Feasibility of electronic Patient-Reported Outcomes in a complex setting for patients with metastatic non-clear cell renal cell carcinoma: Protocol for the INDIGO basket study (Preprint)
Authors: Rasmussen Ida Marie , Soerensen Anne Vest , Hundahl Moeller Anne Kirstine , Persson Gitte , Palshof Jesper Andreas , Taarnhoej Gry Assam , Pappot Helle 
Published: January 2022

BACKGROUND

No phase III studies have yet been conducted for patients with non-clear cell renal cell carcinoma (non-cc RCC) exclusively due to the rare occurrence of the disease and the heterogenicity in tumour morphology. Consequently, there is no evidence of the optimal treatment and new approaches are needed. An active, real-time use of patient-reported outcomes (PRO) is gaining ground in oncology and allows for individualization of care for the patients, which also is warranted in the present population. Recent studies with PRO in patients treated for metastatic cancer have shown a significant benefit in quality of life, median- and overall survival. The use of PRO as endpoint in clinical studies can be of great importance to patients with rare cancer types including patients with non-cc RCC, for whom treatment is highly individualised, and evidence based on phase III studies is difficult to achieve.

OBJECTIVE

We describe the INDIGO study with focus on active and passive use of electronic patient-reported outcomes (ePRO) in a complex study setting with four different treatment arms containing fourteen treatment options for patients with non-cc RCC. By using new treatments and adding ePRO to the treatment trajectory we aim to improve treatment of the rare subtypes of RCC.

METHODS

INDIGO is a prospective phase II trial and 30 patients will be enrolled. The patients receive systemic treatment based on genetic analyses of their tumour tissue. All patients will receive two electronic questionnaires: one questionnaire regarding symptoms and side effects is sent weekly and one regarding health-related quality of life is sent every four weeks. The questionnaires are completed in a dedicated electronic app with an interface developed specifically for the INDIGO study. Healthcare professionals daily assess the incoming responses from the patients and can contact the patient to initiate or improve supportive care.

In relation to PRO, the study has the following endpoints: Use of PRO tools during treatment both in terms of completion of weekly questionnaires, in terms of possible patterns in the completion in different subgroups of the patients and in terms of following the instructions regarding contact to the hospital.

RESULTS

The INDIGO study is approved by the Danish Medicines Agency, the National Committee on Health Research Ethics and complies with Good Clinical Practice guidelines, follow General Data Protection Regulation and is registered at the Capital Region of Denmark. Recruitment of patients started in March 2020.

CONCLUSIONS

We aim to explore methods to improve treatment outcome for patients with non-cc RCC and this feasibility study will demonstrate how ePRO can be implemented in a basket trial by applying active and passive PRO to a phase II treatment study.

CLINICALTRIAL

ClinicalTrials.gov Identifier: NCT04644432
EudraCT: 2019-001316-38
Danish National Committee on health research ethics: H-19041833