Predicting Response to Immune Checkpoint Inhibitors Across Solid Tumors Using a Live Tumor Diagnostic Platform

Recruiting

• Conditions: Early Stage Triple-Negative Breast Carcinoma; Metastatic Bladder Urothelial Carcinoma; Metastatic Cervical Carcinoma; Metastatic Clear Cell Renal Cell Carcinoma; Metastatic Colorectal Carcinoma; Metastatic Endometrial Carcinoma; Metastatic Esophageal Carcinoma; Metastatic Liver Carcinoma; Metastatic Lung Non-Small Cell Carcinoma; Metastatic Malignant Skin Neoplasm
• Interventions: Procedure: Biospecimen Collection; Procedure: Tissue Collection

• Registration Number: NCT06349642
• Lead Sponsor: Mayo Clinic

Brief Summary

The primary purpose of this study is to determine the sensitivity of CYBRID Score for predicting in-vivo clinical response based on surgical response or RECIST 1.1 for neoadjuvant and locally advanced/metastatic patients, respectively. The secondary purposes is to determine the sensitivity of the CYBRID Score for predicting in-vivo clinical response based on surgical response or RECIST 1.1 for neoadjuvant and locally advanced/metastatic patients, respectively.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-04-01
• Study First Submitted QC: 2024-04-01
• Study First Posted: 2024-04-05
• Study Start: 2024-04-24
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-06
• Primary Completion: 2027-05
• Study Completion: 2027-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 324

Inclusion Criteria

• Subjects suspected of or diagnosed with Stage IV/metastatic or patients receiving neoadjuvant CPI for resectable early stage of solid malignancies:

  • Lung: Non-small cell lung cancer (NSCLC)
  • Kidney: Clear Cell Renal Cell Carcinoma (ccRCC)
  • Bladder: Urothelial Carcinoma (UC)
  • Skin: Cutaneous Malignancy, excluding Uveal Melanoma
  • Breast Cancer: Triple negative breast cancer (TNBC)
  • Liver and Esophageal cancers
  • Colon cancer: Mismatch repair deficient (dMMR) colorectal cancer (CRC) only
  • All tumor types with high tumor mutational burden (TMB_ or are microsatellite instability high (MSI-H)Mismatch repair deficient (dMMR) tumors
  • Cervical and endometrial cancer

• LOCALLY ADVANCED/METASTATIC PATIENTS: measurable disease as defined per protocol

  • NOTE: Tumor lesions in a previously irradiated area are not considered measurable disease; Disease that is measurable by physical examination only is not eligible.

• Eligible based on investigator discretion to receive CPI monotherapy or combination therapy (e.g. combining two CPIs such as anti-PD-1 plus anti-CTLA-4) or CPI therapy in combination with chemotherapy or genome-targeted therapy.

  • Subjects must be treatment-naïve at the time of biopsy if they are newly diagnosed
  • Subjects who are Stage I, II, or III and have progressed to metastatic cannot have received any anti-cancer treatment for at least 2-4 weeks (depending upon the washout period of prior anti-cancer treatment) prior to biopsy as per the agents used.
  • Subjects with a confirmed diagnosis who have previously undergone a standard of care (SOC) biopsy must be willing to undergo a separate biopsy procedure solely for the purposes of this study.

• Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0, 1 or 2

• Negative pregnancy test done ≤7 days prior to enrollment, for persons of childbearing potential only

• Female subjects must not be pregnant or breastfeeding

• Female subjects must use appropriate methods of contraception when applicable

• Clinically able, at investigator discretion, to undergo extra core needle biopsy passes during their biopsy from a tumor site that yields a biopsy of at least 10 mm in length

• Subjects with a known secondary cancer diagnosis are eligible to participate if participation does not interfere with systemic anti-cancer standard of care treatment for primary diagnosis

• Able to provide written informed consent

Exclusion Criteria

• Pregnant women because this study involves a greater than minimal risk procedure (study biopsy)

• Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety for the biopsy

• Immunocompromised patients and patients known to be HIV positive and currently receiving antiretroviral therapy

  • NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial

• Uncontrolled intercurrent illness including, but not limited to:

  • ongoing or active infection
  • psychiatric illness/social situations that would limit compliance with study requirements

• Receiving any investigational agent which would be considered as a treatment for the primary neoplasm

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Observational	Biospecimen Collection	Patients undergo tissue biopsy and may optionally undergo blood sample collection and have their medical records reviewed on study. Patients receive standard treatment with checkpoint inhibitors during the study and undergo standard tumor assessments during screening and follow-up.
Observational	Tissue Collection	Patients undergo tissue biopsy and may optionally undergo blood sample collection and have their medical records reviewed on study. Patients receive standard treatment with checkpoint inhibitors during the study and undergo standard tumor assessments during screening and follow-up.

Primary Outcome Measures

Name	Time	Method
Accuracy of the Elephas Score for predicting clinical response in neoadjuvant patients	Up to 3 years	Assessed based on pathologic complete response (pCR) during checkpoint inhibitor (CPI) treatment. Clinical non-response will be defined as non-pathological complete response. Patients who cannot have pathologic complete response determined will be excluded from the primary analysis.
Accuracy of the Elephas Score for predicting clinical response in locally advanced/metastatic patients	Up to 3 years	Assessed based on Response Evaluation Criteria In Solid Tumors (RECIST) score for best overall response \[complete response (CR) or partial response (PR)\] during checkpoint inhibitor (CPI) treatment. Clinical non-response will be defined as either stable disease (SD) or disease progression (PD). Patients who cannot have best response determined will be excluded from the primary analysis.

Trial Locations

Locations (1)

Mayo Clinic in Florida; 🇺🇸 Jacksonville, Florida, United States