Multiple System AtrophyPhase 3MASCOTNCT06706622
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)
A Phase 3 study testing Amlenetug in people with Multiple System Atrophy, run by H. Lundbeck A/S at 74 sites in 10 countries.
Trial summary
The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA). Read more about this study
- Who is it for?
- Ages 40 to 75 years
- Conditions the study is for
- Multiple System Atrophy
- What is being tested?
- Amlenetug
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 401 participants
- Where
- 74 sitesin 10 countries
- Study dates
- 3 Dec 2024to 25 Oct 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The participant has a diagnosis of clinically established multiple system atrophy parkinsonian type (MSA-P) or multiple system atrophy cerebellar type (MSA-C), or clinically probable MSA-P or MSA-C, according to the 2022 Movement Disorders Society (MDS) criteria for the diagnosis of MSA at the Screening Visit.
- The participant had onset of motor MSA symptoms (that is, parkinsonian and/or cerebellar) within 5 years prior to the Screening Visit in the judgement of the investigator.
- The participant has an anticipated survival of >3 years, in the opinion of the investigator, at the Screening Visit.
- The participant has suitable peripheral venous access for investigational medicinal product (IMP) administration and blood sampling.
- The participant has an UMSARS Part I score ≤16 (omitting item 11 on sexual function) at the Screening Visit.
Who cannot join
- The participant has previously been dosed with amlenetug.
- The participant has taken any active IMP within 3 months or 5 half lives of that product, whichever is longer, prior to the first dose of IMP.
- The participant has 2 or more first degree relatives with a history of MSA.
- The participant, if of MSA-P subtype, has unexplained anosmia (not explained by other common causes such as allergic rhinitis or smoking, nasal structural lesions, or nasal surgery) on olfactory testing at the Screening Visit.
- The participant has evidence (clinically or on magnetic resonance imaging (MRI)) and/or history of any clinically significant disease or condition other than MSA, that is, in the investigator's opinion, likely to affect CNS functioning, e.g., serious neurological disorder, other intracranial or systemic disease.
- The participant has a current diagnosis of movement disorders that could mimic MSA, e.g., Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, pharmacological, or post-encephalitic parkinsonism, per investigator discretion. Participants who have previously been incorrectly diagnosed with Parkinson's disease will not be excluded.
- Other protocol-defined inclusion and exclusion criteria apply.
Trial locations and contacts
74 sites in 10 countries.
74 sites in 10 countries
United States23 sites
Johns Hopkins University School of Medicine - The Johns Hopkins Multiple Sclerosis (MS) Center
Baltimore, Maryland, 21287-0005
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486-2359
Brigham and Women's Hospital (BWH)
Boston, Massachusetts, 02115
Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC) - West Campus
Boston, Massachusetts, 02215-5321
Cleveland Clinic - Neurological Institute - Cerebrovascular Center
Cleveland, Ohio, 44195-0001
University of Texas Southwestern Medical Center (UT Southwestern Medical Center)
Dallas, Texas, 75390-7208
CenExel CBH - CenExel Rocky Mountain Clinical Research (CenExel RMCR) (Rocky Mountain Movement Di...
Englewood, Colorado, 80113-2776
QUEST Research Institute
Farmington, Michigan, 48334-2980
University of Florida-College of Medicine - Gainesville
Gainesville, Florida, 32610-3003
Baylor College of Medicine (BCM)
Houston, Texas, 77030-3411
Indiana University (IU) Health-Senior Health Center
Indianapolis, Indiana, 46202-1476
University of Arkansas for Medical Sciences (UAMS) - Medical Center
Little Rock, Arkansas, 72205-7101
Vanderbilt University Medical Center (VUMC) - Vanderbilt Orthopaedic Institute-Nashville
Nashville, Tennessee, 37232-0001
Yale New Haven Health System
New Haven, Connecticut, 06519-1304
NYU Langone Health (NYU Langone Medical Center)
New York, New York, 10016
Columbia University Irving Medical Center - The Neurological Institute of New York
New York, New York, 10032-3726
University of Nebraska Medical Center (UNMC) - The Lied Transplant Center
Omaha, Nebraska, 68198-3285
University of Pennsylvania - Penn Comprehensive Neuroscience Center
Philadelphia, Pennsylvania, 19107-6103
Mayo Clinic - Vascular Center - Arizona
Phoenix, Arizona, 85054-4502
University of Pittsburgh Medical Center-Magee-Womens Hospital (MWH)
Pittsburgh, Pennsylvania, 15213-3108
Mayo Clinic - Children's Center - Rochester
