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Micronutrients

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Folic Acid

Vitamin B Complex

Exatecan

Folinic Acid

Folate

Vitamin B9

Dose Escalation: Escalating doses of ELU001

Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)

ELU001

This research study was planned to focus on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Refractory means the cancer does not respond to treatment.

This study was planned as a Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D). This study will also evaluate the tolerability of ELU001.

ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet.

ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML

Dose Escalation Study to Evaluate the Safety and Tolerability of ELU001 in Pediatric Patients Who Have Relapsed and/or Refractory CBFA2T3::GLIS2 Positive Acute Myeloid Leukemia

Establish the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 in pediatric patients with relapsed or refractory CBFA2T3::GLIS2 positive AML.

Proportion of evaluable patients having achieved at least one of the following

* Complete Remission per IWG (CRIWG)
* Complete Remission With Partial Recovery of Platelet Count (CRp)
* Complete Remission with Incomplete Blood Count Recovery (CRi)
* Complete Remission for Minimal Residual Disease (CRm)

Duration of Complete Remission from CRIWG/CR/CRp/CRi to hematological relapse or death from any cause, whichever comes first

Measure the concentration of ELU001 in the blood.

This includes - Maximum Observed Concentration (Cmax), Time After Dosing at which Maximum Observed Concentration of Drug is Observed (tmax), Area Under the Curve to the End of the Dosing Period (AUC0-tau), and Area Under the Curve to the Last Measurable Concentration (AUC0-t), will be estimated. Other PK parameters, e.g., Terminal Elimination or Disposition Half-Life (T½), Volume of Distribution (Vd), Clearance Rate (CL), and C'Dot, payload on C'Dot

Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).

Elucida Oncology

Anti-Infective Agents

Amino Acids

Trastuzumab

Carboplatin

Etoposide

Cytarabine

Fludarabine

Fludarabine phosphate

Everolimus

Axitinib

Ifosfamide

Etoposide phosphate

Dasatinib

Crizotinib

Tyrosine Kinase Inhibitors

Sorafenib

Isophosphamide mustard

Imatinib Mesylate

Erlotinib Hydrochloride

Vemurafenib

Antineoplastic Agents, Immunological

Antimetabolites

Immunosuppressive Agents

Immunologic Factors

Enzyme Inhibitors

Antiviral Agents

Sirolimus

Temsirolimus

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Protein Kinase Inhibitors

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Alkylating Agents

Tyrosine

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™

CancerSCAN™

Pazopanib

Erlotinib

Imatinib

Ruxolitinib

Vandetanib

Ki Woong Sung, MD, PhD

Ki Woong Sung

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.

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™

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

Event is defined as relapse, disease progression or treatment-related mortality.

Ministry of Health, Republic of Korea

Samsung Medical Center

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML	NCT05622591	Acute Myeloid L... AML, Childhood Relapsed Pediat... Refractory Pedi...	ELU001	1 Month - 9 Years	Elucida Oncology
Genomics-Based Target Therapy for Children With Relapsed or Refractory Malignancy	NCT02638428	Relapsed Pediat... Refractory Pedi... Relapsed Pediat... Refractory Pedi...	CancerSCAN™ Ifosfamide Carboplatin Etoposide Fludarabine Cytarabine Pazopanib Sorafenib Axitinib Crizotinib Dasatinib Erlotinib Everolimus Imatinib Ruxolitinib Vandetanib Vemurafenib Trastuzumab	- 18 Years	Samsung Medical Center

Spots Global Cancer Trial Database for refractory pediatric aml