Safinamide for Multiple System Atrophy (MSA)

• Register: ClinicalTrials.gov
• Last refreshed on: 18 October 2021
• Main ID: NCT03753763
• Date of registration: 19/11/2018
• Prospective Registration: Yes
• Primary sponsor: Zambon SpA
• Public title: Safinamide for Multiple System Atrophy (MSA)
• Scientific title: 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
• Date of first enrolment: October 29, 2019
• Target sample size: 49
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03753763
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Italy, Spain

Contacts

• Name: Charlotte Keywood, MD
• Affiliation: Zambon SpA

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Participant must be 30 to 80 years of age inclusive, at the time of signing the
informed consent;

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;

4. 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;

5. Capable of giving signed informed consent

Exclusion Criteria:

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;

15. Not on a stable regime, for at least 4 weeks prior to the randomization (baseline
visit), of

1. oral levodopa (including controlled release, immediate release or a combination
of controlled release/immediate release), with or without benserazide/carbidopa,
with or without addition of a catechol O-methyltransferase (COMT) inhibitor or

2. dopamine agonist, anticholinergic and/or amantadine.

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;

19. Montreal Cognitive Assessment (MoCA) = 20;

20. Laboratory assessments showing moderate or severe hepatic impairment (2x ULN);

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.



Age minimum: 30 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple System Atrophy

Intervention(s)

Drug: Safinamide Methanesulfonate

Drug: Safinamide Methanesulfonate matching placebo

Primary Outcome(s)

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.]

Secondary Outcome(s)

Change From Baseline to Week 12 in Multiple System Atrophy Health-Related Quality of Life (MSA-QoL) Scale [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in Montreal Cognitive Assessment (MoCA) Scale [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in Unified Dystonia Rating Scale (UDRS) [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in Unified Multiple System Atrophy Rating Scale (UMSARS), Part II (ITT Population) [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in the Goniometric Measurement for Anterior Displacement [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in the Goniometric Measurement for Lateral Displacement [Time Frame: From baseline to week 12]

Change From Baseline to Week 12 in Unified Multiple System Atrophy Rating Scale (UMSARS), Part II (PP Population) [Time Frame: From baseline to week 12]

Secondary ID(s)

2018-004145-16

Z7219K01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/08/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03753763