Parkinsonian variant of Multiple System Atrophy MedDRA version: 20.0 Level: PT Classification code 10064060 Term: Multiple system atrophy System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Xadago
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: safinamide methanesulfonate
CAS Number: 133865-89-1
Other descriptive name: SAFINAMIDE
Concentration unit: mg milligram(
Sponsors
Zambon SpA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: Age 1. Participant must be 30 to 80 years of age inclusive, at the time of signing the informed consent. Type of Participant and Disease Characteristics 2. Participants who are diagnosed (with MRI confirmation) with possible or probable parkinsonian variant of Multiple System Atrophy less than 2 years ago. 3. Participants with an anticipated survival of at least 3 years in the opinion of the investigator. Sex 4. Male or female • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies (see Appendix 4): i. Not a woman of childbearing potential (WOCBP) OR ii. A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 30 days after the last dose of study intervention. Informed Consent 5. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 24 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: Medical Conditions 1. History of neurosurgical procedure, including stereotactic surgery. 2. History of Deep Brain Stimulation (DBS) 3. History of bipolar disorder, severe depression, schizophrenia or other psychotic disorder. 4. 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 5. History of dementia (DSM-V criteria) 6. 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. 7. Active hepatitis B or C 8. History of human immunodeficiency virus (HIV) infection 9. Subjects not able to swallow oral medications 10. Subjects with severe orthostatic symptoms 11. Impaired ambulation, i.e. falling more than once per week, bedridden patients or confined to a wheelchair during the whole day. 12. Subjects with active malignant neoplasms. 13. Movement disorders other than MSA (e.g. Parkinson Disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, pharmacological or post-encephalic parkinsonism). 14. Any clinically significant or unstable medical or surgical condition that, in the opinion of the investigator, might preclude safe completion of the study or might affect the results of the study. Prior/Concomitant Therapy 15. Not on a stable regime, for at least 4 weeks prior to the randomization (baseline visit), of a. oral levodopa (including controlled release [CR], immediate release [IR] or a combination of CR/IR), with or without benserazide/carbidopa, with or without addition of a catechol O-methyltransferase (COMT) inhibitor or b. dopamine agonist, anticholinergic and/or amantadine. Prior/Concurrent Clinical Study Experience 16. Patients should not have received treatment with monoamine oxidase inhibitors in the 2 weeks prior to the randomization visit, nor treatment with levodopa infusion, pethidine, opiates, opioids, fluoxetine, fluvoxamine in the 4 weeks prior to the randomization visit. 17. Patients should not have received treatment with an oral or depot neuroleptic within 12 weeks prior to the randomization visit. 18. Use of any investigational drug within 30 days prior to screening or 5 half-lives, whichever is the longest. Diagnostic assessments 19. Montreal Cognitive Assessment (MoCA) = 20 20. Laboratory assessments showing moderate or severe hepatic impairment (2x ULN) Other Exclusions 21. Allergy/sensitivity or contraindications to the investigational medicinal products (IMPs) or their excipients, anticonvulsants, levodopa or other anti-parkinsonian drugs. 22. Any clinically significant condition which, in the opinion of the Investigator, would not be compatible with study participation or represent a risk for patients while in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Outcome will be assessed after 12 weeks of treatment, when progression is unlikely to be a confounder of change.;Main Objective: To evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo;Secondary Objective: To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on motor function and/or quality of life;Primary end point(s): • The nature, frequency, severity, relationship (to study drug), actions taken, and outcome of TEAEs and SAEs. • Changes in physical and neurological examination findings. • Changes in vital sign (heart rate, systolic and diastolic blood pressure) values, including occurrence of abnormalities. • Changes in 12-lead ECG parameter measures, including occurrence of abnormalities. • Changes in clinical chemistry and hematology values, including shifts from Baseline and occurrence of abnormalities. • Number of withdrawals (and reason if given). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • The change from baseline to week 12 in the goniometric measurement for “lateral” displacement • Change from baseline at week 12 in Unified MSA Rating Scale, MSA Health-Related Quality of Life (MSA-QoL) scale, Montreal Cognitive Assessment (MoCA) scale, Unified Dystonia Rating Scale (UDRS).;Timepoint(s) of evaluation of this end point: Outcome will be assessed after 12 weeks of treatment, when progression is unlikely to be a confounder of change. | — |
Countries
Italy, Spain
Contacts
Public ContactClinical Development
Zambon SpA
Outcome results
None listed