Clinical trial NCT03868852: Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children
Official title: Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Childhood Rhabdomyosarcoma
Keywords: Radiation Therapy
Rhabdomyosarcoma in Children
Lead sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
First posted: 2019-03-11
Enrollment: 48
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Intervention model: SEQUENTIAL
Intervention model description: Simons two-stage design method requires the enrollment of 13 patients in the first stage. If more than 7 patients fail to reach the expected end point, the first stage fails and the patients do not need to enter the second stage. A total of 48 patients were enrolled. If more than 30 patients did not reach the expected end point, the overall trial failed.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Objective response rate (ORR)
timeFrame: First analysis will occur 1 month after accrual of all patients.
Secondary outcomes:
measure: Disease Control Rate (DCR)
timeFrame: First analysis will occur 1 month after accrual of all patients.
measure: Total Survival (OS)
timeFrame: 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.
measure: Quality of Life Score
timeFrame: Weekly assessment , assessed up to 1 month.
measure: Incidence and severity of Adverse Events
timeFrame: From date of enrollment until 30 days after the last medication.
Other outcomes:
Locations:
city: Shanghai
country: China
facility: The Department of Radiation Oncology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
state: Shanghai
Study officials:
affiliation: Xin Hua Hospital affiliated to Shanghai Jiaotong University School of Medicine
name: Mawei Jiang, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Vatner R, James CD, Sathiaseelan V, Bondra KM, Kalapurakal JA, Houghton PJ. Radiation therapy and molecular-targeted agents in preclinical testing for immunotherapy, brain tumors, and sarcomas: Opportunities and challenges. Pediatr Blood Cancer. 2021 May;68 Suppl 2:e28439. doi: 10.1002/pbc.28439. Epub 2020 Aug 22.