ENSAT registry-based randomized clinical trials for adrenocortical carcinoma

Area of Science:

• Oncology
• Clinical Trial Design
• Rare Diseases

Background:

• Adrenocortical carcinoma (ACC) is a rare endocrine malignancy with limited systemic treatment options.
• The low incidence of ACC poses significant challenges for traditional clinical trial funding and recruitment.
• Registry-based clinical trials present a novel, cost-effective strategy for rare disease research.

Purpose of the Study:

• To outline the rationale and feasibility of implementing randomized registry-based trials for adrenocortical carcinoma (ACC).
• To describe the potential adaptation of the European Network for the Study of Adrenal Tumours (ENSAT) registry for clinical trial infrastructure.
• To propose specific trial designs for ACC in adjuvant and advanced settings.

Main Methods:

• Review of the concept of registry-based clinical trials for rare orphan diseases.
• Analysis of the European Network for the Study of Adrenal Tumours (ENSAT) registry's suitability for clinical trial adaptation.
• Survey of ENSAT members regarding their interest and perceived scientific value of registry-based trials.
• Proposal of three distinct randomized registry-based trial designs for ACC.

Main Results:

• ENSAT members expressed strong interest and high perceived scientific potential for registry-based randomized trials in ACC.
• Legal and ethical approval processes for registry-based randomization were identified as key challenges.
• The ENSAT registry infrastructure can be developed to support future randomized trials for ACC.

Conclusions:

• Randomized registry-based trials are a viable and effective approach for advancing treatment strategies in adrenocortical carcinoma.
• Adapting the ENSAT registry provides a framework for conducting such trials, overcoming limitations of traditional studies for rare diseases.
• This approach offers a proof-of-principle for conducting more efficient randomized trials in orphan diseases.