Rochester, Minnesota, 55905-0001
University of California San Francisco - Sandler Neurosciences Center
San Francisco, California, 94158-0003
Inland Northwest Research (INWR)
Spokane, Washington, 99202-1342
Japan11 sites
Juntendo University-Juntendo University Hospital
Bunkyō City, Tokyo-To, 113-8431
Gifu University Hospital
Gifu, Gifu, 501-1194
Saitama Medical University (SMU) - Moro Hospital
Iruma-shi, Saitama, 350-0495
National Center of Neurology and Psychiatry-National Center Hospital
Kodaira-shi, Tokyo-To, 187-8551
National Hospital Organization Higashinagoya National Hospital
Nagoya, Aichi-ken, 465-8620
Niigata University Medical and Dental Hospital
Niigata, Niigata, 951-8510
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
National Hospital Organization Sendai Nishitaga National Hospital
Sendai, Miyagi, 982-8555
Keio University Hospital
Shinjuku-Ku, Tokyo-To, 160-8582
Fujita Health University Hospital
Toyoake-Shi, Aichi-ken, 470-1192
Tottori University Hospital
Yonago-shi, Tottori, 683-8504
France7 sites
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin - Tripode
Bordeaux, New Aquitaine, 33000
Centre Hospitalier Universitaire de Lille (CHU Lille) - Centre Regional de Pharmacovigilance (CRPV)
Lille, Hauts-de-France, 59045
AP-HM- Hôpital de La Timone
Marseille, 13005
Centre Hospitalier Universitaire de Montpellier (CHU Montpellier)
Montpellier, Occitanie, 34295
CHU de la Pitié-Salpêtrière
Paris, 75651
Centre Hospitalier Universitaire de Rennes-Hopital Pontchaillou
Rennes, Brittany Region, 35033
Centre Hospitalier Universitaire de Toulouse-Hopital Purpan
Toulouse, Occitanie, 31300
Germany6 sites
Movement Disorders Clinic Beelitz-Heilstätten
Beelitz-Heilstätten, 14547
Universitaetsklinikum Erlangen - Psychiatrische und Psychotherapeutische Klinik
Erlangen, Bavaria, 91054
Curiositas Ad Sanum - Haag In Oberbayern
Haag I.Ob, Bavaria, 83527
Ludwig-Maximilians-Universitaet Muenchen (LMU) - Klinik und Poliklinik fuer Frauenheilkunde und G...
Munich, Bavaria, 81377
Universitaetsklinikum Muenster (UKM) - Medizinische Klinik A
Münster, North Rhine-Westphalia, 48149
Universitaetsklinikum Tuebingen - Universitaetsklinik fuer Psychiatrie und Psychotherapie
Tübingen, Baden-Wurttemberg, 72076
Italy6 sites
Universita di Bologna - IRCCS Istituto delle Scienze Neurologiche di Bologna
Bologna, 40123
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Centro Dino Ferrari
Milan, 20122
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, 20133
Universita degli Studi della Campania "Luigi Vanvitelli" - Centro Cefalee
Naples, 80138
Azienda Ospedaliero Universitaria Pisana - Stabilimento Ospedaliero di Santa Chiara - Istituto To...
Pisa, 56126
Azienda Ospedaliera Universitaria OO.RR. San Giovanni di Dio e Ruggi d'Aragona - Scuola Medica Sa...
Salerno, 84131
Spain6 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Clinic de Barcelona (Hospital Clinic i Provincial) - Institut Clinic de Neurociencies (ICN)
Barcelona, 08036
Hospital de la Santa Creu I Sant Pau - Institut de Recerca de l'Hospital de la Santa Creu i Sant ...
Barcelona, 08041
Universidad Autonoma de Madrid (UAM) - Hospital Universitario de La Princesa - Instituto de Inves...
Madrid, 28006
Hospital Universitario Virgen del Rocio (HUVR) - Instituto de Biomedicina de Sevilla (IBIS)
Seville, Seville, 41013
Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
Valencia, 46026
United Kingdom6 sites
Royal Devon University Healthcare NHS Foundation Trust - Royal Devon and Exeter Hospital (RD&E) -...
Exeter, South West, EX2 5PW
NHS Greater Glasgow and Clyde-Glasgow Clinical Research Facility - Queen Elizabeth University Hos...
Glasgow, South Western Scotland, G51 4TF
University College London Hospitals NHS Foundation Trust-National Hospital for Neurology & Neuros...
London, WC1N 3BG
Newcastle University - Clinical Ageing Research Unit (CARU)
Newcastle upon Tyne, North East, NE4 5PL
Oxford University Hospitals NHS Foundation Trust-Oxford Centre for Functional MRI of the Brain (F...
Oxford, South East, OX3 9DU
Northern Care Alliance NHS Foundation Trust - Salford Royal Hospital (Hope Hospital)
Salford, North West, United Kingdom, M6 8HD
Australia4 sites
University of New South Wales-Liverpool Hospital - Liverpool Cancer Therapy Centre
Liverpool, New South Wales, 2170
The Alfred Hospital
Melbourne, Victoria, 3004
Gold Coast University Hospital (GCUH) (Gold Coast Hospital)
Southport, Queensland, 4215
Westmead Hospital
Westmead, New South Wales, 2145
South Korea3 sites
Kyung Hee University Hospital
Seoul, 02447
Severance Hospital, Yonsei University Health System
Seoul, 03722
Samsung Medical Center
Seoul, 06351
Canada2 sites
The Ottawa Hospital - Centre for Rehabilitation Research and Development (CRRD) (The Institute fo...
