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

Primary Purpose

CBFA2T3-GLIS2-positive Acute Myeloid Leukemia, AML, Childhood, Relapsed Pediatric AML

Status
Not yet recruiting
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
ELU001
Sponsored by
Elucida Oncology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for CBFA2T3-GLIS2-positive Acute Myeloid Leukemia focused on measuring AML, Relapsed, Refractory, Folate Receptor alpha, Acute Myeloid Leukemia, ELU001, Exatecan, C'Dot drug conjugate, C-Dot, C'Dot, Childhood, CBFA2T3-GLIS2, Nanoparticle, Folate Receptor

Eligibility Criteria

1 Month - 9 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    ELU001

    Arm Description

    Dose Escalation: Escalating doses of ELU001

    Outcomes

    Primary Outcome Measures

    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.

    Secondary Outcome Measures

    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
    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
    Characterize the immunogenicity of ELU001
    Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).

    Full Information

    First Posted
    November 8, 2022
    Last Updated
    November 14, 2022
    Sponsor
    Elucida Oncology
    Collaborators
    Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL)
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05622591
    Brief Title
    ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML
    Acronym
    Pediatric AML
    Official Title
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2022
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    March 2023 (Anticipated)
    Primary Completion Date
    September 2025 (Anticipated)
    Study Completion Date
    April 2026 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Elucida Oncology
    Collaborators
    Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL)

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    This research study focuses 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. ELU001 is a new chemical entity described as a C'Dot drug conjugate (CDC), consisting of payloads (exatecans) and targeting moieties (folic acid analogs) covalently bound by linkers to the C'Dot particle carrier. ELU001 will be the first drug-conjugate of its kind to be introduced into the clinic, a first in class, and a novel molecular entity.
    Detailed Description
    Treatment will be given for 2 cycles of 28 days, for a total treatment of 2 months. At the end of each treatment cycle, there is an evaluation to see how the leukemia is responding to therapy. After completing two cycles, patients may enter into the Optional Treatment Continuation Period if the doctor believes that there is clinical benefit. This is 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. The drug is given by an infusion through a vein over a period of about one hour. ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet. But it is believed that ELU001 could play a role in stopping this type of leukemia from getting worse and offer a treatment option that may have less side effects. This is because it focuses on leukemic cells that have increased levels of FRα more than it targets healthy cells which express normal or no FRα.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    CBFA2T3-GLIS2-positive Acute Myeloid Leukemia, AML, Childhood, Relapsed Pediatric AML, Refractory Pediatric AML
    Keywords
    AML, Relapsed, Refractory, Folate Receptor alpha, Acute Myeloid Leukemia, ELU001, Exatecan, C'Dot drug conjugate, C-Dot, C'Dot, Childhood, CBFA2T3-GLIS2, Nanoparticle, Folate Receptor

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Model Description
    Dose Escalation
    Masking
    None (Open Label)
    Masking Description
    Open Label
    Allocation
    N/A
    Enrollment
    21 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    ELU001
    Arm Type
    Experimental
    Arm Description
    Dose Escalation: Escalating doses of ELU001
    Intervention Type
    Drug
    Intervention Name(s)
    ELU001
    Intervention Description
    Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)
    Primary Outcome Measure Information:
    Title
    Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001
    Description
    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.
    Time Frame
    28 days
    Secondary Outcome Measure Information:
    Title
    Evaluate preliminary anti-leukemic activity of ELU001
    Description
    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
    Time Frame
    First dose of study drug until 42 days after last cycle.
    Title
    Characterize the pharmacokinetics of ELU001
    Description
    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
    Time Frame
    First dose of study drug until 42 days after last cycle.
    Title
    Characterize the immunogenicity of ELU001
    Description
    Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).
    Time Frame
    First dose of study drug until 42 days after last cycle.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Month
    Maximum Age & Unit of Time
    9 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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.

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Links:
    URL
    http://www.elucidaoncology.com
    Description
    Elucida Oncology Corporate Website

    Learn more about this trial

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

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