Study TRX-005 was designed to assess the safety and efficacy of LMTX® at two doses in 800 patients diagnosed with mild Alzheimer’s disease. The main measures of efficacy (“primary outcomes”) used in the study were:
This 18-month study took place at 98 clinical sites in 12 countries. All 800 subjects were randomized but 1 site was removed from the study for compliance reasons, reducing the intent-to-treat population to 795 of which:
The modified intent-to-treat population (all patients taking the drug and having at least one post-baseline efficacy assessment) was 761 and the overall retention rate was 66%.
In light of the data from the Study 015 analysis suggesting that only those patients benefitted who received LMTX® as monotherapy, a decision was taken to revise the statistical analysis plan for Study 005 prior to database lock. The revised plan defined cohort analyses as the primary statistical outcomes permitting two comparisons in parallel (each at a critical p-value threshold of 0.025):
The top-line results, which were consistent with those seen in Study TRX-015, were presented at the CTAD meeting in San Diego in December. A paper containing the full study results is expected to be published in the first half of 2017.