A feasibility study of patient-proposed healthcare services with oral entrectinib in patients with ROS1-fusion-positive pediatric brain tumors - ROSE study

Kurimoto Michihiro0 sites1 target enrollmentJuly 10, 2020

ConditionsPediatric brain tumor

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Pediatric brain tumor
Sponsor: Kurimoto Michihiro
Enrollment: 1
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

In this case, profiling by gene panel testing revealed a mutation in the driver gene, which led to patient-directed therapy for molecular targeted therapy. Although this therapy did not reduce the size of the target lesion, SD was achieved, which is an improvement over the clinical course expected from the original tumor grade. The safety profile was also acceptable, with no unexpected adverse events, no hematologic toxicity during the treatment period, and only grade 2 constipation.

Registry

who.int

Start Date

July 10, 2020

End Date

August 4, 2021

Last Updated

2 years ago

Study Type

Interventional

Sex

All

Investigators

Sponsor

Kurimoto Michihiro

Eligibility Criteria

Inclusion Criteria

•1\.Primary brain tumors with ROS1 gene fusions that are locally advanced; gene fusions are defined as those predicted to translate into a fusion protein with a functional ROS1 kinase domain, without a concomitant second oncodriver (e.g. known, activating mutations in EGFR, KRAS) as determined by a nucleic acid\-based diagnostic testing method approved in Japan.
•2\.Patients who have no satisfactory treatment options for primary CNS tumors.
•3\.Performance status: PS (ECOG) 0\-2 and minimum life expectancy of at least 4 weeks.
•4\.Age: Male or female from birth to age \<\=15 years.
•5\.Patients must have measurable or evaluable disease, as defined in RANO. Measurable disease is defined as bidimensionally contrast enhancing lesions with clearly defined margins by CT or MRI scan, with two perpendicular diameters of at least 10 mm, visible on two or more axial slices that are preferably, at most, 5 mm apart with 0\-mm skip.
•6\.No prior treatment with approved or investigational ROS1 inhibitors.
•7\.Patients must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to enrollment.
•8\.XRT/External Beam Irradiation: \>\=14 days after whole brain XRT;\>\=7 days after stereotactic radiation surgery.
•9\.Organ function;
•i. ANC \>\= 1,000/microL;

Exclusion Criteria

•1\.Synchronous or metachronous (within 5 years) malignancies except for carcinoma in situ or mucosal tumors curatively treated with local therapy.
•2\. Active infection requiring systemic therapy.
•3\.Body temperature \>\= 38 degrees Celsius at registration.
•4\.Women during pregnancy, possible pregnancy or breast\-feeding.
•5\.Continuous systemic use of immunosuppressant including steroid.
•6\.Uncontrolled diabetes mellitus or routine administration of insulin.
•7\.Congestive heart failure within 3 months or ejection fraction \<\= 50%.
•8\.Inadequately controlled hypertension.
•9\.Known congenital long QT syndrome.
•10\.Familial history of long QT syndrome.