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

NCT05622591 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: ELU-FRα-AML-1
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Acute Myeloid Leukemia; AML, Childhood; Relapsed Pediatric AML; Refractory Pediatric AML

Keywords

CBFA2T3-GLIS2; CBFA2T3::GLIS2; Refractory; Folate Receptor alpha; Acute Myeloid Leukemia; ELU001; Exatecan; C'Dot drug conjugate; C-Dot; C'Dot; Childhood; Nanoparticle; Folate Receptor; AML; Relapsed

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Evaluate preliminary anti-leukemic activity of ELU001

  First dose of study drug until 42 days after last cycle.

• Characterize the pharmacokinetics of ELU001

  First dose of study drug until 42 days after last cycle.

• Characterize the immunogenicity of ELU001

  First dose of study drug until 42 days after last cycle.

Study Arms (1)

ELU001

EXPERIMENTAL

Dose Escalation: Escalating doses of ELU001

Drug: ELU001

Interventions

ELU001 DRUG

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

ELU001

Eligibility Criteria

• Age: 1 Month - 9 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Elucida Oncology: lead

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose Escalation

Study Record Dates

• First Submitted: November 8, 2022
• First Posted: November 18, 2022
• Study Start: June 7, 2024
• Primary Completion: June 7, 2024
• Study Completion: June 7, 2024
• Last Updated: August 7, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share