12-weeks, Multicentre, Randomized, Double-blind, Placebo-controlled, Exploratory, Pilot Study to Evaluate the Safety and Efficacy of Safinamide 200 mg OD, as add-on Therapy, in Patients With Possible or Probable Parkinsonian Variant of MSA
Overview
- Phase
- Phase 2
- Intervention
- Safinamide Methanesulfonate
- Conditions
- Multiple System Atrophy
- Sponsor
- Zambon SpA
- Enrollment
- 49
- Locations
- 19
- Primary Endpoint
- TEAEs (Treatment Emergent Adverse Events) and SAEs (Serious Adverse Events)
- Status
- Completed
- Last Updated
- 4 years ago
Overview
Brief Summary
The study is a placebo controlled study, with two parallel arms, in which participants will be randomly assigned in a 2:1 ratio to receive either active (200 mg safinamide) or placebo in a double blind manner. Study population is patients diagnosed, with possible or probable parkinsonian variant of Multiple System Atrophy who are on a stable treatment of levodopa
Detailed Description
The overall design is a parallel group, placebo controlled, double blind study. The target population are participants diagnosed with possible or probable parkinsonian variant of Multiple System Atrophy who are on stable doses of levodopa. Trial participation will be up to a maximum duration of 14 weeks and will comprise a screening period (up to 2 weeks), a 2-week run in period during which subjects will receive 1 tablet (either 100 mg safinamide or matching placebo), followed by a 10-week period, during which study participants will take 2 tablets of study medication (200 mg safinamide or placebo) once daily, taken in the morning in addition to their morning levodopa dose. A telephone follow-up call will be performed 2 weeks after the end of treatment.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Participant must be 30 to 80 years of age inclusive, at the time of signing the informed consent;
- •Participants who are diagnosed (with MRI confirmation) with possible or probable parkinsonian variant of Multiple System Atrophy less than 2 years ago;
- •Participants with an anticipated survival of at least 3 years in the opinion of the investigator;
- •Female not pregnant, not breastfeeding, and at least one of the following conditions applies:
- •Not a woman of childbearing potential OR
- •A woman of childbearing potential who agrees to follow the contraceptive guidance during the treatment period and for at least 30 days after the last dose of study intervention;
- •Capable of giving signed informed consent
Exclusion Criteria
- •History of neurosurgical procedure, including stereotactic surgery;
- •History of Deep Brain Stimulation (DBS);
- •History of bipolar disorder, severe depression, schizophrenia or other psychotic disorder;
- •History of drug and/or alcohol abuse within 12 months prior to screening as defined by the current edition of the Diagnostic and Statistical Manual of Mental Disorders;
- •History of dementia (DSM-V criteria);
- •Ophthalmologic history including any of the following conditions: albinism, uveitis, retinitis pigmentosa, retinal degeneration, active retinopathy, severe progressive diabetic retinopathy, inherited retinopathy or family history of hereditary retinal disease;
- •Active hepatitis B or C;
- •History of human immunodeficiency virus (HIV) infection;
- •Subjects not able to swallow oral medications;
- •Subjects with severe orthostatic symptoms;
Arms & Interventions
Active
Safinamide methanesulfonate film-coated tablets once daily
Intervention: Safinamide Methanesulfonate
Placebo
Safinamide Methanesulfonate matching placebo film-coated tablets once daily
Intervention: Safinamide Methanesulfonate matching placebo
Outcomes
Primary Outcomes
TEAEs (Treatment Emergent Adverse Events) and SAEs (Serious Adverse Events)
Time Frame: Throughout the study, from baseline (and at each interim visit) to telephone follow-up visit at 14 week.
While evaluating safety and tolerability of safinamide, 200 mg od, compared with placebo, severity of TEAEs, their relationship to study drug, their seriousness and their consequences were assessed. TEAEs were defined as adverse events (AEs) that started after the first dose of study drug.
Secondary Outcomes
- Change From Baseline to Week 12 in Unified Multiple System Atrophy Rating Scale (UMSARS), Part II (PP Population)(From baseline to week 12)
- Change From Baseline to Week 12 in Montreal Cognitive Assessment (MoCA) Scale(From baseline to week 12)
- Change From Baseline to Week 12 in the Goniometric Measurement for Anterior Displacement(From baseline to week 12)
- Change From Baseline to Week 12 in the Goniometric Measurement for Lateral Displacement(From baseline to week 12)
- Change From Baseline to Week 12 in Unified Multiple System Atrophy Rating Scale (UMSARS), Part II (ITT Population)(From baseline to week 12)
- Change From Baseline to Week 12 in Multiple System Atrophy Health-Related Quality of Life (MSA-QoL) Scale(From baseline to week 12)
- Change From Baseline to Week 12 in Unified Dystonia Rating Scale (UDRS)(From baseline to week 12)