NCCH2220 trial

JRCT ID: jRCTs031230544

Registered date:18/01/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Pediatric and AYA solid tumor patients aged 0-29 years with no standard treatment or refractory/into
• Date of first enrollment: 18/01/2024
• Target sample size: 50
• Study type: Interventional
• Intervention(s): molecular target therapy

Outcome(s)

• Primary Outcome: Percentage of adverse events equivalent to dose-limiting toxicities in each drug cohort

Secondary Outcome

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)

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: <= 29age old
• Gender: Both

Include 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 by histopathological 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 equivalent to standard treatment) exists, the standard treatment (or treatment equivalent to standard treatment) has been ineffective or discontinued due to toxicity.
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 days prior 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.
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 trial, or advanced medical treatment being conducted at a medical institution in Japan. However, patients who do not meet the eligibility criteria or who have justifiable reasons for difficulty in participating in these clinical trials, such as geographical reasons, may participate in this study. Confirm the existence of clinical trials and advanced medical treatments using the drug in question in accordance with the C-CAT Clinical Trial Timeline provided to Core Hospitals for Cancer Genome Medicine, etc., and the reference procedures prescribed separately.
9) The patient agrees to all of the following
When a covered gene panel test is conducted, patient information must be registered with the Cancer Genome Information Center (C-CAT).
The data collected for this study should be provided to the pharmaceutical company that provided the drug in question.
10) Patients must not have cancer meningitis or symptomatic brain metastases.
11) No pericardial, pleural, or ascites effusions requiring periodic puncture
12) Performance status of 60 or higher on the KPS or LPS
13) No prior administration of the same drug
14) No prior discontinuation of the same drug due to adverse events or exacerbations.
15) No Grade 3 or higher complications in CTCAE v5.0-JCOG.
16) No anticancer drugs (chemotherapy, molecular targeted therapy, immunotherapy, endocrine therapy, etc.) within 28 days prior to enrollment (not including bone resorption modifying drugs such as bisphosphonate or denosumab for bone metastases)
17) Have not undergone surgery involving general anesthesia within 14 days prior to enrollment
18) Have not received radiotherapy or radiopharmaceuticals (excluding radiopharmaceuticals for diagnostic purposes) within 21 days prior to enrollment (palliative radiotherapy for bone lesions completed within 14 days prior to enrollment is acceptable, but the patient must have recovered prior to enrollment from any adverse events related to radiotherapy)
19) Laboratory tests performed within 7 days prior to enrollment meet all of the following However, the subject must not have received granulocyte colony stimulating factor (G-CSF) administration or blood transfusion within 7 days prior to blood collection. In the case of a continuous G-CSF (PEG) product, the patient must not have received such a product within 14 days prior to the date of blood collection.

20) For women of childbearing potential, consent to contraception for at least 5 months after obtaining consent and at least 5 months after the last dose of study treatment. For males of childbearing potential, the patient agrees to use contraception from the time of consent for at least 7 months after the last dose of study treatment.
21) Consent to participate in the study has been obtained in writing from the subject if the subject is 18 years of age or older; consent has been obtained in writing from a surrogate if the subject is 17 years of age or younger (and from the subject if possible); consent has been obtained in writing from the subject if the subject is 7 years of age or older, in principle, in an age-appropriate assent document.

Exclude 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.

Related Information

• Primary Sponsor: Ogawa Chitose
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development