Rationale and study design of a randomized, placebo-controlled, double-blind phase 2b trial to evaluate efficacy, safety, and tolerability of an oral glutaminyl cyclase inhibitor varoglutamstat (PQ912) in study participants with MCI and mild AD—VIVIAD
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Rationale and study design of a randomized, placebo-controlled, double-blind phase 2b trial to evaluate efficacy, safety, and tolerability of an oral glutaminyl cyclase inhibitor varoglutamstat (PQ912) in study participants with MCI and mild AD—VIVIAD. / Vijverberg, E. G.B.; Axelsen, T. M.; Bihlet, A. R.; Henriksen, K.; Weber, F.; Fuchs, K.; Harrison, J. E.; Kühn-Wache, K.; Alexandersen, P.; Prins, N. D.; Scheltens, Philip.
I: Alzheimer's Research and Therapy, Bind 13, Nr. 1, 142, 2021.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - Rationale and study design of a randomized, placebo-controlled, double-blind phase 2b trial to evaluate efficacy, safety, and tolerability of an oral glutaminyl cyclase inhibitor varoglutamstat (PQ912) in study participants with MCI and mild AD—VIVIAD
AU - Vijverberg, E. G.B.
AU - Axelsen, T. M.
AU - Bihlet, A. R.
AU - Henriksen, K.
AU - Weber, F.
AU - Fuchs, K.
AU - Harrison, J. E.
AU - Kühn-Wache, K.
AU - Alexandersen, P.
AU - Prins, N. D.
AU - Scheltens, Philip
N1 - Publisher Copyright: © 2021, The Author(s).
PY - 2021
Y1 - 2021
N2 - Background: Varoglutamstat (formerly PQ912) is a small molecule that inhibits the activity of the glutaminyl cyclase to reduce the level of pyroglutamate-A-beta (pGluAB42). Recent studies confirm that pGluAB42 is a particular amyloid form that is highly synaptotoxic and plays a significant role in the development of AD. Methods: This paper describes the design and methodology behind the phase 2b VIVIAD-trial in AD. The aim of this study is to evaluate varoglutamstat in a state-of-the-art designed, placebo-controlled, double-blind, randomized clinical trial for safety and tolerability, efficacy on cognition, and effects on brain activity and AD biomarkers. In addition to its main purpose, the trial will explore potential associations between novel and established biomarkers and their individual and composite relation to disease characteristics. Results: To be expected early 2023 Conclusion: This state of the art phase 2b study will yield important results for the field with respect to trial methodology and for the treatment of AD with a small molecule directed against pyroglutamate-A-beta. Trial registration: ClinicalTrials.gov Identifier: NCT04498650.
AB - Background: Varoglutamstat (formerly PQ912) is a small molecule that inhibits the activity of the glutaminyl cyclase to reduce the level of pyroglutamate-A-beta (pGluAB42). Recent studies confirm that pGluAB42 is a particular amyloid form that is highly synaptotoxic and plays a significant role in the development of AD. Methods: This paper describes the design and methodology behind the phase 2b VIVIAD-trial in AD. The aim of this study is to evaluate varoglutamstat in a state-of-the-art designed, placebo-controlled, double-blind, randomized clinical trial for safety and tolerability, efficacy on cognition, and effects on brain activity and AD biomarkers. In addition to its main purpose, the trial will explore potential associations between novel and established biomarkers and their individual and composite relation to disease characteristics. Results: To be expected early 2023 Conclusion: This state of the art phase 2b study will yield important results for the field with respect to trial methodology and for the treatment of AD with a small molecule directed against pyroglutamate-A-beta. Trial registration: ClinicalTrials.gov Identifier: NCT04498650.
KW - Abeta
KW - Alzheimer
KW - Amyloid
KW - Clinical trials; Small molecules; Puroglutamate
KW - Placebo
U2 - 10.1186/s13195-021-00882-9
DO - 10.1186/s13195-021-00882-9
M3 - Journal article
C2 - 34425883
AN - SCOPUS:85113804626
VL - 13
JO - Alzheimer's Research and Therapy
JF - Alzheimer's Research and Therapy
SN - 1758-9193
IS - 1
M1 - 142
ER -
ID: 281706233