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Table e-1. Subjects in MRI analyses (see Supplementary Figure e-2).
Low dose / Placebo / PN with serial scans / 62 / 62
Baseline MMSE / 21.2 ± 3.5 / 20.7 ± 3.5 / 0.42
Age (years) / 71.7 ± 13.2 / 70.8 ± 13.9 / 0.76
Annualized hippocampal volume changes (mL) / 248 ± 135 / 215 ± 183 / 0.48
Table e-2. CSF analyses of A-beta, Tau and P-tau181 (see Supplementary Figure e-3).
Mean changes in CSF biomarkers as % of baseline levelsHigh dose / Low dose / Placebo
A-beta 1-x / -12.0 ± 23.2 (12) / -17.6 ± 4.7 (19) / -22.3 ± 4.5 (21)
A-beta 1-40 / -24.4 ± 4.7 (11) / -25.3 ± 5.6 (19) / -25.5 ± 5.5 (21)
A-beta 1-42 / -5.7 ± 7.7 (11) / -6.1 ± 4.8 (15) / -2.6 ± 4.2 (18)
Tau / -4.2 ± 10.1 (11) / 3.2 ± 5.7 (15) / -4.2 ± 6.0 (19)
P-tau181 / -5.1 ± 9.2 (10) / -3.2 ± 7.8 (15) / -9.2 ± 5.5 (15)
Data are mean % change +/- standard error, with number of subjects (N) shown in parentheses. Some of the assays did not yield usable data on all CSF samples collected at 12 months, therefore the numbers of subjects in change analyses of Tau and P-tau differs slightly from those with A-beta data.
There were no significant differences in changes of any of these CSF biomarkers at 12 months between treatment groups (Wilcoxon test, P > 0.05 for all comparisons).
Table e-3a.Frequencies of adverse events, by treatment group (given as numbers of subjects).
P values are from Fisher’s exact tests.
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Types of disorders(MEDRA groupings) / Overall / High Dose / Low Dose / Placebo / P (Low dose vs placebo)
Nervous system / 225 / 66 / 76 / 83 / 0.451
Psychiatric / 212 / 62 / 53 / 97 / <0.001
Injury, poisoning and procedures / 177 / 74 / 57 / 46 / 0.207
Infections and infestations / 168 / 60 / 47 / 61 / 0.103
Gastrointestinal / 164 / 63 / 60 / 41 / 0.022
Musculoskeletal and connective tissue / 122 / 40 / 40 / 42 / 0.894
General / 119 / 39 / 47 / 33 / 0.081
Investigations / 101 / 28 / 40 / 33 / 0.409
Respiratory, thoracic and mediastinal / 77 / 21 / 30 / 26 / 0.652
Skin and subcutaneous tissue / 66 / 22 / 20 / 24 / 0.621
Renal and urinary / 49 / 14 / 18 / 17 / 1.000
Metabolism and nutrition / 40 / 13 / 16 / 11 / 0.417
Cardiac / 37 / 7 / 17 / 13 / 0.561
Surgical and medical procedures / 36 / 13 / 9 / 14 / 0.383
Vascular / 32 / 9 / 10 / 13 / 0.663
Eye / 22 / 7 / 5 / 10 / 0.287
Neoplasms, benign, malignant, unspecified / 22 / 10 / 8 / 4 / 0.376
Reproductive system and breast / 12 / 2 / 4 / 6 / 0.749
Ear and labyrinth / 9 / 4 / 4 / 1 / 0.370
Blood and lymphatic system / 8 / 3 / 3 / 2 / 1.000
Endocrine / 2 / 0 / 1 / 1 / 1.000
Hepatobiliary / 2 / 0 / 1 / 1 / 1.000
Social circumstances / 1 / 0 / 1 / 0 / 1.000
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Table e-3b. Number of subjects who experienced AE in preferred terms with p values < 0.1
P values are from Fisher’s exact tests.
Overall / High Dose / Low Dose / Placebo / P (Low vs placebo)Agitation / 23 / 7 / 4 / 12 / 0.068
Back pain / 29 / 8 / 15 / 6 / 0.067
Crying / 12 / 4 / 1 / 7 / 0.066
Insomnolence / 24 / 7 / 13 / 4 / 0.042