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Preliminary Clinical Study of Entinostat in Children With Malignant Soft Tissue Sarcoma Harboring PAX-family Gene Abnormalities

RecruitingPhase 1Ages 1 Year - 18 YearsAllBeijing Tongren Hospital
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In plain words

What the trial is, and whether it could suit you

Pediatric soft tissue sarcomas (STS) are a group of malignant tumors originating from primitive mesenchymal tissues and account for approximately 7% of childhood cancers. STS are broadly classified into rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS). RMS accounts for approximately one half of pediatric STS. As reported, PAX fusion-positive RMS is more aggressive than fusion-negative RMS, with higher metastatic potential and poorer prognosis. Based on the unmet therapeutic need in pediatric sarcoma with PAX-family abnormalities, this study is intended to evaluate the preliminary safety and antitumor activity of entinostat with or without chemontherapy.

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๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: 1. Age \>1 year and โ‰ค18 years. 2. Histologically confirmed malignant soft tissue sarcoma that is PAX3-FOXO1- or PAX7-FOXO1-fusion positive or has another PAX-family gene abnormality. 3. Clinical disease status: (a) monotherapy cohort: completion of standard backbone chemotherapy and/or radiotherapy with a favorable clinical response (CR or PR); (b) combination cohort: inadequate response following standard chemotherapy and/or radiotherapy, or recurrent/progressive disease. 4. Karnofsky Performance Status (KPS) โ‰ฅ50 or Lansky Performance Status (LPS) โ‰ฅ50. 5. Adequate bone marrow function: ANC โ‰ฅ0.5 ร— 10\^9/L, platelet count โ‰ฅ50 ร— 10\^9/L, hemoglobin โ‰ฅ70 g/L, and WBC โ‰ฅ1.5 ร— 10\^9/L, with values not exceeding the upper limit of normal. 6. Life expectancy of at least 6 months. 7. Written informed consent obtained. Exclusion Criteria: 1. Previous hematopoietic stem cell transplantation or organ transplantation. 2. Participation in another drug clinical trial within 2 months before enrollment. 3. Uncontrolled infection or body temperature โ‰ฅ38ยฐC. 4. Total bilirubin, ALT, and AST \>2.5 ร— the upper limit of normal. 5. Serum creatinine \>2.0 ร— the upper limit of normal. 6. Any other condition considered by the investigator to constitute a contraindication to study participation.
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What it tests

  • Entinostat
  • VAC/VTC chemotherapy
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Where

1 site
Beijing Tongren Hospital
Beijing, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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