Phase II study of multimodality treatment for ATRT

JRCT ID: jRCTs051200071

Registered date:22/10/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Atypical teratoid rhabdoid tumor
• Date of first enrollment: 27/10/2020
• Target sample size: 50
• Study type: Interventional

Intervention(s)

Multimodality treatment will be provided for each risk group as a recommended regimen.

Standard risk group (M0-1): 6 courses of multi-agent chemotherapy including intrathecal chemotherapy followed by high-dose chemotherapy and local radiation.
High-risk group (M2-3): 6 courses of multi-agent chemotherapy including intrathecal chemotherapy followed by high-dose chemotherapy and local radiation with additional treatment intensification (maintenance chemotherapy via intrathecal injection for patients under 3 years, while craniospinal irradiation for patients over 4 years).

Outcome(s)

• Primary Outcome: 3-year progression free survival (3yr-PFS)

Secondary Outcome

Efficacy evaluation
1. 3-year and 5year overall survival
2. 5-year progression-free survival
3. Response rate
4. Tumor location at the onset and relapse
5. The proportion of 2nd look surgery
6. Site of recurrence
7. Response rate with topotecan window (M2-3 only)
8. Recurrence/progression rate during treatment

Safety evaluation
1. Rate of the adverse event as CTCAE Grade 3 or higher.
2. Periods from surgery to the initiation of protocol treatment
3. Rate of completion of protocol treatment and rate of leukoencephalopathy
4. Rate of late complication

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: < 21age old
• Gender: Both

Include criteria

1) Locally diagnosed as atypical teratoid rhabdoid tumor (ATRT) after tumor resection or biopsy.
2) Registered in an observational study for pediatric solid tumors by Japan Children's Oncology Group (JCCG).
3) A sample for a central diagnosis has been submitted or is scheduled to be submitted.
4) Protocol treatment can be started within 6 weeks after surgery
5) Less than 21 years
6) No prior radiation therapy or chemotherapy
7) ECOG Performance Status (PS) is 3 or less
8) The following conditions are met within 14 days prior to registration
1. WBC>=2000/maicroL
2. PLT>=100,000/maicroL
3. AST<=100IU/L
4. D-Bil<=1.0mg/dl
5. Serum creatinine level is below the upper limit of normal value
9) Written informed consent is obtained from the patient or his/her legal guardian.

Exclude criteria

1) Active double cancer (synchronous double cancer and metachronous double cancer with disease-free period of 5 years or less).
2) Presence of extracranial metastasis (M4) or extracranial rhabdoid tumor.
3) Complicated heart disease requiring treatment.
4) You are pregnant or nursing.
5) Ddeemed inappropriate at the discretion of the attending physician.

Related Information

• Primary Sponsor: Junichi Hara
• Secondary Sponsor: Japan Ag ency for Medical Research and Development
• Secondary ID(s): Nil known