Accepted for/Published in: JMIR Research Protocols
Date Submitted: Jan 19, 2022
Date Accepted: Jun 13, 2022
How to individualise oncological treatment of patients with metastatic non-clear cell renal cell carcinoma by using gene sequencing and Patient-Reported Outcomes: INDIGO study protocol
ABSTRACT
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. One approach is individualising treatment based on gene sequencing of tumour tissue. Also, recent studies with Patient-Reported Outcome (PRO) in patients treated for metastatic cancer have shown a significant benefit in quality of life, median- and overall survival. The use of gene sequencing and PRO can be of great importance to patients with rare cancer types including patients with non-cc RCC, and should be investigated in clinical trials, especially where evidence based on phase III studies is difficult to achieve.
Objective:
We describe the INDIGO study where patients based on gene analyses are allocated into four treatment arms containing fourteen treatments and use electronic PRO (ePRO). We aim to improve the treatment of patients with non-cc RCC and the endpoints in the study is overall response rate (ORR), both complete and partial, in the total patient population based on RECIST v1.1 criteria, and time to treatment failure (TTF).
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 electronic questionnaires in a dedicated app: one questionnaire regarding symptoms and side effects and one regarding health-related quality of life (HRQoL). Depending on treatment regimen, the patient is seen by a medical doctor every third, fourth, or sixth week and the effect of the systemic treatment is evaluated every six weeks with a CT-scan.
Results:
Recruitment started in March 2020 and at the time of submitting the manuscript 9 patients have been enrolled. The 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.
Conclusions:
We aim to explore methods to improve treatment outcome for patients with non-cc RCC and the INDIGO study will contribute with further data on personalised medicine in rare types of renal cell carcinoma and provide new knowledge on an active use of ePRO. Clinical Trial: ClinicalTrials.gov Identifier: NCT04644432 EudraCT: 2019-001316-38 Danish National Committee on health research ethics: H-19041833
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