Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia

BackgroundCancer cachexia is associated with reduced body weight, appetite and quality of life (QOL) with no approved treatments. Growth hormone secretagogues like macimorelin have potential to mitigate these effects. MethodsThis pilot study assessed the safety and efficacy of macimorelin for 1 week. Efficacy was defined a priori as 1-week change in body weight (>= 0.8 kg), plasma insulin-like growth factor (IGF)-1 (>= 50 ng/mL) or QOL (>= 15%). Secondary outcomes included food intake, appetite, functional performance, energy expenditure and safety laboratory parameters. Patients with cancer cachexia were randomized to 0.5 or 1.0 mg/kg macimorelin or placebo; outcomes were assessed non-parametrically. ResultsParticipants receiving at least one of either macimorelin dose were combined (N = 10; 100% male; median age = 65.50 +/- 2.12) and compared with placebo (N = 5; 80% male; median age = 68.00 +/- 6.19). Efficacy criteria achieved: body weight (macimorelin N = 2; placebo N = 0; P = 0.92); IGF-1 (macimorelin N = 0; placebo N = 0); QOL by Anderson Symptom Assessment Scale (macimorelin N = 4; placebo N = 1; P = 1.00) or Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F; macimorelin N = 3; placebo N = 0; P = 0.50). No related serious or non-serious adverse events were reported. In macimorelin recipients, change in FACIT-F was directly associated with change in body weight (r = 0.92, P = 0.001), IGF-1 (r = 0.80, P = 0.01), and caloric intake (r = 0.83, P = 0.005), and inversely associated with change in energy expenditure (r = -0.67, P = 0.05). ConclusionsDaily oral macimorelin for 1 week was safe and numerically improved body weight and QOL in patients with cancer cachexia compared with placebo. Longer term administration should be evaluated for mitigation of cancer-induced reductions in body weight, appetite and QOL in larger studies.

Automated summary

This pilot study evaluated the safety and efficacy of using macimorelin, a growth hormone secretagogue, in cancer cachexia patients. The results showed that one-week administration of macimorelin improved body weight and quality of life in these patients. The importance of this study lies in providing a potential new approach for treating cancer cachexia.