Overcoming obstacles in the design of cancer anorexia/weight loss trials

- To date, no intervention has been demonstrated to improve all outcomes in patients who suffer from cancer anorexia/weight loss.
- The design of high-quality clinical trials may lead to improved clinical outcomes in patients with advanced cancer.
- In designing trials for cancer anorexia/weight loss, multiple design issues should be carefully considered, as reviewed here.

Most advanced cancer patients suffer loss of appetite (anorexia) and loss of weight. Despite the fact that cancer anorexia and weight loss are associated with a poor prognosis and detract from quality of life, no interventions have been demonstrated to palliate this syndrome in its entirety, particularly in patients with treatment-refractory malignancies. Recently, two registration trials − one with anamorelin and another with enobosarm − failed to reach their primary endpoints, thus raising questions. Were both these agents ineffective? Alternatively, did study design issues compromise the ability of these trials to identify effective agents? Thus, this review is timely insofar it serves as an introduction to study design, offers guidance on how to test promising agents for cancer anorexia/weight loss, and provides advice for overcoming trial design obstacles.