Phase 3 Study Update for AEZS-130 for Diagnosis of Adult Growth Hormone Deficiency
Aeterna Zentaris announced that Phase 3 results for AEZS-130 show that it has promise as a safe and simple oral diagnostic test for adult growth hormone deficiency (AGHD), with accuracy comparable to available testing procedures.
This multicenter open-label study was originally designed as a cross-over trial of AEZS-130 vs growth hormone-releasing hormone (GHRH)+L-Arginine (ARG) in AGHD patients and in controls matched for Body Mass Index (BMI), estrogen status, gender and age. After 43 AGHD patients and 10 controls had been tested, GHRH became unavailable. The study was completed by testing 10 more AGHD patients and 38 controls with AEZS-130 alone. Of the 53 AGHD subjects enrolled, 52 received AEZS-130, and 50 who had confirmed AGHD prior to study entry were included in this analysis, along with 48 controls. Two AGHD subjects could not be matched due to the combination of young age, high BMI and estrogen use. The objective of this clinical trial was to determine the efficacy and safety of AEZS-130 in the diagnosis of AGHD.
Mean peak growth hormone (GH) levels in AGHD patients and controls following AEZS-130 administration were 2.36ng/mL (range 0.03–33) and 17.71ng/mL (range 10.5–94), respectively. The receiver operating characteristic (ROC) plot analysis yielded an optimal GH cut-point of 2.7ng/mL, with 82% sensitivity, 92% specificity and a 13% misclassification rate. Obesity (BMI>30) was present in 58% of cases and controls, and peak GH levels were inversely associated with BMI in controls.
Responses in normal subjects classified as obese, with BMI's above 30, were significantly lower than in leaner subjects. Since GH deficiency can lead to increased body fat, many of the patients also met criteria for obesity, and therefore, a lower peak GH cutoff is more accurate in separating obese normals from obese patients. Based upon these study results, a cut-off of 2.7μg/L was optimal for subjects with a BMI ≥30 and a cutoff of 6.8μg/L for subjects with a BMI of <30. Age had a much weaker effect on test performance and gender made no difference. Thus GH stimulation with oral AEZS-130 may provide a simple, rapid, safe, and well-tolerated diagnostic test for AGHD, with accuracy comparable to that of the GHRH-ARG test.
AEZS-130 is an orally-active, ghrelin agonist that stimulates the secretion of growth hormone.
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