Clinical trial NCT05406947: Establishment and Clinical Application of Risk Classification Model Based on Molecular Typing of Medulloblastoma in Children
Official title: Establishment and Clinical Application of Risk Classification Model Based on Molecular Typing of Medulloblastoma in Children
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Medulloblastoma, Childhood
Keywords: Medulloblastoma
Glioma
Medulloblastoma, Childhood, Recurrent
Neuroepithelioma
Lead sponsor: Beijing Tiantan Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Neurosurgical Institute
name: Beijing Children's Hospital
name: Beijing Shijitan Hospital, Capital Medical University
name: Xuanwu Hospital, Beijing
name: Peking University Third Hospital
First posted: 2022-06-07
Enrollment: 350
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 350 medulloblastoma patients aged 0.5-18 years (except newborns)

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Molecular typing
timeFrame: 2022-2030
measure: Overall survival
timeFrame: 2022-2030
measure: Progression free survival
timeFrame: 2022-2030
measure: Quality of life scale
timeFrame: 2022-2030
measure: Adjunctive treatment
timeFrame: 2022-2030
Secondary outcomes:
measure: Age at diagnosis
timeFrame: 2022-2030
measure: BMI
timeFrame: 2022-2030
Other outcomes:
Locations:
Study officials:
affiliation: Beijing TianTanHospital, China Capital Medical University
name: Yongji Tian, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Beijing Neurosurgical Institute
name: FU ZHAO, MD
role: PRINCIPAL_INVESTIGATOR
References: