Genomics-Based Target Therapy for Children With Relapsed or Refractory Malignancy

Samsung Medical Center

Status and phase

Unknown

Phase 2

Conditions

Refractory Pediatric AML

Refractory Pediatric Solid Tumor

Relapsed Pediatric Solid Tumor

Relapsed Pediatric AML

Treatments

Drug: Ruxolitinib

Drug: Crizotinib

Drug: Pazopanib

Drug: Axitinib

Drug: Etoposide

Drug: Imatinib

Drug: Ifosfamide

Procedure: CancerSCAN™

Drug: Carboplatin

Drug: Fludarabine

Drug: Cytarabine

Drug: Vemurafenib

Drug: Dasatinib

Drug: Everolimus

Drug: Trastuzumab

Drug: Vandetanib

Drug: Sorafenib

Drug: Erlotinib

Study type

Interventional

Funder types

Other

Identifiers

NCT02638428

2015-08-008

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy and feasibility of combination chemotherapy with target agents according to the result of targeted deep sequencing in pediatric patients with relapsed/refractory solid tumor or AML.

Full description

Outcome of pediatric cancer has been improved substantially over the past few decades, but the prognosis of relapsed/refractory pediatric cancer still remains poor. Advances in genomic technologies have improved the ability to detect diverse somatic and germline genomic aberrations of cancer patients, and it has been incorporated in the clinical management of cancer.

Samsung Genomic Institute developed a targeted next-generation sequencing (NGS) platform, CancerSCAN™, which can detect clinically significant genomic aberrations of tumors. In this study, tumor samples of refractory/relapsed pediatric cancer patients will be tested with CancerSCAN™ and the patients will receive combination chemotherapy with matched single-targeted agent or multi-targeted receptor tyrosine kinase inhibitor according to the result of CancerSCAN™.

I. Relapsed/refractory solid tumor

Perform CancerSCAN™ at enrollment
Conventional chemotherapy (ifosfamide, carboplatin, etoposide) with matched single-targeted agent (axitinib, crizotinib, dasatinib, erlotinib, everolimus, imatinib, pazopanib, ruxolitinib, sorafenib, vandetanib, vemurafenib, or trastuzumab) or multi-targeted receptor tyrosine kinase inhibitor (pazopanib or sorafenib) according to the result of CancerSCAN™

II. Relapsed/refractory AML

Perform CancerSCAN™ at enrollment
Conventional chemotherapy (fludarabine, cytarabine) with matched single-targeted agent (axitinib, crizotinib, dasatinib, erlotinib, everolimus, imatinib, pazopanib, ruxolitinib, sorafenib, vandetanib, vemurafenib, or trastuzumab) or multi-targeted receptor tyrosine kinase inhibitor (pazopanib or sorafenib) according to the result of CancerSCAN™

Enrollment

90 estimated patients

Sex

All

Ages

Under 18 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Under 18 years of age at initial diagnosis
Patients with refractory/relapsed solid tumor or AML (Solid tumor: Stable or progressive disease after 1st-line treatment or relapse; AML: Persistence after 2 cycles of induction chemotherapy or relapse)
Patient with tumor sample which is adequate for targeted deep sequencing

Exclusion criteria

Patients who had salvage chemotherapy previously
Patients with organ dysfunction as follows (creatinine elevation ≥ 3 x upper limit of normal (ULN), ejection fraction <40%, significant arrhythmia or conduction disturbance)
Patients who are not eligible to have scheduled treatment due to the other significant impaired organ function
Patients whose tumor samples are not sufficient for targeted deep sequencing
Pregnant or nursing women

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

90 participants in 1 patient group

Refractory/relapsed solid tumor or AML

Experimental group

Description:

Conventional chemotherapy (Ifosfamide carboplatin etoposide for solid tumor and fludarabine cytarabine for AML) with matched single-targeted agent (axitinib, crizotinib, dasatinib, erlotinib, everolimus, imatinib, pazopanib, ruxolitinib, sorafenib, vandetanib, vemurafenib, or trastuzumab) or multi-targeted receptor tyrosine kinase inhibitor (pazopanib or sorafenib) according to the result of CancerSCAN™

Treatment: