Ahamadi M

Akalu M

Bani M

Boroojerdi B

Burn D

Burton J

Chen C

Conrado DJ

Dexter D

Gallagher J

Lawson R

Macha S

Maciuca R

Marek K

Nicholas T

Romero K

Sinha V

Smit H

Stafford B

Stephenson D

Stone J

Venuto C

Weidemann J

Yang M

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

Newcastle University E-Prints

Development of a disease progression model for leucine-rich repeat kinase 2 in Parkinson\u27s disease to inform clinical trial designs

Development of a disease progression model for leucine-rich repeat kinase 2 in Parkinson\u27s disease to inform clinical trial designs

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