drugs, or any other anti-cancer agents. If you're on corticosteroids, the dose must be stable or decreasing for the prior 7 days.
What data supports the effectiveness of the drug combination Sirolimus + Chemotherapy for high-risk pediatric cancers?
Research shows that combining rapamycin (another name for sirolimus) with chemotherapy drugs like cyclophosphamide and vincristine can be more effective than using these drugs alone in treating childhood tumors. This combination was well-tolerated and showed promising results in preclinical models.12345
What safety data exists for Sirolimus combined with chemotherapy in pediatric cancers?
A study on sirolimus combined with oral cyclophosphamide and topotecan in children with relapsed solid tumors aimed to find the maximum tolerated dose and noted toxicities, indicating that safety was a key focus. Another study assessed the safety of oral etoposide in children with sarcomas, highlighting its toxicity profile.46789
How is the drug Sirolimus combined with chemotherapy unique for high-risk pediatric cancers?
The combination of Sirolimus with chemotherapy drugs like cyclophosphamide and etoposide is unique because it shows enhanced effectiveness compared to using these chemotherapy drugs alone, particularly in pediatric cancer models. This combination is relatively well tolerated and has shown promising results in preclinical studies, making it a novel approach for treating high-risk pediatric cancers.12346
Research Team
Kathryn Sutton, MD
Principal Investigator
Emory University
Eligibility Criteria
Children and young adults aged 1 to 30 with high-risk solid tumors, such as osteosarcoma or Ewing sarcoma, who have completed standard therapy and are in remission. They must have good performance status, adequate organ function (liver, kidney), normal blood sugar levels, not be pregnant or breastfeeding, and agree to use contraception.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a 12-month course of maintenance chemotherapy with continuous sirolimus, celecoxib, and alternating low-dose oral etoposide and cyclophosphamide
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Celecoxib
- Cyclophosphamide
- Etoposide
- Sirolimus
- VP-16
Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
Emory University
Lead Sponsor
PeachBowl LegACy Fund
Collaborator