Clinical trial NCT02638428: Genomics-Based Target Therapy for Children With Relapsed or Refractory Malignancy
Official title: Genomics-Based Target Therapy for Children With Relapsed or Refractory Malignancy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Relapsed Pediatric Solid Tumor
Refractory Pediatric Solid Tumor
Relapsed Pediatric AML
Refractory Pediatric AML
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
Collaborators: name: Ministry of Health, Republic of Korea
First posted: 2015-12-23
Enrollment: 90
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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™

Eligibility criteria: 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

Primary outcomes:
measure: Rate of event free survival
timeFrame: Up to 5 years
Secondary outcomes:
measure: Rate of treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: Up to 1 year
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
affiliation: Samsung Medical Center
name: Ki Woong Sung, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: