Establishment and Clinical Application of Risk Classification Model Based on Molecular Typing of Medulloblastoma in Children

The purpose of this study:(1) Development of a new risk classification model for childhood medulloblastoma. (2) Evaluation and improvement of existing individualized treatment protocols.

Study Overview

• Status: Not yet recruiting
• Conditions: Medulloblastoma, Childhood
• Intervention / Treatment: Other: Prospective observational study, no intervention

Detailed Description

Medulloblastoma (medulloblastoma, MB) is the most common intracranial malignant tumor in children, accounting for 20% of all central nervous system tumors in children, seriously affecting the quality of life and life span of children. Based on a retrospective analysis of previous MB cases in our center, we found that the clinical prognosis of previous MB patients in our center was worse than that in foreign countries, with an overall five-year survival rate of about 65%, and nearly 30% of the patients had tumor recurrence and metastasis within 2 years after the operation, and the prognosis was poor. We analyzed the possible reasons as follows: (1) the compliance to radiotherapy and chemotherapy in children with MB in our center was poor, and some of the patients only completed radiotherapy and had poor compliance with chemotherapy; (2) due to the lack of family doctor system, the tumor of the newly diagnosed patients was huge, which seriously affected the important brain function and clinical prognosis; (3) the patients were not followed up strictly and regularly after operation to monitor tumor recurrence, which led to poor treatment effect after recurrence.

(4) the unified treatment standard has not been formed yet, and the treatment mode for patients is complex.

As the largest neurosurgery and pediatric neurosurgery center in China, the center intends to prospectively establish a high-quality homogeneous MB observation cohort in children, make use of the center's case resources and biological sample processing advantages, and carry out accurate treatment research on children's MB through regular follow-up and systematic management of the clinical cohort.

• Study Type: Observational
• Enrollment (Anticipated): 350

Contacts and Locations

• Study Contact: Name: yongji TIAN, MD; Phone Number: 15801593549; Email: tianyongji@bjtth.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

350 medulloblastoma patients aged 0.5-18 years (except newborns)

Description

Inclusion Criteria:

• Age 0.5-18 years (except neonates).
• Pathologically confirmed medulloblastoma.
• Not having received any other relevant treatment before surgery.
• Completion of enhanced MRI of the head and spinal cord.
• Availability of tumor samples and determination of molecular typing.
• Postoperative KPS score ≥ 70.
• Voluntary enrollment in the group and the ability to receive long-term follow-up.
• The patient or the patient's family voluntarily signed the informed consent form.

Exclusion Criteria:

• Patients who have recently received other drugs or radiation therapy.
• Patients suffering from acute or chronic infectious diseases
• Patients suffering from neurological or psychiatric diseases or mental disorders that cannot be easily controlled, or poor compliance.
• Patients who cannot receive enhanced MRI scans.
• Other conditions that the investigator believes make the patient unfit to participate in this trial.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Low risk group; Based on clinicopathological diagnosis and molecular pathological diagnosis, the children were classified into low risk groups, individualized radiotherapy and chemotherapy plan was made, and a perfect registration and follow-up system was established.	Other: Prospective observational study, no intervention; Prospective observational study, no intervention
Middle risk group; Based on clinicopathological diagnosis and molecular pathological diagnosis, the children were classified into middle risk groups, individualized radiotherapy and chemotherapy plan was made, and a perfect registration and follow-up system was established.	Other: Prospective observational study, no intervention; Prospective observational study, no intervention
High risk group; Based on clinicopathological diagnosis and molecular pathological diagnosis, the children were classified into high risk groups, individualized radiotherapy and chemotherapy plan was made, and a perfect registration and follow-up system was established.	Other: Prospective observational study, no intervention; Prospective observational study, no intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Molecular typing	Medulloblastoma can be classified into four molecular types: WNT, SHH, G3 and G4.	2022-2030
Overall survival	the time from operation to death	2022-2030
Progression free survival	The time from operation to disease progression	2022-2030
Quality of life scale	A scale for evaluating the quality of life of patients after operation	2022-2030
Adjunctive treatment	Patients receive postoperative radiotherapy or chemotherapy	2022-2030

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Age at diagnosis		2022-2030
BMI	weight and height will be combined to report BMI in kg/m^2	2022-2030

Collaborators and Investigators

• Sponsor: Beijing Tiantan Hospital
• Collaborators: Peking University Third Hospital; Xuanwu Hospital, Beijing; Beijing Shijitan Hospital, Capital Medical University; Beijing Children's Hospital; Beijing Neurosurgical Institute
• Investigators: Principal Investigator: Yongji Tian, MD, Beijing TianTanHospital, China Capital Medical University; Principal Investigator: FU ZHAO, MD, Beijing Neurosurgical Institute

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): January 30, 2023
• Primary Completion (ANTICIPATED): December 31, 2028
• Study Completion (ANTICIPATED): December 31, 2030

Study Registration Dates

• First Submitted: June 1, 2022
• First Submitted That Met QC Criteria: June 4, 2022
• First Posted (ACTUAL): June 7, 2022

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 6, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: Glioma; Medulloblastoma; Medulloblastoma, Childhood, Recurrent; Neuroepithelioma
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Medulloblastoma
• Other Study ID Numbers: ChiCTR2200058760

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No