An Observational Study on Safinamide, Rasagiline and Other Standard of Care in PD

Study Overview
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Brief Summary
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The purpose of this study is to evaluate how safinamide, rasagiline and other SoC drugs are associated with the quality of life of PD patients by means of the Parkinson's Disease Questionnaire (PDQ)-39 items.

Detailed Description
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Safinamide is an alpha-aminoamide derivative, structurally unrelated to any other drug for the treatment of PD, with a dual mechanism of action (dopaminergic and non-dopaminergic). In particular, it is a potent, selective and reversible MAO-B inhibitor, and it is a glutamate modulator through the sodium channels blockade. Safinamide has been approved in Europe for the treatment of mid- to late-stage patients with idiopathic PD and fluctuations as add-on therapy to a stable dose of levodopa (alone or in combination with other PD medications). Rasagiline is an irreversible MAO-B inhibitor, with unknown activity on other neurotransmitters. Rasagiline has been approved in Europe for the treatment of idiopathic PD as monotherapy or as add-on to levodopa in patients with end of dose fluctuations. The aim of this observational study is to evaluate the effectiveness of safinamide, rasagiline and other standard of care (SoC) drugs when prescribed in clinical routine as add-on to L-dopa in terms of quality of life, improvement of chronic pain, change in Anti-Parkinson treatment (modification of doses, addition or withdrawal or other Anti-Parkinson drugs, etc.), use of concomitant pain-killer medications, compliance to the PD treatment, hospitalizations and use of other healthcare resources, and number of lost working days.

Official Title
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An Observational, Prospective, Multinational, Multicentre Study Comparing the Effectiveness of Safinamide, Rasagiline and Other Standard Of Care as Add-On Therapy to Levodopa (L-Dopa) in Parkinson's Disease (Pd) Fluctuating Patients

Conditions
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Parkinson's Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients of both genders ≥ 18 years of age, with a clinical diagnosis of idiopathic PD according to UK Brain Bank diagnostic criteria (12) for whom safinamide, rasagiline or any other anti-Parkinson drugs are prescribed according to the current Summary of Product Characteristics (SmPC). Willing to participate in the study and able to understand and sign the written informed consent form. Patients on a stable anti-Parkinson therapy, always including L-dopa + dopa-decarboxylase inhibitor (DDI), with or without other anti-Parkinson medications. Patients must be treated with safinamide, rasagiline or other SoC drugs as add-on to L-dopa for no more than 2 months prior to the baseline visit, according to the clinical practice. Exclusion Criteria: Patients with any form of Parkinsonism other than idiopathic PD. Patients for whom safinamide, rasagiline or any other anti-Parkinson drug are contraindicated according to the current SmPC. Patients known to be pregnant. Patients treated with safinamide or rasagiline who receive other concomitant MAO-B inhibitors. Patients treated with other SoC drugs who receive safinamide or rasagiline. Previous participation in a clinical trial with an investigational drug or medical device in the 3 months prior to the baseline visit.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Group 1<br>500 patients already receiving safinamide (50 or 100 mg/day) as add-on to L-dopa for no more than 2 months. | |
| Group 2<br>500 patients receiving rasagiline 1 mg/day as add-on to L-dopa for no more than 2 months. | |
| Group 3<br>235 patients receiving other SoC drugs as add-on to L-dopa for no more than 2 months. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The change from baseline to the end of study of the PDQ-39 total score. | Over a period of 12 months | The validated PDQ-39 assesses health-related quality improvement (Qi); an improvement in Qi corresponds to a decrease of the PDQ-39 total score. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PDQ-39 total score | The change from baseline to 6 months in the PDQ-39 total score. | 6 months | 
| PDQ-39 sub-scores (domains and single items) | The change from baseline to 6 months and to the end of study (12 months) in the PDQ-39 sub-scores (domains and single items) | 6 and 12 months | 
| UPDRS III score | The change from baseline to 6 months and to the end of study (12 months) in the UPDRS III score. | 6 and 12 months | 
| NRS. | The change from baseline to 6 months and to the end of study (12 months) in the NRS | 6 and 12 months | 
| anti-Parkinson drugs number | The change in anti-Parkinson drugs number from baseline to 6 months and to the end of the study (12 months). | 6 and 12 months | 
| new anti-Parkinson drugs | The introduction of new anti-Parkinson drugs, withdrawal, augmentation and decrease at 6 and 12 months, respectively. | 6 and 12 months | 
| The use of concomitant pain-killer medications | The use of concomitant pain-killer medications at 6 and 12 months, respectively. | 6 and 12 months | 
| number of pain-killer medications | The change in the number of pain-killer medications from baseline to 6 months and to the end of the study (12 months). | 6 and 12 months | 
| new pain-killer medications and daily dosage of pain-killer medications | The introduction of new pain-killer medications, withdrawal, augmentation, decrease and daily dosage of pain-killer medications at 6 and 12 months, respectively. | 6 and 12 months | 
| Healthcare resources | The use of healthcare resources from baseline to 6 months and to the end of study (12 months): number of and reason for hospitalizations, number of hospitalization days, number of visits to the emergency room, number of visits to PD specialists, number of diagnostic exams, number of rehabilitation visits. | 6 and 12 months | 
| number of working-days lost | The number of working-days lost from baseline to 6 months and to the end of study (12 months). | 6 and 12 months | 
| Safety Endpoints | The nature, frequency, severity, relationship (to study drug), actions taken, and outcome of adverse events (AEs) and serious adverse events (SAEs). | 6 and 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Parkinson's, PD, Safinamide, Rasagiline, Levodopa, L-DOPA, Fluctuating Patients, Add-on Therapy, PDQ, UPDRS, NRS, quality of life, observational