A Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma

NCT07355855 | Not Yet Recruiting Phase 2

• 1 other identifier: RMS-ATRA-IIT-01
• Study Type: interventional
• Target: 106
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a prospective, multi-cohort, multi-center clinical trial targeting patients with intermediate-to-high-risk rhabdomyosarcoma who have not previously received systemic anti-tumor treatment. It aims to evaluate the efficacy and safety of all-trans retinoic acid combined with VAC chemotherapy.

Conditions

Rhabdomyosarcoma

Outcome Measures

Primary Outcomes (1)

• 2-year event-free survival (EFS) rate

  up to 2 years

Secondary Outcomes (6)

• Overall survival (OS)

  up to 2 years

• Disease control rate (DCR)

  up to 2 years

• Objective response rate (ORR)

  up to 2 years

• adverse event

  up to 2 years

• Quality of life (Functional Assessment of Cancer Therapy - General)

  up to 2 years

Study Arms (2)

ATRA+VAC

EXPERIMENTAL

Drug: ATRA+VAC

ATRA+VAC+Anlotinib

EXPERIMENTAL

Drug: ATRA+VAC+Anlotinib

Interventions

ATRA+VAC+Anlotinib DRUG

Cohort 2: High risk group RMS+: ATRA+VAC, ATRA+anlotinib maintenance therapy The specific medication is: Vincristine (V), static push, 1.5 mg/m2 (maximum not exceeding 2mg), D1, 8, 15, q3w. Dactinomycin (A), intravenous drip, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. After chemotherapy, Anlotinib, 12mg, d1-d14, po, q3w+ATRA maintenance therapy. Both are maintained for a period of time until disease progression or toxicity is intolerable, or for at least 2 years. All trans retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd., capsule, 10mg/capsule, 20mg bid orally, administered continuously daily for 21 days as a course of treatment. If safety is ensured, the dosage will be increased to 30mg bid.

ATRA+VAC+Anlotinib

ATRA+VAC DRUG

Cohort 1: Intermediate-risk RMS: ATRA+VAC treatment The specific medication is: Vincristine (V), administered intravenously, at a dose of 1.5 mg/m2 (with a maximum of 2 mg), on days 1, 8, and 15, every three weeks. Dactinomycin (A), intravenous infusion, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. All-trans-retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd. Capsules, 10mg/capsule, 20mg bid, orally, administered continuously every day, with 21 days constituting one course of treatment. If safety is confirmed, the dosage can be increased to 30mg bid.

ATRA+VAC

Eligibility Criteria

• Age: 14 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age ≥ 14 years old and ≤ 60 years old;
• Histologically confirmed medium to high risk rhabdomyosarcoma (excluding pleomorphic rhabdomyosarcoma);
• The physical fitness status score of the Eastern Cancer Collaboration Group (ECOG) is 0-1;
• Have not received any anti-tumor drug treatment in the past;
• Expected survival time ≥ 3 months;
• Possess sufficient organ and bone marrow function, with laboratory test values meeting the following requirements within 7 days prior to enrollment (no blood components, cell growth factors, albumin, or other corrective treatment drugs are allowed within 14 days prior to obtaining laboratory tests), as follows Blood routine: Absolute neutrophil count (ANC) ≥ 1.5 × 109/L, platelet count (PLT) ≥ 100 × 109/L, hemoglobin (HGB) ≥ 100 g/L (no transfusion or erythropoietin dependence within 14 days) Liver function: serum total bilirubin ≤ 1.25 times the upper limit of normal (ULN); ALT and AST ≤ 2.5 x ULN (≤ 5x ULN for patients with liver metastases); Serum albumin ≥ 30 g/L; Alkaline phosphatase (ALP) ≤ 5 × ULN.
• Renal function: Serum creatinine (Cr) ≤ 1.25 × ULN, or creatinine clearance rate ≥ 60 mL/min (using the standard Cockcroft Gault formula): Urine routine results show urinary protein\<2+; For patients whose baseline urine routine test shows urinary protein ≥ 2+, 24-hour urine collection should be performed with a 24-hour urine protein quantification of\<1g.
• Coagulation function: International normalized ratio (INR) or prothrombin time (PT) ≤ 1.5 times ULN; If the subject is receiving anticoagulant therapy, as long as the INR is within the intended range of use of the anticoagulant drug.
• For female subjects of childbearing age, a urine or serum pregnancy test should be conducted 3 days before receiving the first study drug and the result should be negative;
• Participants and their sexual partners are required to use a medically approved contraceptive measure (such as intrauterine devices, birth control pills, or condoms) during the study treatment period and within 6 months after the end of the study treatment period.

You may not qualify if:

• Previously received anti-tumor treatment other than surgery and radiation therapy for any malignant tumor;
• Subjects who cannot accept or tolerate this chemotherapy regimen for various reasons;
• Biopsy confirmed a patient with bone marrow infiltration;
• Patients who have undergone major surgical procedures unrelated to medium to high risk rhabdomyosarcoma within the 4 weeks prior to enrollment, or who have not fully recovered from such surgical procedures;
• Serious heart disease or discomfort, including but not limited to the following diseases:
• Diagnosed history of heart failure or systolic dysfunction (LVEF\<50%);
• High risk uncontrolled arrhythmias, such as atrial tachycardia, resting heart rate\>100bpm, significant ventricular arrhythmias (such as ventricular tachycardia), or higher-level atrioventricular block (i.e. Mobitz II second or third degree atrioventricular block);
• Angina requiring treatment with anti angina drugs;
• Clinically significant heart valve disease;
• ECG shows transmural myocardial infarction;
• Poor control of hypertension (systolic blood pressure\>180mmHg and/or diastolic blood pressure\>100mmHg)
• Individuals with a known history of allergies to the components of this medication regimen;
• The researcher believes that the patient is not suitable to participate in any other circumstances of this study.

Sponsors & Collaborators

• Fudan University: lead

Study Sites (1)

Fudan university cancer hospital

Shanghai, China

Location

MeSH Terms

Conditions

Rhabdomyosarcoma

Condition Hierarchy (Ancestors)

Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma

Central Study Contacts

xin Liu

CONTACT

021-64175590-88503; jeanettexin@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician

Study Record Dates

• First Submitted: December 30, 2025
• First Posted: January 21, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2029
• Last Updated: January 21, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)