Clinical trial · Interventional
Temsirolimus and Valproic Acid in Treating Young Patients With Relapsed Neuroblastoma, Bone Sarcoma, or Soft Tissue Sarcoma
A Multi-center Phase I Trial of Temsirolimus in Combination With Valproic Acid in Children and Adolescents With Multiply Relapsed Pediatric Solid Tumors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Funding has become unavailable
Summary
Brief summary (as posted)
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.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain and Central Nervous System Tumors | — | UNRESOLVED | — |
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
| Sarcoma | Sarcoma | ONTOLOGY_EXACT | 0.98 |
| Unspecified Childhood Solid Tumor, Protocol Specific | Childhood Solid Neoplasm | ALIAS | 0.85 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Temsirolimus | Drug | Temsirolimus | ALIAS |
| Valproic Acid | Drug | Valproic Acid | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- OTHER
- label
- Single Arm Temsirolimus + Valproic Acid
- description
- Drug: temsirolimus 60-230mg/m2 weekly during each 28 day course, for up to 12 courses Drug: valproic acid (VPA) All patients will be given oral VPA (5 mg/kg, 3 times a day for each 28 day course, up to 12 courses
- interventionNames
- Drug: Temsirolimus
- Drug: Valproic Acid
Primary outcomes (1)
- measure
- Maximum tolerated dose (MTD) of temsirolimus in combination with valproic acid
- timeFrame
- 4 weeks
- description
- The planned starting dose of Temsirolimus is 60mg/M2. The traditional 3+3 design will be used, where the MTD is defined as the dose with the probability of a DLT of 0.20
Secondary outcomes (3)
- measure
- Objective response rate
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
- Maximum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available