The best treatment for recurrent cancers or those that do not respond to therapies is not known. Typically, patients with these cancers receive a combination of cancer drugs (chemotherapy), surgery, or radiation therapy. These treatments can prolong their life but may not offer a long-term cure.
This study proposes using a drug called Sirolimus in combination with common chemotherapy drugs to treat patients with recurrent and refractory solid tumors. Sirolimus has been found to inhibit cell growth and to have anti-tumor activity in pediatric solid tumors in previous studies and, therefore, has the potential to increase the effectiveness of the chemotherapy drugs when given together.
This study wil investigate the highest dose of Sirolimus that can be given orally with other oral chemotherapy drugs. Cohorts of 2 subjects will be started at the minimum dose. The dose will be increased in the next 2 subjects as long as there were no major reactions in the previous groups. This study will also seek to learn more about the side effects of sirolimus when used in this combination and what effects the drug has on the white cells and the immune system. Successful use of this drug will impact the cancer population greatly by providing an increased chance of survival to those with resistant or recurrent cancers.
Who can participate
Age range
30 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* must be \<=30 years of age at time of study enrollment
* histologic verification of malignancy at original diagnosis or relapsis except in patients with intrinsic brain stem tumors, optic pathway gliomas or patients wtih pineal tumors and evaluations of serum or CSF alpha-fetoprotein or beta-HCG
* measurable or evaluable disease
* disease state must be one for which there is no known curative therapy
* Performance level \>=50%
* Patients must have fully recovered from acute toxic effects of all prior chemotherapy, immunotherapy or radiotherapy
* no evidence of acute graft vs. host disease and \>=3 months since transplant
* organ function as defined in eligibility section of protocol
Exclusion Criteria:
* patients cannot be pregnant or breast-feeding
* patients must agree to use of an effective contraceptive method
* no growth factors that support platelet or white cell number or function for at least 7 days prior to enrollment
* patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the prior 7 days are not eligible
* patients receiving any other investigational drugs
* patients receiving any other anti-cancer drugs
* patients who have an uncontrolled infection
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial was a Phase 1 study focused on finding the maximum tolerated dose of sirolimus rather than proving it works, what do the safety and dosing findings from this completed study mean for my child's specific tumor type?
2This trial included a wide range of pediatric solid tumors — from Ewing's sarcoma to neuroblastoma to Wilms tumor — so how relevant are the results to my child's particular diagnosis, and does the data apply equally across all those tumor types?
3Now that this trial is completed, have its findings about sirolimus dosing and tolerability led to any follow-up Phase 2 or Phase 3 studies that my child might be eligible for?
4Given that sirolimus is being studied here as a targeted therapy, would you consider standard chemotherapy or surgery a more proven first step for us before looking at trials built on this research?
5What side effects did children in this Phase 1 trial experience at the doses being studied, and how would those risks compare to what my child is already facing with their current treatment options?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.