Breathomics as Predictive Biomarker for Checkpoint Inhibitor Response

NCT04146064 | Completed

• 2 other identifiers: IO-Breathomics19-5936
• Study Type: observational
• Target: 190
• Locations: 1 country
• Sites: 1

Brief Summary

Immunotherapy with agents stimulating the immune system to act against cancer are now a new standard of care in various cancers as lung cancer and melanoma, but also bladder cancer, kidney cancer and head \& neck cancer. However, even though a subset of patients derives long-term benefit from these agents, depending of cancer type still at least half of patients do not respond to these new drugs. Our understanding of possible factors predicting whether a patient might actually benefit from immunotherapy is poor. Volatile organic compounds (VOCs) are gases exhaled with a person's breath, which are released into the lung from blood and bacteria and therefore can give information about infections as well as inflammation and possibly cancer cells in a person's body. Breath analysis of these VOCs with special devices called electronic noses (eNose) generate a specific electric signals patterns called breathprints. There is early evidence that specific breathprints can actually help to select patients who will be likely to benefit from immunotherapy. This study is being undertaken in an effort to evaluate breathprint analysis as a potential predicting factor for benefit from immunotherapy, so that treatment selection can further be improved. This study is designed to help us identify the role of breathprint analysis to better select patients for immunotherapy.

Conditions

NSCLC; Melanoma; Kidney Cancer; Urothelial Carcinoma; Head and Neck Cancer

Keywords

breathprint analysis; immunotherapy; predictive biomarker

Outcome Measures

Primary Outcomes (1)

• 12 week Progression Rate in validation cohort

  12 weeks

Secondary Outcomes (2)

• Overall Survival (OS) in validation cohort

  5 years

• Overall Response Rate (ORR) in validation cohort

  5 years

Other Outcomes (7)

• Progression-free survival (PFS) in validation cohort

  5 years

• 6 months clinical benefit rate (CR, PR or SD at 6 months after treatment start) in validation cohort

  6 months

• 12 week Progression Rate in exploratory cohorts

  12 weeks

Study Arms (5)

Validation cohort: NSCLC

Patients with advanced/metastatic NSCLC planned for IO-treatment in one of the following categories * Pembrolizumab monotherapy first-line * Pembrolizumab or nivolumab monotherapy in second or later line

Other: Breathprint analysis and patient-reported outcomes

Cohort 1: NSCLC

Patients with advanced/metastatic NSCLC planned for Pembrolizumab-chemotherapy combination therapy first-line

Other: Breathprint analysis and patient-reported outcomes

Cohort 2: Melanoma

Patients with advanced/metastatic melanoma planned for IO-treatment in one of the following categories * Nivolumab/ipilimumab combination treatment 1L * Pembrolizumab or nivolumab monotherapy treatment 1L * Ipilimumab monotherapy 2L

Other: Breathprint analysis and patient-reported outcomes

Cohort 3: Mixed solid tumor cohort

Patients with advanced/metastatic solid tumors such as Head\&Neck tumors, kidney cancer and urothelial cancer planned for IO-treatment

Other: Breathprint analysis and patient-reported outcomes

Cohort 4: NSCLC

Patients with advanced/metastatic NSCLC planned for treatment with Chemotherapy-only (either platinum-based combination treatment or docetaxel monotherapy)

Other: Breathprint analysis and patient-reported outcomes

Interventions

Breathprint analysis and patient-reported outcomes OTHER

Breathprint analysis: Patients will be providing breathprint samples into the eNose device at baseline and every 12 weeks thereafter as long as on immunotherapy treatment. Questionnaires will be completed at the same timepoints.

Cohort 1: NSCLC; Cohort 2: Melanoma; Cohort 3: Mixed solid tumor cohort; Cohort 4: NSCLC; Validation cohort: NSCLC

Eligibility Criteria

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

Study Population

Subjects included in this study are patients over the age of 18 years with a histologically-confirmed diagnosis of non-small cell lung cancer, melanoma or other solid tumor for which ICI-treatment is a standard of care. Patients must have advanced/metastatic disease and planning to receive ICI treatment (categories defined above) or chemotherapy treatment (cohort 4) at the Princess Margaret Cancer Centre/University Health Network. Study subjects will be consented specifically for that study.

You may qualify if:

• Patients 18 years of age or older
• Histologically confirmed advanced/metastatic non-small cell lung cancer, melanoma or solid tumor such as urothelial, kidney or head and neck cancer and planned treatment with
• NSCLC validation cohort: Pembrolizumab or Nivolumab
• NSCLC Cohort 1: Pembrolizumab-chemotherapy combination therapy 1L
• Melanoma Cohort 2: Nivolumab/ipilimumab combination treatment 1L, Pembrolizumab or nivolumab monotherapy treatment 1L , Ipilimumab
• Solid tumors Cohort 3: Any ICI-treatment, any line
• NSCLC Cohort 4: Chemotherapy-only (either platinum-based combination treatment or docetaxel monotherapy)
• At least one measurable lesion as defined by RECIST 1.1. A lesion at a previously irradiated site may only be counted as a target lesion if there is clear sign of progression since the irradiation.
• Able to provide informed consent.

You may not qualify if:

• Patients who are unable to perform the breathing manoeuvres needed for eNose-analysis of exhaled air.
• Patients who are unable to independently consent to participation in the trial.
• Patients with severe, acute, or chronic medical conditions (including uncontrolled diabetes mellitus) or psychiatric conditions or laboratory abnormalities that in the opinion of the Investigator or their physician may cause undue harm or inconvenience to the patient, or that may interfere with the interpretation of study results.

Sponsors & Collaborators

• University Health Network, Toronto: lead
• University of Amsterdam: collaborator

Study Sites (1)

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Melanoma; Kidney Neoplasms; Carcinoma, Transitional Cell; Head and Neck Neoplasms

Interventions

Patient Reported Outcome Measures

Condition Hierarchy (Ancestors)

Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Nevi and Melanomas; Skin Neoplasms; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases; Urologic Neoplasms; Urogenital Neoplasms; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Carcinoma; Neoplasms, Glandular and Epithelial

Intervention Hierarchy (Ancestors)

Health Care Surveys; Surveys and Questionnaires; Data Collection; Epidemiologic Methods; Investigative Techniques; Health Services Research; Health Planning; Health Care Economics and Organizations; Patient Outcome Assessment; Outcome Assessment, Health Care; Outcome and Process Assessment, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation; Health Care Evaluation Mechanisms; Public Health; Environment and Public Health

Study Officials

• Geoffrey Liu, MD MSc

  UHN

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2019
• First Posted: October 31, 2019
• Study Start: February 24, 2020
• Primary Completion: February 9, 2026
• Study Completion: February 9, 2026
• Last Updated: May 8, 2026

Record last verified: 2026-05

Locations

CA (1)