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

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.

Study Overview

• Status: Withdrawn
• Conditions: Acute Myeloid Leukemia; AML, Childhood; Relapsed Pediatric AML; Refractory Pediatric AML
• Intervention / Treatment: Drug: ELU001

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 Type: Interventional
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 9 years (Child)
• Accepts Healthy Volunteers: No

Description

Key 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ELU001; Dose Escalation: Escalating doses of ELU001	Drug: ELU001; Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001	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.	28 days

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Elucida Oncology
• Collaborators: Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL)

Publications and helpful links

Helpful Links

• Elucida Oncology Corporate Website

Study record dates

Study Major Dates

• Study Start (Actual): June 7, 2024
• Primary Completion (Actual): June 7, 2024
• Study Completion (Actual): June 7, 2024

Study Registration Dates

• First Submitted: November 8, 2022
• First Submitted That Met QC Criteria: November 14, 2022
• First Posted (Actual): November 18, 2022

Study Record Updates

• Last Update Posted (Actual): August 7, 2024
• Last Update Submitted That Met QC Criteria: August 5, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Relapsed; Refractory; AML; Childhood; Folate Receptor alpha; ELU001; Exatecan; C'Dot drug conjugate; C-Dot; C'Dot; CBFA2T3-GLIS2; Nanoparticle; Folate Receptor; CBFA2T3::GLIS2
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: ELU-FRα-AML-1; T2022-001 (Other Identifier: Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No