Safinamide for Multiple System Atrophy (MSA)

NCT03753763 | Completed Phase 2 Results

• 2 other identifiers: Z7219K012018-004145-16
• Study Type: interventional
• Target: 49
• Locations: 2 countries
• Sites: 19

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

Conditions

Multiple System Atrophy

Keywords

MSA, safinamide

Outcome Measures

Primary Outcomes (1)

• TEAEs (Treatment Emergent Adverse Events) and SAEs (Serious Adverse Events)

  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.

  Throughout the study, from baseline (and at each interim visit) to telephone follow-up visit at 14 week.

Secondary Outcomes (7)

• 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 Unified Multiple System Atrophy Rating Scale (UMSARS), Part II (PP 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

Study Arms (2)

Active

EXPERIMENTAL

Safinamide methanesulfonate film-coated tablets once daily

Drug: Safinamide Methanesulfonate

Placebo

PLACEBO COMPARATOR

Safinamide Methanesulfonate matching placebo film-coated tablets once daily

Drug: Safinamide Methanesulfonate matching placebo

Interventions

Safinamide Methanesulfonate DRUG

100 mg (free base)

Also known as: Xadago

Active

Safinamide Methanesulfonate matching placebo DRUG

100 mg placebo

Also known as: placebo

Placebo

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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;
• Impaired ambulation, i.e. falling more than once per week, bedridden patients or confined to a wheelchair during the whole day;
• Subjects with active malignant neoplasms;
• Movement disorders other than MSA (e.g. Parkinson Disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, pharmacological or post-encephalic parkinsonism);
• 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;
• Not on a stable regime, for at least 4 weeks prior to the randomization (baseline visit), of

Sponsors & Collaborators

• Zambon SpA: lead

Study Sites (19)

Università di Bologna

Bologna, 40123, Italy

Location

AAST degli Spedali Civili di Brescia

Brescia, 25123, Italy

Location

San Raffaele Cassino

Cassino, 03043, Italy

Location

Fondazione Università "G. D'Annunzio"

Chieti, 66013, Italy

Location

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

Location

Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli

Napoli, 80138, Italy

Location

Azienda Ospedaliera di Padova

Padua, 35128, Italy

Location

Azienda Opsedaliero Universitaria Pisana

Pisa, 56126, Italy

Location

Istituto Clinico Humanitas

Rozzano, 20089, Italy

Location

Azienda Ospedaliera Universitaria OO.RR. San Giovanni di Dio - Ruggi d'Aragona

Salerno, 84131, Italy

Location

Azienda Ospedaliera Santa Maria di Terni

Terni, 05100, Italy

Location

Hospital General Universitario de Elche

Alicante, 03203, Spain

Location

Hospital de Cruces

Barakaldo, 48903, Spain

Location

Hospital de la Santa Creu i Sant Pau Barcelona

Barcelona, 08025, Spain

Location

Hospital Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Universitario Puerta de Hierro Majadahonda

Madrid, 28222, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28304, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Hospital Universitario Virgen de Rocio

Seville, 41013, Spain

Location

MeSH Terms

Conditions

Multiple System Atrophy; Thyroid Dyshormonogenesis 2A

Interventions

safinamide

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Results Point of Contact

• Title: Director of Clinical Trial - Charlotte Keywood, MD
• Organization: Zambon S.p.A.

charlotte.keywood@zambongroup.com

+39 02 665241

Study Officials

• Charlotte Keywood, MD

  Zambon SpA

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2018
• First Posted: November 27, 2018
• Study Start: October 29, 2019
• Primary Completion: January 5, 2021
• Study Completion: January 5, 2021
• Last Updated: October 13, 2021
• Results First Posted: August 24, 2021

Record last verified: 2021-07

Locations

IT (11); ES (8)