Temsirolimus and Valproic Acid in Treating Young Patients With Relapsed Neuroblastoma, Bone Sarcoma, or Soft Tissue Sarcoma

• Status: Terminated
• Trial end date: 2013-03-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: UNC Lineberger Comprehensive Cancer Center
• Collaborator: National Cancer Institute (NCI)
• Treatments: Everolimus; Sirolimus; Valproic Acid

Criteria

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 antihistamines

PRIOR CONCURRENT THERAPY:

• See Disease Characteristics

• More than 2 weeks since prior chemotherapy, immunotherapy, or radiotherapy and recovered

• No concurrent anticonvulsants, including valproic acid

• No concurrent strong inducers or inhibitors of CYP3A4, including grapefruit juice