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Trial record 21 of 91 for:    "Brain Diseases" AND "Multiple System Atrophy"

Safinamide for Multiple System Atrophy (MSA)

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ClinicalTrials.gov Identifier: NCT03753763
Recruitment Status : Not yet recruiting
First Posted : November 27, 2018
Last Update Posted : June 12, 2019
Sponsor:
Information provided by (Responsible Party):
Zambon SpA

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

Condition or disease Intervention/treatment Phase
Multiple System Atrophy Drug: Safinamide Methanesulfonate Drug: Safinamide Methanesulfonate matching placebo Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Estimated Study Start Date : July 31, 2019
Estimated Primary Completion Date : March 31, 2020
Estimated Study Completion Date : March 31, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Safinamide

Arm Intervention/treatment
Experimental: Active
Safinamide methanesulfonate film-coated tablets once daily
Drug: Safinamide Methanesulfonate
100 mg (free base)

Placebo Comparator: Placebo
Safinamide Methanesulfonate matching placebo film-coated tablets once daily
Drug: Safinamide Methanesulfonate matching placebo
100 mg placebo




Primary Outcome Measures :
  1. TEAEs (Treatment Emergent Adverse Events) and SAEs (Serious Adverse Events) [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring nature, frequency, severity, relationship (to study drug), actions taken, and outcome of TEAEs and SAEs.

  2. Change in vital signs [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring the change in vital signs (heart rate, systolic and diastolic blood pressure) including occurrence of abnormalities

  3. Change in physical and neurological examination [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring the change in physical and neurological examination findings

  4. Change in ECG [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring the change in 12-lead ECG

  5. Change in laboratory values [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring the change in clinical chemistry and haemathology values

  6. Number of withdrawals [ Time Frame: 12 weeks ]
    Evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo monitoring the number of withdrawals from the study


Secondary Outcome Measures :
  1. Change in lateral displacement [ Time Frame: 12 weeks ]
    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on quality of life (change in lateral displacement). The goniometric measurement consists in the posture evaluation of the patient, measuring through a goniometer the angle of the flexion of the trunk.

  2. Change in Unified MSA Rating Scale [ Time Frame: 12 weeks ]

    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on quality of life (change from baseline in Unified MSA Rating Scale). UMSARS is a validated, disease-specific scale representing the diverse signs and symptoms in MSA. Higher scores on the UMSARS scales mean poorer health.

    UMSARS has an Activities of Daily Living score (UMSARS-1, 12 questions) that evaluates motor including autonomic activities and the Motor Examination score (UMSARS-2, 14 questions). UMSARS-3 measures supine/standing BP and UMSARS-4 is a disability scale.


  3. Change in MSA Health-Related Quality of Life (MSA-QoL) scale [ Time Frame: 12 weeks ]
    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on quality of life (change from baseline in MSA-QoL Scale). The MSA-QoL is a self-reported questionnaire focusing on MSA-specific symptoms and has a scale ranging from 0 - 160, with 0= 'no problem' and 160= "extreme problem".

  4. Change in Montreal Cognitive Assessment (MoCA) scale [ Time Frame: 12 weeks ]
    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on quality of life (change from baseline in MoCA Scale). The total score is out of 30 with higher scores indicating better cognitive functioning.

  5. Change in Unified Dystonia Rating Scale (UDRS) [ Time Frame: 12 weeks ]
    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on quality of life (change from baseline in UDRS). UDRS consists of a Historical Section, divided into questionnaires about 1) on-dyskinesia and 2) off -dystonia, and an Objective Section, divided into 3) impairment and 4) disability scales. The Historical Section is scored from 0-60, and the Objective section is scored 0-44, where higher scores reflect greater difficulty or impairment.



Information from the National Library of Medicine

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Ages Eligible for Study:   30 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03753763


Contacts
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Contact: Elena Tiberio +39 02 6652 ext 4602 elena.tiberio@zambongroup.com

Locations
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Italy
Università di Bologna Not yet recruiting
Bologna, Italy, 40123
AAST degli Spedali Civili di Brescia Not yet recruiting
Brescia, Italy, 25123
San Raffaele Cassino Not yet recruiting
Cassino, Italy, 03043
Fondazione Università "G. D'Annunzio" Not yet recruiting
Chieti, Italy, 66013
Fondazione IRCCS Istituto Neurologico Carlo Besta Not yet recruiting
Milano, Italy, 20133
Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli
Napoli, Italy, 80138
Azienda Ospedaliera di Padova Not yet recruiting
Padova, Italy, 35128
Azienda Opsedaliero Universitaria Pisana Not yet recruiting
Pisa, Italy, 56126
Istituto Clinico Humanitas Not yet recruiting
Rozzano, Italy, 20089
Azienda Ospedaliera Universitaria OO.RR. San Giovanni di Dio - Ruggi d'Aragona
Salerno, Italy, 84131
Azienda Ospedaliera Santa Maria di Terni Not yet recruiting
Terni, Italy, 05100
Spain
Hospital General Universitario de Elche Not yet recruiting
Alicante, Spain, 03203
Hospital de Cruces Not yet recruiting
Barakaldo, Spain, 48903
Hospital de la Santa Creu i Sant Pau Barcelona Not yet recruiting
Barcelona, Spain, 08025
Hospital Universitario Gregorio Maranon Not yet recruiting
Madrid, Spain, 28007
Hospital Universitario Puerta de Hierro Majadahonda Not yet recruiting
Madrid, Spain, 28222
Hospital Universitario Ramon y Cajal Not yet recruiting
Madrid, Spain, 28304
Hospital Universitario Virgen Macarena Not yet recruiting
Sevilla, Spain, 41009
Hospital Universitario Virgen de Rocio Not yet recruiting
Sevilla, Spain, 41013
Sponsors and Collaborators
Zambon SpA
Investigators
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Study Director: Charlotte Keywood, MD Zambon SpA

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Responsible Party: Zambon SpA
ClinicalTrials.gov Identifier: NCT03753763     History of Changes
Other Study ID Numbers: Z7219K01
First Posted: November 27, 2018    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: November 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Multiple System Atrophy
Shy-Drager Syndrome
Brain Diseases
Atrophy
Pathological Conditions, Anatomical
Primary Dysautonomias
Autonomic Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
Central Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Hypotension
Vascular Diseases
Cardiovascular Diseases