Patients’ Experience of Parkinson’s Disease Following Treatment with Inhaled Levodopa: Results from a Phase 2b Study (P5.351)

Objective: To report patient experiences from a clinical trial regarding Parkinson’s disease (PD) control following treatment with inhaled levodopa. Background: CVT-301, an inhaled levodopa powder formulation, is being investigated for treatment of OFF episodes in patients with PD. Data from a phase 2b study of CVT-301 showed improvement in motor function and in daily OFF time. Patient Global Impression of Change (PGI-C), a patient-reported outcome, was included in this study. Design/Methods: This was a randomized, double-blind, placebo-controlled study in patients with PD experiencing >2hours/day OFF time. CVT-301 or placebo was used as an adjunct to usual PD medications for OFF symptoms for 4 weeks. Two doses were studied (35 mg [dosing level 1 (DL1), weeks 1-2]; 50 mg [dosing level 2 (DL2), weeks 3-4]). PGI-C ratings were measured at DL1 (end of week 2) and DL2 (end of week 4) to assess the effect of CVT-301 on patient impression of Parkinsonian control. PGI-C ratings were compared between placebo and CVT-301 using chi-square test. PGI-C ratings per baseline demographics and clinical characteristics were explored. Safety and tolerability were assessed. Results: Eighty-six patients with PD were randomized 1:1 to CVT-301 or placebo. Seventy-nine and 74 patients completed the PGI-C at DL1 and DL2, respectively. The majority of patients reported an improved impression of Parkinsonian control with CVT-301 (DL1:65.0[percnt]; DL2:71.8[percnt]), whereas less than half reported improvements with placebo (DL1:43.6[percnt]; DL2:45.7[percnt]). Compared with placebo, treatment with CVT-301 was associated with a significantly favorable impression of PD at DL2 (71.8[percnt]vs45.7[percnt], P=0.0225) and a directional favorable impression (65.0[percnt]vs43.6[percnt], P=0.0561) at DL1. Baseline demographic and clinical characteristics did not impact PGI-C ratings. CVT-301 was generally tolerated during the 4-week trial. Conclusions: Most patients reported an improved PGI-C rating with CVT-301, an inhaled levodopa, supportive of the positive findings in motor function and daily OFF time. Disclosure: Dr. LeWitt has received personal compensation for activities with Acorda Pharmaceuticals. Dr. Niyazov has received personal compensation for activities with Acorda Therapeutics, Inc. Dr. Sedkov has received personal compensation for activities with Acorda Therapeutics, Inc. Dr. Murck has received personal compensation for activities with Acorda Therapeutics, Inc. Dr. Guo has received personal compensation for activities with Acorda Therapeutics as an employee.