A Study to Evaluate ELU001 in Patients With Solid Tumors That Overexpress Folate Receptor Alpha (FRα)

Sponsor
Elucida Oncology (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05001282
Collaborator
(none)
166
Enrollment
7
Locations
1
Arm
42
Anticipated Duration (Months)
23.7
Patients Per Site
0.6
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study, ELU- FRα-1, is focused on adult subjects who have advanced, recurrent or refractory folate receptor alpha (FRα) overexpressing tumors considered to be topoisomerase 1 inhibitor-sensitive based on scientific literature, and, in the opinion of the Investigator, have no other meaningful life-prolonging therapy options available.

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.

Condition or DiseaseIntervention/TreatmentPhase
Phase 1/Phase 2

Detailed Description

The study has two parts: Part 1 Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D), and Part 2 Tumor Group Expansion Cohort(s) where specific cancer types will be evaluated for efficacy and safety at the RP2D.

Part 1, subjects with cancer types with a high likelihood of having FRα overexpressing tumors based on historical data, specifically, ovarian, endometrial, colorectal, gastric, gastroesophageal junction, triple negative breast, or non-small cell lung cancers, or cholangiocarcinoma, will be enrolled in a basket clinical study. Retrospective analysis of folate receptor alpha (FRα) expression status will be determined.

Part 2 Stage 1 of a Simon's Two-Stage design, tumor group expansion cohorts, each consisting of subjects with cancer types studied as part of the basket in Part 1.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
166 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Dose Escalation Dose Expansion, Simon's 2-stageDose Escalation Dose Expansion, Simon's 2-stage
Masking:
None (Open Label)
Masking Description:
Open Label
Primary Purpose:
Treatment
Official Title:
Dose Escalation and Expansion Clinical Study to Evaluate the Safety and Efficacy of ELU001 in Subjects Who Have Advanced, Recurrent or Refractory FRα Overexpressing Tumors
Actual Study Start Date :
Sep 13, 2021
Anticipated Primary Completion Date :
Mar 15, 2024
Anticipated Study Completion Date :
Mar 15, 2025

Arms and Interventions

ArmIntervention/Treatment
Experimental: ELU001

Dose Escalation: Escalating doses of ELU001 Dose Expansion: Recommended Dose for Expansion (or RP2D)

Drug: ELU001
Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)
Other Names:
  • FA-CDC
  • Outcome Measures

    Primary Outcome Measures

    1. Part 1 Dose Escalation: The Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 for IV infusion in patients with over-expressing Folate Receptor Alpha tumors. [28 days]

      To determine the MTD and/or RP2D of ELU001 in patients with over-expressing Folate Receptor Alpha tumors. When at least 2 out of 6 patients in a given dose-level experience a dose-limiting toxicity (DLT), the dose is considered to have exceeded the MTD. The MTD, therefore, is defined as the previous highest tested dose of ELU001 that did not cause a DLT in the first cycle of treatment. In the event of emerging data during Part 1, the Sponsor may, in consultation with a dose-level safety review group, decide to define a recommended dose for expansion (in for Part 2) (RP2D) instead of the MTD. DLTs are defined as a treatment-emergent adverse event (TEAE) or abnormal laboratory value related to ELU001 treatment that result in a failure to meet the criteria for re-treatment.

    2. Part 2 Dose Expansion: To determine overall response rate (ORR) (complete response [CR] + partial response [PR]) of ELU001 for IV infusion in patients with over-expressing Folate Receptor Alpha tumors, per RECIST v1.1. [First dose of study drug until responses of CR or PR, assessed up to 12 months.]

      Percentage of Participants with confirmed objective response as assessed by the investigator and per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 CR is defined as the disappearance of all target or non-target lesions. PR is defined as at least 30 percent (%) decrease in the sum of the longest diameters (SoD) of target lesions, taking as reference the baseline SoD.

    Secondary Outcome Measures

    1. Part 1 Dose Escalation: To determine overall response rate (ORR) (complete response [CR] + partial response [PR]) of ELU001 for IV infusion in patients with over-expressing Folate Receptor Alpha tumors, per RECIST v1.1. [First dose of study drug until responses of CR or PR, assessed up to 12 months.]

      Percentage of Participants with confirmed objective response as assessed by the investigator and per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 CR is defined as the disappearance of all target or non-target lesions. PR is defined as at least 30 percent (%) decrease in the sum of the longest diameters (SoD) of target lesions, taking as reference the baseline SoD.

