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

Elucida Oncology

Status and phase

Withdrawn

Phase 1

Conditions

AML, Childhood

Refractory Pediatric AML

Acute Myeloid Leukemia

Relapsed Pediatric AML

Treatments

Drug: ELU001

Study type

Interventional

Funder types

Industry

Other

Identifiers

NCT05622591

ELU-FRα-AML-1

Details and patient eligibility

About

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.

Full 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.

Sex

All

Ages

1 month to 9 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.