Prospective Study to Evaluate Patient Reported Outcomes (PRO) During Rechallenge With Trabectedin in Sarcoma Patients

Study Overview
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Brief Summary
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Chemotherapy with Trabectedin is an effective treatment for sarcoma patients in the second line setting or in first line for patients who are deemed unsuitable to receive anthracycline therapy. The prospective study will primarily investigate the PRO of Trabectedin rechallenge in patients with soft tissue sarcomas that had to discontinue initial Trabectedin treatment due to various reasons such as progression, side effects or surgery, as is commonly the case in real-life settings. Embedding this trial in the GISAR registry enables the evaluation of a large number of patients with long-term follow-up which allows multiple analyses regarding different questions that remain unanswered until today.

Detailed Description
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Chemotherapy with Trabectedin is an effective treatment for sarcoma patients in the second line setting or in first line for patients who are deemed unsuitable to receive anthracycline therapy. Several features differentiate its clinical performance from other chemotherapeutic agents, including durability of response and the absence of cumulative toxicity even upon re-exposition to treatment after interruption of therapy. In addition, the results of the T-Dis trial evaluating the impact of Trabectedin rechallenge demonstrated that it retains its activity when patients are rechallenged on progression after a treatment break. Since sarcoma patient usually report a worse global quality of life (QoL) compared to the general population, one of the main goals of treating advanced-stage patients with Trabectedin, in addition to its efficacy, is to improve the QoL, represented by symptom control and other patient-related outcomes. However, little is known to date about the QoL during a rechallenge with Trabectedin, after patients had to discontinue Trabectedin treatment. Patient-reported outcome (PRO) measures are standardized questionnaires that collect information on health outcomes directly from the patients, including symptoms, health-related QoL parameters and functional status. In general, there are two types of PROMs, disease-specific and general measures that can also be used for healthy populations. A growing number of articles supports the need for, as well as the benefit of, deriving information directly from patients. Thus, the application of PRO measures, initially developed for the use in research, has become more common nowadays and their application is included in supporting clinical decision making, comparing outcomes among health-care providers, stimulating quality improvement and evaluating practices and policies. Therefore, the collection of real-world data from patients subject to Trabectedin rechallenge would enable further estimations on the feasibility and efficacy of Trabectedin rechallenge. The prospective PROTraSarc sub-study will primarily investigate the PRO of Trabectedin rechallenge in patients with soft tissue sarcomas that had to discontinue initial Trabectedin treatment due to various reasons such as progression, side effects or surgery, as is commonly the case in real-life settings. Embedding this trial in the GISAR registry enables the evaluation of a large number of patients with long-term follow-up which allows multiple analyses regarding different questions that remain unanswered until today.

Official Title
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PROTraSarc Prospective Study to Evaluate Patient Reported Outcomes (PRO) During Rechallenge With Trabectedin in Sarcoma Patients - A GISAR Sub-study

Conditions
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Soft Tissue Sarcoma

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Enrolled participant of the GISAR registry and their sub-studies Patient with histologically confirmed soft tissue sarcomas Pretreatment with Trabectedin, termination of this therapy regardless of reason Planned rechallenge or started rechallenge with Trabectedin in any line of therapy within 2 weeks before inclusion Interval between last Trabectedin treatment and Trabectedin rechallenge at least 3 months Exclusion Criteria: Not able to understand all implications of study participation No written informed consent Age ≤ 18 years

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Trabectedin Rechallenge in Soft sarcoma patients<br>Adult GISAR participants with Trabectedin-pretreated soft tissue sarcomas and rechallenge with at least one cycle of Trabectedin in any line of therapy, with the interval between Trabectedin interruption and Trabectedin rechallenge being at least 3 months. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patient reported outcome regarding symptoms and side effects during Trabectedin re-challenge in soft tissue sarcoma patients | PRO-CTCAE questionnaire | through study completion, an average of 2 years | 
| Patient reported outcome regarding distress during Trabectedin re-challenge in soft tissue sarcoma patients | Distress-Thermometer | through study completion, an average of 2 years | 
| Patient reported outcome regarding quality of life during Trabectedin re-challenge in soft tissue sarcoma patients | QLQ-C30 Questionnaire | through study completion, an average of 2 years | 
| Patient reported outcome regarding cancer behaviour inventory | CBI-B Questionnaire | every nine to 18 weeks during Trabectedin re-challenge, every 3 Month in follow up | 
| Patient reported outcome measuring anxiety and depression | PHQ-4 Questionnaire | through study completion, an average of 2 years | 
| Patient reported outcome measuring self-efficacy for managing chronic disease | SES6G Questionnaire | through study completion, an average of 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Analysis of correlation of PROs with clinical parameters | Evaluation of different PROs and analysis of correlation with clinical parameters, e.g. therapy response and side effects experienced during therapy | through study completion, an average of 2 years | 
| Characterization of patients receiving Trabectedin rechallenge in real life setting | Characterization of patients receiving Trabectedin rechallenge in real life setting, e.g. demographics, previous therapies | through study completion, an average of 2 years | 
| Characterization of Trabectedin rechallenge in real life conditions | Characterization of Trabectedin rechallenge in real life conditions, e.g. number of Trabectedin cycles applied, response to therapy, reason for end of Trabectedin rechallenge (e.g. disease progression, intolerable toxicity, death) | through study completion, an average of 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PRO, Re-Challenge, Trabectedin, Sarcoma, registry