    2. Part 1 and Part 2: To determine Duration of Response (DOR) of ELU001 for IV infusion in patients with over-expressing Folate Receptor Alpha tumors. [Date of first response (CR or PR) until the date of disease progression or up to 12 months, whichever occurs first.]

      Time Measurement: DOR will begin at the date that a response has been identified (stable disease, partial response, or complete response) until the date of progressive disease (PD). PD is defined of at least a 20% increase in the sum of the longest diameters of the target lesions from data of initial response.

    3. Part 1 and Part 2: Number of participants with adverse events as assessed by CTCAE v5.0 Safety Evaluations. [First dose of study drug up to 28 days after the last dose of study drug or up to 12 months, whichever occurs first.]

      The number of participants Frequency/severity of abnormalities in vital signs measurements, physical examination findings, changes in clinical laboratory parameters, and incidence of adverse events after taking ELU001.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:
    Patients must meet the following criteria to enroll in this study:
    • Documented diagnosis of ovarian cancer, endometrial cancer, colorectal cancer, gastric cancer, gastroesophageal junction cancer, triple negative breast cancer, non-small cell lung cancer, or cholangiocarcinoma

    • No other meaningful life-prolonging therapy option available

    • Must provide archival tumor tissue or a newly obtained tumor biopsy specimen prior to the first dose of ELU001 for folate receptor alpha (FRα) expression analysis

    • Adequate organ function

    • Measurable disease, or in the absence of measurable disease, non-measurable disease as per Response evaluation criteria in solid tumors (RECIST) v1.1

    • Part 1: Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2; Part 2: ECOG performance status of 0 or 1.

    • Recovered from previous surgeries

    • Agree to highly effective contraception, not to get pregnant, or for men, not father a child during study participation

    Key Exclusion Criteria:
    Patients who meet any of the following are not eligible to enroll in this study:
    • Active or ongoing eye disorders

    • Taken any treatments that use the protein folate receptor alpha or FRα to work

    • Taken any other experimental treatments

    • History of significant cardiac issues or other cancers within 3 years.

    • Significant anemia, significant neutropenia, or significant thrombocytopenia (e.g., not enough platelets in your blood - platelets held stop bleeding in your body)

    • Detectable viral load for HIV (human immunodeficiency virus), hepatitis B or C.

    • If you are pregnant.

    • Part 1: Cannot have active autoimmune diseases such as rheumatoid arthritis, SLE (systemic lupus erythematosus), ulcerative colitis, Crohn's Disease, MS (multiple sclerosis), ankylosing spondylitis, thyroiditis that require treatments that suppress your immune system.

    • Part 1: if your cancer has spread to your brain.

    • Part 2: You can have cancer that has spread to your brain but there are exceptions. The cancer in your brain cannot be causing any symptoms, it cannot be larger than 3 cm, there can be no evidence on a scan that shows your brain tissue has shifted from its expected position inside the skull (called "herniation") or be bleeding in the skull or brain itself (called "hemorrhage").

    Contacts and Locations

    Locations

    SiteCityStateCountryPostal Code
    1Mayo Clinic - Phoenix, AZPhoenixArizonaUnited States85054
    2Mayo Clinic - Jacksonville, FLJacksonvilleFloridaUnited States32224
    3Sarah Cannon Research Institute at Florida Cancer SpecialistsOrlandoFloridaUnited States32827
    4Mayo Clinic - Rochester, MNRochesterMinnesotaUnited States55905
    5Sarah Cannon Research Institute at Tennessee OncologyNashvilleTennesseeUnited States37203
    6Mary Crowley Cancer ResearchDallasTexasUnited States75230
    7New Experimental Therapeutics of San Antonio (NEXT Oncology)San AntonioTexasUnited States78229

    Sponsors and Collaborators

    • Elucida Oncology

    Investigators

    • Study Director: Eliel Bayever, MBBCh, MRCP, Elucida Oncology, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    Responsible Party:
    Elucida Oncology
    ClinicalTrials.gov Identifier:
    NCT05001282
    Other Study ID Numbers:
    • ELU-FRα-1
    First Posted:
    Aug 11, 2021
    Last Update Posted:
    Mar 22, 2022
    Last Verified:
    Mar 1, 2022

    Study Results

    No Results Posted as of Mar 22, 2022