Soft Tissue SarcomaPhase 1NCT07888114
Preliminary Clinical Study of Entinostat in Children With Malignant Soft Tissue Sarcoma Harboring PAX-family Gene Abnormalities
A Phase 1 study testing Entinostat and VAC/VTC chemotherapy in people with Soft Tissue Sarcoma, run by Beijing Tongren Hospital at 1 site in China.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Soft Tissue Sarcoma
- What is being tested?
- Entinostat and VAC/VTC chemotherapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteChina
- Study dates
- 15 Mar 2025to 31 Mar 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age >1 year and ≤18 years.
- Histologically confirmed malignant soft tissue sarcoma that is PAX3-FOXO1- or PAX7-FOXO1-fusion positive or has another PAX-family gene abnormality.
- 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.
- Karnofsky Performance Status (KPS) ≥50 or Lansky Performance Status (LPS) ≥50.
- Adequate bone marrow function: ANC ≥0.5 × 10^9/L, platelet count ≥50 × 10^9/L, hemoglobin ≥70 g/L, and WBC ≥1.5 × 10^9/L, with values not exceeding the upper limit of normal.
- Life expectancy of at least 6 months.
- Written informed consent obtained.
Who cannot join
- Previous hematopoietic stem cell transplantation or organ transplantation.
- Participation in another drug clinical trial within 2 months before enrollment.
- Uncontrolled infection or body temperature ≥38°C.
- Total bilirubin, ALT, and AST >2.5 × the upper limit of normal.
- Serum creatinine >2.0 × the upper limit of normal.
- Any other condition considered by the investigator to constitute a contraindication to study participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing Tongren Hospital
Beijing, Beijing Municipality, 100730
Yi Zhang, 010-58266699, yichang-1@163.com
About this study
Official title: A Single-Center Clinical Study Evaluating the Safety and Antitumor Activity of Entinostat in Children With Malignant Soft Tissue Sarcoma Harboring PAX-family Gene Abnormalities
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.
Read the detailed description
Cohort 1: Entinostat Monotherapy cohort After completion of prior chemotherapy and/or radiotherapy, patients who have achieved CR/PR will receive entinostat as consolidation therapy.
Cohort 2: Entinostat Combination Cohort Patients with recurrent or progressive disease will receive entinostat in combination with alternating VAC/VTC chemotherapy. If prior treatment with the VAC/VTC regimen is ineffective because of resistance or is not tolerated for safety reasons, the investigator team will discuss and develop an individualized combination chemotherapy regimen.
- Conditions
- Soft Tissue Sarcoma
- Being tested
- Entinostat, VAC/VTC chemotherapy
- Keywords
- soft tissue sarcoma;, rhabdomyosarcoma;, entinostat;, histone deacetylase inhibitor, PAX-FOXO1
- Medical subject headings
- Sarcoma, Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EntinostatDrug
For patients aged 1-3 years or weighing <40 kg: starting dose 3 mg orally once weekly, maintained at 3 mg.
For patients aged >3 years or weighing ≥40 kg: starting dose 3 mg orally once weekly, with gradual escalation to a maintenance dose of 5 mg, or a starting dose of 5 mg.
VAC/VTC chemotherapyDrug
VAC regimen: vincristine 1.5 mg/m² on Day 1; dactinomycin 12-15 µg/kg on Days 1-5; and cyclophosphamide 300 mg/m² on Days 1-4.
VTC regimen: vincristine 1.5 mg/m² on Day 1; topotecan 1.2 mg/m² on Days 1-5; and cyclophosphamide 250 mg/m² on Days 1-5.
If prior treatment with the VAC/VTC regimen is ineffective because of resistance or is not tolerated for safety reasons, the investigator team will discuss and develop an individualized combination chemotherapy regimen
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1- entinistat monotherapy | Experimental | After completion of prior chemotherapy and/or radiotherapy, patients who have achieved CR/PR will receive entinostat as consolidation therapy. Receives: Drug: Entinostat |
| Cohort 2 - entinostat combined with chemotherapy | Experimental | Patients with recurrent or progressive disease will receive entinostat in combination with alternating VAC/VTC chemotherapy. Receives: Drug: Entinostat; Drug: VAC/VTC chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: incidence of adverse events (AEs) and Grade ≥3 AEs according to NCI-CTCAE v5.0.
AEs will be coded using MedDRA and graded according to NCI-CTCAE v5.0.
Measured: AE recorded from infromed consent to 28 days after treatment completion or initiation of another anticancer treatment, whichever occurs first.
Secondary outcomes (4)
Progress-free survival (PFS)
The time from the date of first study-drug administration to radiographic disease progression or death from any cause, whichever occurs first.
Measured: 2 years
Objective response rate (ORR)
the proportion of participants achieving CR or PR according to RECIST v1.1
Measured: 2 years
Disease control rate (DCR)
The proportion of participants achieving CR, PR, or SD according to RECIST v1.1.
Measured: 2 years
Overall survival (OS)
The time from the first administration of study drug to death from any cause.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2026
- Study start15 Mar 2025
- Primary completion31 Mar 2027 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Soft Tissue SarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Tongren Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07888114
- Sponsor's protocol ID
- TRIITKX-2025-029
- Status last verified
- Oct 2026
- First submitted
- 15 Oct 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided