Development and Validation of a Health-related Quality of Life Instrument

Study Overview
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Brief Summary
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This study aims to develop a health related quality of life (HRQOL) patient reported outcome (PRO) for patients receiving immune checkpoint modulator (ICM) therapy. This instrument could be used to in clinical trials to evaluate HRQOL for patients treated with novel ICMs and ICM combinations that might ultimately influence decisions about regulatory approval, as well as improved understanding of chronic treatment effects on patient well-being, understanding reasons for treatment non-adherence, and developing strategies to improve adherence and evaluating clinical outcomes.

Detailed Description
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Immune Checkpoint Modulators (ICMs) are associated with significant adverse events. Currently there are no validated patient reported outcome (PRO) tools to assess health related quality of life (HRQOL) specifically in patients receiving ICMs. HRQOL is a multidimensional concept of a patient's perceived well-being that can be affected by disease and treatment. There is an urgent need to assess the impact of ICMs on HRQOL, to better understand the balance between efficacy and toxicity with these agents. This study aims to develop a health related quality of life (HRQOL) patient reported outcome (PRO) for patients receiving immune checkpoint inhibitor (ICM) therapy (FACT-ICM). The core domains of the FACT-ICM will include physical, emotional, family and social, and functional well being. In addition, the proposed study will develop a toxicity sub-scale related to resultant toxicities from ICM therapy. The HRQOL will be developed through focus groups and interviews with patients treated with ICMs, and clinicians treating patients with ICMs, to understand how treatment with ICMs impacts HRQOL, as well as explore general experiences, insights and concerns of those receiving ICM therapies.

Official Title
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Development and Validation of a Health-related Quality of Life Instrument: Functional Assessment of Cancer Therapy - Immune Checkpoint Modulators, an Investigator Initiated Study (FACT-ICM).

Conditions
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Cancer

Intervention / Treatment
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* Other: Focus groups and individual interviews
* Other: Questionnaire


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient Criteria Cancer diagnosis (any type, advanced or metastatic stage); Treatment with an agent or agents that modulate an immune checkpoint (any line of therapy); Age ≥ 18; English-speaking; ECOG PS 0-3 Able to complete questionnaires independently Able to provide informed consent. Completed treatment within a year of enrolment Clinician Criteria Must either treat patients with ICMs or manage side effects of patients treated with ICMs Be able to attend interviews and participate in discussions as part of the study

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| 1A: Content Validation<br>Up to 4 focus groups of 6-10 cancer patients each will be conducted each lasting approximately 90 mins. An additional 10 to 15 patients will undergo individual semi-structured interviews each lasting around 60 minutes. 10-12 expert clinicians experienced in treating patients with ICMs or managing ICM toxicities will participate in a survey, and group or individual interviews. | Other: Focus groups and individual interviews<br>* Focus groups conducted to explore general experiences, insights and concerns of those receiving ICM therapies. Individual semi-structured interviews to provide in-depth data on the themes identified in the focus groups.<br>|
| 1B: Face Validity<br>Patients who have been and are being treated with ICMs to complete draft questionnaire (FACT-ICM). Some patients who were involved in the first round of interviews will be re-interviewed, and interview naïve patients will also be included. | Other: Questionnaire<br>* Health related quality of life questionnaire (FACT-ICM) to be completed by patients<br>|
| 2A: To measure test-retest reliability<br>Patients to complete FACT-ICM at at two time points separated by 5 to 14 days. | Other: Questionnaire<br>* Health related quality of life questionnaire (FACT-ICM) to be completed by patients<br>|
| 2B: To confirm construct validity<br>To evaluate discriminative properties of FACT-ICM, scores will be compared between pre-defined groups of patients where differences are expected. | Other: Questionnaire<br>* Health related quality of life questionnaire (FACT-ICM) to be completed by patients<br>|
| 2C: To determine responsiveness and MCID<br>Responsiveness testing: Patients will complete FACT-ICM within a week of starting treatment, then while on treatment and within 30 days after end of treatment (EOT) with ICMs. MCID testing: In addition to the FACT-ICM score, patients undergoing serial assessment for responsiveness will also indicate how much better or worse they are using a 5-point rating scale. | Other: Questionnaire<br>* Health related quality of life questionnaire (FACT-ICM) to be completed by patients<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Confirmatory factor analysis of FACT-ICM PRO tool variables compared to content analysis and qualitative descriptives of patient focus groups and interviews in target group |  | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change of score for each domain and also the total FACT-ICM score at baseline to 5-14 days for patients with minimal change in status |  | 5-14 days | 
| Comparison of FACT-ICM scores between pre-defined groups of patients where differences are expected |  | 6 months | 
| FACT-ICM change scores from baseline to within 30 days after end of treatment |  | Baseline, an average of 3 months, and within 30 days after end of treatment | 
| Change in MCID values from baseline to within 30 days after end of treatment |  | Baseline, an average of 3 months, and within 30 days after treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ICMs, Immune checkpoint modulators