Objective: Safety results from a phase 3, double-blind study of CVT-301 in patients with PD were combined with results from a 4-week, phase 2b, double-blind, placebo-controlled study and a 12-month safety study compared to an observational cohort (OC) control.

Background: CVT-301 is approved for the treatment of OFF episodes in PD patients on an oral carbidopa/levodopa regimen. In a phase 3, double-blind study, CVT-301 84 mg improved motor function in PD patients with OFF periods within 30 minutes as measured at 12 weeks by change in Unified Parkinson’s Disease Rating Scale Part III scores. More patients achieved and maintained an ON state within 60 minutes postdose vs placebo. CVT-301 was generally well tolerated.

Method: Patients were receiving oral dopa-decarboxylase inhibitor/levodopa, experiencing >2 hours/day of OFF periods and randomized to CVT-301 (60 mg or 84 mg; only the 84 mg is included in this analysis) or placebo or OC (no study drug administered), to be taken up to 5 times/day when subjects experienced return of OFF symptoms. Safety measurements included treatment-emergent adverse events (TEAEs), and serious AEs (SAEs) and monitoring special-interest AEs. Risk differences (RD) were compared between treatments by meta-analysis.

Results: The combined safety population was 709 patients. Combined study TEAEs were higher in CVT-301 but were not significantly different vs placebo or OC (RD 0.074, 95% CI -0.058, 0.206). SAEs and AEs leading to discontinuation showed no significant difference between groups (RD 0.003, 95% CI -0.031,0.038 [SAE]; RD 0.031, 95% CI -0.035,0.098 [discontinuation]). Severe and drug-related TEAEs were higher in the CVT-301 group, but differences were not significant (RD 0.021, 95% CI -0.027,0.069 [severe]; 0.185 95% CI -0.038, 0.409 [drug-related]). Respiratory system AEs were higher in CVT-301, but not significant (RD 0.073, 95% CI -0.065,0.211). Additional data will be presented.

Conclusion: Overall, TEAEs, SAEs and AEs leading to discontinuation were not significantly different in frequency between CVT-301 and placebo/OC groups, demonstrating that CVT-301 is generally safe and well tolerated.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/cvt-301-levodopa-inhalation-powder-meta-analysis-of-safety-in-patients-with-parkinsons-disease-pd/