Pediatric and AYA solid tumor patients aged 0-29 years with no standard treatment or refractory/into Solid Tumors in Children and AYAs
Conditions
Interventions
Children/AYAs, multigene panel test, molecular target therapy, basket trial, patient proposed health system.
Sponsors
Arakawa Ayumu
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Tumors (excluding I-II) included in the International Classification of Childhood Cancer (ICCC-3, International Classification of Childhood Cancer, third edition) have been diagnosed byhistopathological examination. If a histopathological examination has been performed at another institution, the pathology specimen must be ordered, and the criteria must be confirmed to be met by the pathology diagnosis at the implementing institution. 2) Patients have advanced (metastatic or locally advanced) disease that is not amenable to curative resection (including cases in which resection is difficult except for amputation of a limb or other major functional loss) and meet either of the following criteria (regardless of the number of prior treatment regimens) (1) No standard treatment (or treatment equivalent to standard treatment) exists. (2) If standard treatment (or treatment equivalentto standard treatment) exists, the standard treatment (or treatment equivalent to standardtreatment) has been ineffective or discontinued due to toxicity. However, in cohorts using pharmaceutical agents approved for maintenance therapy after prior treatment in Japan or overseas (by the FDA or EMA), the presence of evaluable disease at the time of registration is not required. 3) Age at enrollment is 0-29 years (but does not meet the age exclusion criteria for each drug cohort). 4) Treatment is not approved in Japan for the patient's age or cancer type in the disease (i.e the treatment is off-label or unapproved for the patient). 5) One of the following conditions is met (i) Neoplastic lesions can be confirmed by contrast-enhanced CT or MRI (head, chest, abdomen, pelvis: slice thickness of 5 mm or less) within 28 daysprior to enrollment (regardless of whether measurable lesions are present). (ii) Bone marrow examination (bone marrow puncture or bone marrow biopsy) within 28 days prior to registration shows tumor cells. *Up to 4 weeks prior to the same day of the week *In the case of maintenance therapy as defined in Eligibility Criteria 2), it is not required to meet Eligibility Criteria 5). 6) One of the following is met (1) The patient has undergone a gene panel test that is covered by insurance or provided as an evaluation treatment in Japan, and has been found to have an actionable genetic abnormality. (2) The patient has an expert panel report that presents actionable genetic abnormalities judged to have an evidence level of D or higher and treatment options based on the abnormalities, as well as the gene panel report that served as the basis for the expert panel report. (ii) Pathologically diagnosed as a type of cancer indicated for a molecular-targeted drugapproved by the regulatory authorities in Japan or overseas (FDA or EMA).Specifically, the following Drugs that have been approved in Japan for use in adults but not for use in children (no pediatric dosage and administration information) for cancer types for which the molecular target drug has been shown to be effective.Drugs that have been approved overseas (FDA or EMA) for pediatric use but not approved in Japan for pediatric use in cancers for which molecular target drugs have been shown to be effective 7) A record in the medical record of the selection of the therapeutic drug (drug submitted by the patient) based on consultation between the principal investigator or subinvestigator and the patient. 8) The subject is not a subject of a corporate clinical trial, investigator-initiated clinical tria
Exclusion criteria
Exclusion criteria: 1) Patients with psychiatric disorders or psychiatric symptoms that interfere with daily life and make it difficult for them to participate in the study 2) Has an active infectious disease requiring systemic treatment 3) Has an active gastrointestinal ulcer 4) Have active multiple cancers (however, completely resected basal cell, spinous cell, intraepithelial, intramucosal, superficial bladder, endoscopically resected gastrointestinal cancers, or other cancers that have not recurred for at least 5 years may be enrolled) 5) Complicated or pre-existing interstitial lung disease or pulmonary fibrosis diagnosed by imaging or clinical findings (6) Interstitial shadows on imaging studies or inflammatory changes in the lungs, such as active radiation pneumonitis or infectious pneumonia. 7) Positive HIV antibody, HTLV-1 antibody, HBs antigen, or HCV antibody (patients with positive HCV antibody but no detectable HCV-RNA are not excluded). All of these should be confirmed by blood test. 8) HBs antigen negative, positive for HBs antibody or HBc antibody, and positive for HBV-DNA quantification (registration is acceptable if the HBV-DNA quantification is below the detection sensitivity). However, HBV-DNA quantification is not required if a history of hepatitis B vaccination can be confirmed in writing. 9) Pregnant, lactating (even if lactation has been interrupted, registration is not possible), or women who may be pregnant (10) QT corrected for HR using Fridericia's method (QTcF) interval > 470 ms on a 12-lead ECG performed within 14 days prior to enrollment (11) Cardiac complications such as heart failure or arrhythmia syndrome that would make the principal investigator or study investigator consider the patient ineligible 12) Allogeneic hematopoietic stem cell transplantation within 90 days prior to enrollment 13) Ineligible for other reasons as deemed by the principal investigator or subinvestigator (including cases in which the investigator or subinvestigator determines that the investigator or subinvestigator is ineligible for the individual characteristics of each drug) 14) Meets the exclusion criteria in the Protocol Appendix for each drug.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of adverse events equivalent to dose-limiting toxicities in each drug cohort | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Safety endpoints: adverse event rate 2) Efficacy endpoints: response rate, disease control rate, progression-free survival, and overall survival 3) Pharmacokinetic parameters (to be defined in a drug-specific appendix for each drug cohort) | — |
Contacts
Public ContactMiho Nakajima
National Cancer Center Hospital
Outcome results
None listed