Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children

Phase 1

• Conditions: Childhood Rhabdomyosarcoma
• Interventions: Drug: Apatinib mesylate tablets; Radiation: Three-dimensional conformal intensity modulation

• Registration Number: NCT03868852
• Lead Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of radiotherapy combined with apatinib mesylate in the treatment of rhabdomyosarcoma in children.

Detailed Description

This study is a Interventional study. The target group was 3-18 years old, and the pathological stage was group II-IV recurrence or distant metastasis. A total of 48 patients were enrolled in this study. The treatment was radiotherapy combined with apatinib. The main outcome measures were ORR, DCR , OS , quality of life and drug safety. The main safety indicators were vital signs, laboratory indicators, adverse events (AE), and severe adverse events (SAE). This study is expected to be enrolled for 6 months.

Study Timeline

• Study Start: 2019-01-01
• Status Verified: 2019-02
• Study First Submitted: 2019-02-11
• Study First Submitted QC: 2019-03-07
• Last Update Submitted: 2019-03-07
• Study First Posted: 2019-03-11
• Last Update Posted: 2019-03-11
• Primary Completion: 2020-04-01
• Study Completion: 2020-04-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

• Patients must be ≥ 3 and ≤ 18 years of age.

• The predicted survival period is more than 3 months.

• According to RECIST version 1.1, there is at least one measurable lesion.

• Postoperative pathological stage was group II-IV.

• Patients have not received radiotherapy before, and can accept chemotherapy and surgical treatment.

• No other anticancer therapy should be used during radiotherapy.

• The main organs are functioning normally, which meets the following criteria:

  1. Blood routine examination standards should be met: (no blood transfusion within 14 days) A.HB>90g/L; B.ANC>1.5*109/L; C.PLT>80*109/L
  2. Biochemical tests should meet the following criteria:

A. BIL < 1.25 times normal upper limit (ULN); B. ALT and AST < 2.5 ULN; C. Serum Cr < 1 ULN, endogenous creatinine clearance > 50 ml/min

• Subjects volunteered to participate in this study, patients or legal guardians signed informed consent through patient consent, good compliance, with follow-up.
• Doctors believe that treatment can benefit patients.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Radiotherapy plus apatinib mesylate	Three-dimensional conformal intensity modulation	All eligible patients signed informed consent. Three-dimensional conformal intensity modulation (IMRT) technique was used to treat the patients with radiation doses 45 Gy - 54 Gy. All patients took apatinib mesylate tablets 250 mg QD orally from 1 week before radiotherapy to the whole radiotherapy period. They were required to take apatinib mesylate tablets with warm boiling water half an hour after meal. The daily medication time should be as consistent as possible.
Radiotherapy plus apatinib mesylate	Apatinib mesylate tablets	All eligible patients signed informed consent. Three-dimensional conformal intensity modulation (IMRT) technique was used to treat the patients with radiation doses 45 Gy - 54 Gy. All patients took apatinib mesylate tablets 250 mg QD orally from 1 week before radiotherapy to the whole radiotherapy period. They were required to take apatinib mesylate tablets with warm boiling water half an hour after meal. The daily medication time should be as consistent as possible.

Primary Outcome Measures

Name	Time	Method
Objective response rate (ORR)	First analysis will occur 1 month after accrual of all patients.	Refers to the proportion of patients whose tumors have shrunk to a certain extent and maintained for a certain period of time, including CR and PR cases. Objective remission of solid tumors was assessed by RECIST 1.1. Subjects must be accompanied by measurable tumor lesions at baseline. According to RECIST 1.1, the criteria for evaluating efficacy are complete remission (CR), partial remission (PR), stability (SD), and progression (PD).

Secondary Outcome Measures

Name	Time	Method
Incidence and severity of Adverse Events	From date of enrollment until 30 days after the last medication.	The safety of the drug was evaluated by NCI-CTCAE 4.0. Common Terminology Criteria Adverse Events Version 4.0 prepared by National Cancer Institute.
Quality of Life Score	Weekly assessment , assessed up to 1 month.	Quality of life score refer to EORTC qlq-c30 (version 3, Chinese version). Quality of Life Questionnare-Core 30 prepared by The European O-rganization for Reasearch and Treatment of Cancer.; EORTC qlq-c30 (V3.0) is the core scale for all cancer patients, with a total of 30 items. Items 29 and 30 are rated on a scale of 1 to 7 according to their answer choices. The other items are divided into four levels: totally not, a little bit, a lot and very much, and are scored on a scale of 1 to 4.; The higher the score of functional domain and general health domain, the better the functional status and quality of life. The higher the score of symptom domain, the more symptoms or problems (the worse the quality of life).
Disease Control Rate (DCR)	First analysis will occur 1 month after accrual of all patients.	The percentage of patients whose tumors shrink or remain stable for a certain period of time
Total Survival (OS)	From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months.	The time from enrollment to death for any reason. For those who had lost interviews before death, the last follow-up time was usually calculated as the time of death.

Trial Locations

Locations (1)

The Department of Radiation Oncology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine; 🇨🇳 Shanghai, Shanghai, China