Genomics-Based Target Therapy for Childhood Malignancy
- Conditions
- Neoplasms
- Registration Number
- KCT0002007
- Lead Sponsor
- Samsung Medical Center
- Brief Summary
? Validity evaluation Evaluation of whether event free survival is improved compared to history control when using a combination of targeted anticancer drugs 2 Evaluation of Tumor Response Rate when Targeted Anticancer Drugs and Existing Therapy are Combined ? Safety evaluation Evaluate using the Common Terminology Criteria (version 4.03) of the U.S. National Cancer Institute Therapeutic response - Targeted anticancer drugs were used for secondary anticancer abnormalities, and responses were evaluated for patients with evaluable diseases. - CR 4, PR 14, SD 28, PD 14 out of 60 Diagnostic distribution Neuroblastoma: 25 AML:13 Brain tumor: 11 NRSRS:9 RMS:9 OSA:5 HBL:3 Others: 8 Actionable target, tier classification, medication Tier 1a: Clinical evidence in the same disease (2 patients) Tier 1b: Clinical evidence in different diseases (4 patients) Tier 2a: Preclinical evidence in the same disease (5 patients) Tier 2b: Preclinical evidence in different diseases (6 patients) Tier 3: Consensus (3 people) Tier 4: Available in other countries as clinical rials or FDA-approved drugs for other diseases (5 people) Tier 5: No target variant (50 people) Treatment-related toxicity - A total of 21 patients out of 84 subjects experienced cessation of targeted anticancer drugs due to toxicity. - Six of them were eliminated for reasons other than toxicity, and three were eliminated from the study because toxicity did not improve. In the remaining 12 patients, toxicity improved and the administration of targeted anticancer drugs was resumed, and the study was completed without any problems.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 84
• Under 18 years of age at initial diagnosis
• Patients with refractory/relapsed solid tumor or acute myeloid leukemia (Solid tumor: Stable or progressive disease after 1st-line treatment or relapse; acute myeloid leukemia: Persistence after 2 cycles of induction chemotherapy or relapse)
• Patient with tumor sample which is adequate for targeted deep sequencing
• Patients who had salvage chemotherapy previously (solid tumor)
• 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
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Rate of event free survival
- Secondary Outcome Measures
Name Time Method Rate of treatment-related adverse events