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

NCT05001282 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: ELU-FRα-1
• Study Type: interventional
• Target: 79
• Locations: 1 country
• Sites: 18

Brief Summary

This study, ELU- FRα-1, was 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.

Conditions

Ovarian Cancer; Ovarian Neoplasms; Ovarian Carcinoma; Endometrial Cancer; Ovary Cancer; Ovary Neoplasm; Ovary Disease; Ovary Metastasis; Ovarian Diseases; Ovarian Cancer Stage; Ovarian Epithelial Cancer; Ovarian Adenocarcinoma; Ovarian Serous Adenocarcinoma; Ovarian Neoplasm Epithelial; Ovarian Cancer Recurrent; Endometrial Diseases; Endometrial Adenocarcinoma; Endometrial Carcinosarcoma; Endometrial Clear Cell Adenocarcinoma; Endometrioid Adenocarcinoma; Endometrial Neoplasms; Endometrial Cancer Recurrent; Endometrioid Tumor; Fallopian Tube Cancer; Peritoneal Cancer

Keywords

Folate Receptor Alpha; Carcinomas; Recurrent, Refractory; Folate Receptor alpha moderate expression; Folate Receptor alpha high expression; ELU001; Exatecan; C'Dot; C-Dot; Folic Acid; Payload; Nanoparticle; C'Dot drug conjugate; CDC; Linker; ELU-FRa-1; FOLR1

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  28 days

• 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.

  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.

  First dose of study drug until responses of CR or PR, assessed up to 12 months.

Secondary Outcomes (3)

• 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.

• 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.

• 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.

Study Arms (1)

ELU001

EXPERIMENTAL

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

Drug: ELU001

Interventions

ELU001 DRUG

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

Also known as: FA-CDC

ELU001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must meet the following criteria to enroll in this study:
• Part 1 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
• Part 2 Ovarian Cancer or Endometrial Cancer
• 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. Previous FRα expression test results may be used in certain circumstances
• 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

You may not qualify if:

• Patients who meet any of the following are not eligible to enroll in this study:
• Clinically significant 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").

Sponsors & Collaborators

• Elucida Oncology: lead

Study Sites (18)

Mayo Clinic - Phoenix, AZ

Phoenix, Arizona, 85054, United States

Location

Providence Medical Foundation

Fullerton, California, 92835, United States

Location

Mayo Clinic - Jacksonville, FL

Jacksonville, Florida, 32224, United States

Location

D&H Cancer Research Center

Margate, Florida, 33063, United States

Location

Sarah Cannon Research Institute at Florida Cancer Specialists

Orlando, Florida, 32827, United States

Location

Hope and Healing Cancer Services

Hinsdale, Illinois, 60521, United States

Location

OSF Saint Francis Medical Center

Peoria, Illinois, 61606, United States

Location

Mayo Clinic - Rochester, MN

Rochester, Minnesota, 55905, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Medical Center - Duke Cancer Institute

Durham, North Carolina, 27719, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Thomas Jefferson University, Sidney Kimmel Cancer Center

Philadelphia, Pennsylvania, 19107, United States

Location

Women & Infants Hospital of Rhode Island

Providence, Rhode Island, 02905, United States

Location

Avera Cancer Institute

Sioux Falls, South Dakota, 57105, United States

Location

Sarah Cannon Research Institute at Tennessee Oncology

Nashville, Tennessee, 37203, United States

Location

Mary Crowley Cancer Research

Dallas, Texas, 75230, United States

Location

New Experimental Therapeutics of San Antonio (NEXT Oncology)

San Antonio, Texas, 78229, United States

Location

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Ovarian Neoplasms; Endometrial Neoplasms; Ovarian Diseases; Krukenberg Tumor; Carcinoma, Ovarian Epithelial; Uterine Diseases; Carcinoma, Endometrioid; Fallopian Tube Neoplasms; Carcinoma; Recurrence

Condition Hierarchy (Ancestors)

Endocrine Gland Neoplasms; Neoplasms by Site; Neoplasms; Adnexal Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Neoplasms, Female; Urogenital Neoplasms; Genital Diseases; Endocrine System Diseases; Gonadal Disorders; Uterine Neoplasms; Carcinoma, Signet Ring Cell; Adenocarcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Cystic, Mucinous, and Serous; Fallopian Tube 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 Dose Expansion, Simon's 2-stage

Study Record Dates

• First Submitted: July 14, 2021
• First Posted: August 11, 2021
• Study Start: September 13, 2021
• 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

Locations

US (18)