The three main cohorts in the trial received a single weekly dose of hGH-CTP for a period of four weeks, containing 30%, 45% or 100% of the equivalent cumulative commercial hGH dose these patients would usually inject each day over the course of seven days (referred to as the “30%,” “45%” and “100%” cohorts, respectively.) The top-line data reflect results from 39 patients, with 13 patients in each cohort comprised of 11 males and two females.
Efficacy for the three main cohorts receiving a single weekly injection of hGH-CTP was defined by measuring daily insulin-like growth factor 1 (IGF-1) levels within the desired therapeutic range over a period of seven days during the last week of treatment in the study. The desired therapeutic range is defined as an IGF-1 level that is between +2 standard deviations through -2 standard deviations from the average IGF-1 levels expected in a normal population stratified by age group and gender. In addition, the trial measured IGF-1 levels within a narrower range of +/- 1.5 standard deviations from the average normal population IGF-1 levels, for the purpose of learning more about the variance of patients within the normal range.
Phase II Trial Top-Line Data Analysis
As shown in the table below, the study data show that patients in all three cohorts (the “30%,” “45%” and “100%” cohorts) achieved average IGF-1 levels that were within the normal range on 100% of the days when they were assessed. In addition, the data show that patients in each of the cohorts achieved average IGF-1 levels within the narrow definition of the normal range on many or most of the days when they were assessed, indicating a favorable variance profile within the normal range.
hGH-CTP demonstrated excellent safety and tolerability in all patients across all trial cohorts, with no apparent issues. In addition, there were no indications that hGH-CTP can induce excessive levels of IGF-1 in patients above the normal range when used in high doses.
|
Cohort |
% Days Within Normal Range (+/- 2 SD) |
Avg. Cmax (preferred below +2 SD) |
Variance Measure: % Days Within Narrow Normal Range
(+/-1.5 SD) |
|
30% |
100% |
-0.9 |
57% |
|
45% |
100% |
0.1 |
100% |
|
100% |
100% |
0.4 |
86% |
· The table shows the average percent of days within the normal therapeutic range (+/- 2 SD), average percent of days within a narrower normal therapeutic range (+/- 1.5 SD), and average Cmax (highest concentration level) of IGF-1 for males, measured during the last treatment week, expressed in standard deviations from the mean IGF-1 levels expected in the normal population.