TY - JOUR AU - Scheltens, Philip AU - Hallikainen, Merja AU - Grimmer, Timo AU - Duning, Thomas AU - Gouw, Alida A. AU - Teunissen, Charlotte E. AU - Wink, Alle Meije AU - Maruff, Paul AU - Harrison, John AU - van Baal, Caroline M. AU - Bruins, Suzanne AU - Lues, Inge AU - Prins, Niels D. PY - 2018 DA - 2018/10/12 TI - Safety, tolerability and efficacy of the glutaminyl cyclase inhibitor PQ912 in Alzheimer’s disease: results of a randomized, double-blind, placebo-controlled phase 2a study JO - Alzheimer's Research & Therapy SP - 107 VL - 10 IS - 1 AB - PQ912 is an inhibitor of the glutaminyl cyclase enzyme that plays a central role in the formation of synaptotoxic pyroglutamate-A-beta oligomers. We report on the first clinical study with PQ912 in subjects with biomarker-proven Alzheimer’s disease (AD). The aim was to determine the maximal tolerated dose, target occupancy and treatment-related pharmacodynamic effects. The exploratory efficacy readouts selected were tailored to the patient population with early AD. The therapeutic approach focuses on synaptic dysfunction as captured by various measures such as electroencephalography (EEG), synaptic biomarkers and sensitive cognitive tests. SN - 1758-9193 UR - https://doi.org/10.1186/s13195-018-0431-6 DO - 10.1186/s13195-018-0431-6 ID - Scheltens2018 ER -