Effect of GTx-024, a selective androgen receptor modulator (SARM), on stair climb performance and quality of life (QOL) in patients with cancer cachexia.

Abstract

9147 Background: Cancer cachexia is a catabolic muscle wasting condition that leads to a reduction in lean body mass (LBM). The associated changes not only affect patient's health, but also QOL. We conducted a randomized, double-blind, placebo-controlled multi-center study to evaluate the effect of GTx-024, a SARM, on total LBM in patients with cancer cachexia. Methods: Subjects (n=159) were randomized to oral GTx-024 (3 mg or 1 mg) or placebo daily for 16 weeks. Subjects were males >45 years and postmenopausal females, had experienced ≥2% weight loss, had a BMI <35 kg/m2 and were diagnosed with either non-small cell lung cancer, colorectal cancer, non-Hodgkin's lymphoma, chronic lymphocytic leukemia or breast cancer. The primary endpoint was change in total LBM measured by dual energy X-ray absorptiometry (DXA). Secondary endpoints included muscle performance and QOL assessments. QOL questionnaires were analyzed to determine if there was a correlation between improvement in stair climb and QOL. Clinically meaningful differences were defined as 6 points between groups for FAACT-TOI and 3 points for FACIT-F TOI and the Fatigue sub-scale. Results: We observed a statistically significant increase in LBM in both treatment groups compared to baseline (1 mg, p=0.001; 3 mg, P=0.045). Ninety-five subjects were eligible for QOL analyses. Responders were defined as follows: stair climb time (SCT) >1 second decrease; stair climb power (SCP) >9.8 watt increase. SCT responders showed an 8.46 point improvement in FACIT-F TOI (p=0.020) and a 9.02 point increase in FAACT TOI (p=0.012). SCP responders showed a 9.96 point improvement in the FACIT-F TOI (p=0.006) and 10.75 point improvement in the FACCT TOI (p=0.002). In the Fatigue sub-scale, SCT and SCP responders demonstrated a change of 4.69 (p=0.028) and 5.44 points (p=0.016), respectively. Subjects with the greatest improvement in stair climb had the greatest improvement in QOL measures. Conclusions: GTx-024 improved LBM, muscle performance and QOL. These data suggest that GTx-024 not only provides benefit in muscle mass, but is also associated with clinically meaningful improvements in physical function and QOL. Author Disclosure Employment or Leadership Position Consultant or Advisory Role Stock Ownership Honoraria Research Funding Expert Testimony Other Remuneration GTx GTx