Temsirolimus and Valproic Acid in Treating Young Patients With Relapsed Neuroblastoma, Bone Sarco… (NCT01204450) | Clinical Trial Compass
TerminatedPhase 1
Temsirolimus and Valproic Acid in Treating Young Patients With Relapsed Neuroblastoma, Bone Sarcoma, or Soft Tissue Sarcoma
Stopped: Funding has become unavailable
United States7 participantsStarted 2009-11
Plain-language summary
RATIONALE: Drugs such as temsirolimus and valproic acid may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Valproic acid may also stop the growth of solid tumors by blocking blood flow to the tumor.
PURPOSE: This phase I trial is studying the side effects and the best dose of temsirolimus when given together with valproic acid in treating young patients with relapsed neuroblastoma, bone sarcoma, or soft tissue sarcoma.
Who can participate
Age range
2 Years – 18 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.
DISEASE CHARACTERISTICS:
* Histologically confirmed malignant solid tumor at original diagnosis, including the following:
* Neuroblastoma
* Bone sarcomas (primary neuroectodermal tumors/ Ewing sarcoma (PNET/ES), osteosarcoma)
* Soft tissue sarcomas (rhabdosarcoma and related tumors)
* Histologically confirmed of relapsed disease is highly recommended but not mandatory
* Measurable disease according to RECIST
* Refractory or progressive disease after ≥ 1 and ≤ 4 prior chemotherapy regimens
* Patients with neuroblastoma, PNET/ES, or rhabdosarcoma must have failed a cyclophosphamide/topotecan-containing regimen
* Stem cell transplantation, including preparative regimen and post-transplant immunotherapy, is considered to be 1 regimen
PATIENT CHARACTERISTICS:
* Karnofsky performance status (PS) 50-100% (or Lansky PS 50-100%)
* Life expectancy ≥ 8 weeks
* ANC ≥ 750/mm\^3
* Platelet count ≥ 75,000/mm\^3 (transfusion independent)
* Hemoglobin 8.0 g/dL (may receive RBC transfusions)
* Patients with tumor metastatic to bone marrow are allowed to receive transfusions to maintain hemoglobin and platelet counts
* Serum creatinine normal
* Total bilirubin ≤ 1.5 times upper limit of normal (ULN) OR direct bilirubin \< 1.0 mg/dL (if total bilirubin \> 2.0 mg/dL)
* ALT \< 5 times ULN
* Negative pregnancy test
* Not pregnant or nursing
* Fertile patients must use effective contraception
* Families must be able to give consent in English or Spanish
* No allergy to H1 antihista…
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.
What they're measuring
1
Maximum tolerated dose (MTD) of temsirolimus in combination with valproic acid