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

Phase 1

Withdrawn

• Conditions: Acute Myeloid Leukemia; Refractory Pediatric AML; Relapsed Pediatric AML; AML, Childhood
• Interventions: Drug: ELU001

• Registration Number: NCT05622591
• Lead Sponsor: Elucida Oncology

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study First Submitted: 2022-11-08
• Study First Submitted QC: 2022-11-14
• Study First Posted: 2022-11-18
• Study Start: 2024-06-07
• Primary Completion: 2024-06-07
• Study Completion: 2024-06-07
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-05
• Last Update Posted: 2024-08-07

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Patients must meet the following criteria to enroll in this study:

• Infants (>1 month) and children (≤9 years) at time of enrollment.
• Relapsed or refractory CBFA2T3::GLIS2 positive AML
• CNS1 or CNS2 during screening
• Performance Status: Lansky ≥ 50
• Adequate Organ Function including liver, kidney, and heart

Key

Exclusion Criteria

Patients who meet any of the following are not eligible to enroll in this study:

• CNS3 Disease
• AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome or Diamond-Blackfan anemia, or bone marrow failure associated with inherited syndromes.
• Acute promyelocytic leukemia.
• Clinically significant active or chronic corneal disorder, particularly corneal epitheliopathy or any eye disorder that may predispose patient to this condition, or unable to comply with an age-appropriate ophthalmologic examination.
• Prior treatment with folate receptor-targeting anti-cancer agent(s) ≤ 21 days (or 2 half-lives must have elapsed before enrollment, whichever is longer), or received investigational anti-cancer treatment ≤ 4 weeks, or within a time interval less than at least 5 half-lives of the investigational agent, prior to starting study drug, whichever is shorter.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ELU001	ELU001	Dose Escalation: Escalating doses of ELU001

Primary Outcome Measures

Name	Time	Method
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001	28 days	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.

Secondary Outcome Measures

Name	Time	Method
Evaluate preliminary anti-leukemic activity of ELU001	First dose of study drug until 42 days after last cycle.	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
Characterize the pharmacokinetics of ELU001	First dose of study drug until 42 days after last cycle.	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
Characterize the immunogenicity of ELU001	First dose of study drug until 42 days after last cycle.	Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).