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Lookup NU author(s): Dr Rachael LawsonORCiD
This is the authors' accepted manuscript of an article that has been published in its final definitive form by Wiley, 2020.
For re-use rights please refer to the publisher's terms and conditions.
A quantitative assessment of Parkinson’s disease (PD) progression is critical for optimizing clinicaltrials design. Disease progression model was developed using pooled data from the ProgressionMarker Initiative study and the Incidence of Cognitive Impairment in Cohorts with LongitudinalEvaluation in Parkinson’s Disease study. Age, gender, concomitant medication, and study arms werepredictors of baseline. A mutation in the leucine-rich repeat kinase 2 (LRRK2) encoding gene wasassociated with the disease progression rate. The progression rate in subjects with PD who carriedLRRK2 mutation was slightly slower (~0.170 points/month) than that in PD subjects without themutation (~0.222 points/month). For a non-enriched placebo-controlled clinical trial, approximately70 subjects/arm would be required to detect a drug effect of 50% reduction in the progression ratewith 80% probability. Whereas, 85, 93 and 100 subjects/arm would be required for an enrichedclinical trial with 30%, 50% and 70% subjects with LRRK2 mutations, respectively.
Author(s): Ahamadi M, Conrado DJ, Macha S, Sinha V, Stone J, Burton J, Nicholas T, Gallagher J, Dexter D, Bani M, Boroojerdi B, Smit H, Weidemann J, Chen C, Yang M, Maciuca R, Lawson R, Burn D, Marek K, Venuto C, Stafford B, Akalu M, Stephenson D, Romero K
Publication type: Article
Publication status: Published
Journal: Clinical Pharmacology and Therapeutics
Print publication date: 01/03/2020
Online publication date: 23/09/2019
Acceptance date: 05/09/2019
Date deposited: 07/10/2019
ISSN (print): 0009-9236
ISSN (electronic): 1532-6535
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