A study evaluating the safety and efficacy of safinamide for patients with Multiple System Atrophy

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 49

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

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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