Ottawa, Ontario, K1H 8M2
University Health Network-Toronto Western Hospital - Krembil Neuroscience Centre (KNC)
Toronto, Ontario, M5T 2S8
About this study
Official title: Interventional, Randomized, Double-blind, Placebo-controlled, Optional Open-label Extension Trial of Lu AF82422 in Participants With Multiple System Atrophy
The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA).
Read the detailed description
This study will consist of a screening period of 10 days up to 6 weeks, a 72-week placebo-controlled period (PCP), and will include a 72-week optional open-label treatment extension (OLE) period. Participants in the PCP will be randomized to amlenetug or placebo. All participants entering the OLE will receive amlenetug during the OLE. Participants will receive intravenous infusions approximately every 4 weeks during both the PCP and OLE.
- Conditions
- Multiple System Atrophy
- Being tested
- Amlenetug, Placebo
- Keywords
- Lu AF82422, MSA, Neurodegenerative Disorder
- Medical subject headings
- Multiple System Atrophy, Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmlenetugDrug
Also called Lu AF82422
Solution for infusion
PlaceboDrug
Commercially available saline solution
| Study group | Role | What they receive |
|---|---|---|
| Amlenetug | Experimental | Participants will receive amlenetug by intravenous infusion Receives: Drug: Amlenetug |
| Placebo | Placebo comparator | Participants will receive commercially available saline solution for infusion Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rest of the World (RoW; All Countries Except European Union [EU] and Japan [JP]) Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
Mortality-adjusted clinical progression will be assessed by the composite endpoint modified Unified Multiple System Atrophy Rating Scale (mUMSARS) score and time-to-death (any cause).
Measured: Baseline up to Week 72
EU and JP Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
Mortality-adjusted clinical progression will be assessed by the composite endpoint Unified Multiple System Atrophy Rating Scale (UMSARS) Total Score (TS) and time-to-death (any cause).
Measured: Baseline up to Week 72
Secondary outcomes (27)
RoW Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression
Mortality-adjusted clinical progression will be assessed by the composite endpoint UMSARS TS and time-to-death (any cause).
Measured: Baseline up to Week 72
EU and JP Regional-specific Outcome Measure: Change from Baseline in UMSARS TS
Measured: Baseline, Week 72
RoW Regional-specific Outcome Measure: Change from Baseline in UMSARS TS
Measured: Baseline, Week 72
Mortality-adjusted Clinical Progression
Mortality-adjusted clinical progression will be assessed by the composite endpoint UMSARS Part I and time-to-death (any cause).
Measured: Baseline up to Week 72
Mortality-adjusted Clinical Progression
Mortality-adjusted clinical progression will be assessed by the composite endpoint UMSARS Part II and time-to-death (any cause).
Measured: Baseline up to Week 72
Change from Baseline in mUMSARS Score
Measured: Baseline, Week 72
Change from Baseline in UMSARS Part I Score
Measured: Baseline, Week 72
Change from Baseline in UMSARS Part II Score
Measured: Baseline, Week 72
Change from Baseline in Clinical Global Impression - Severity of Illness (CGI-S) Score
Measured: Baseline, Week 72
Change from Baseline in Patient Global Impression - Severity of Illness (PGI-S) Score
Measured: Baseline, Week 72
Change from Baseline in Observer-reported Global Impression-Severity of Illness (OGI-S) Score
Measured: Baseline, Week 72
Change from Baseline in UMSARS Part IV Score
Measured: Baseline, Week 72
Change from Baseline in Schwab and England Activities of Daily Living (SE-ADL) Score
Measured: Baseline, Week 72
Change from Baseline in UMSARS Part I Item 1: Speech Score
Measured: Baseline, Week 72
Time to Disability
Measured: Baseline up to Week 72
Change from Baseline in EuroQol 5-dimensions, 5-levels (EQ-5D-5L) Domain Score
Measured: Baseline, Week 72
Change from Baseline in EQ-5D-5L Visual Analog Scale (VAS) Score
Measured: Baseline, Week 72
Change from Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Questionnaire Domain Scores
Measured: Baseline, Week 72
Combined Clinical Progression and Survival Score
Measured: Baseline, Week 72
Time from Baseline to Death (Any Cause)
Measured: Baseline up to Week 72
Number of Participants with an Absolute Increase in mUMSARS Score of <5, <7, and <9 points
Measured: Baseline to Week 72
Number of Participants with an Absolute Increase in UMSARS TS of <16, <21, and <26 Points
Measured: Baseline to Week 72
Percentage Change from Baseline in Brain Volume in Brain Regions of Interest (ROIs)
Measured: Baseline, Week 72
Area Under the Curve (AUC) of amlenetug
Measured: Baseline up to Week 72
Maximum Observed Concentration (Cmax) of Amlenetug
Measured: Baseline up to Week 72
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Measured: Day 1 up to Week 144
Number of Participants with Anti-amlenetug Antibodies (ADAs)
Measured: Baseline up to Week 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2024
- Study start3 Dec 2024
- Primary completion17 Feb 2028 (estimated)
- Study completion25 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System AtrophyStudies of the same condition, open and under way.
- More trials run by H. Lundbeck A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 401 participants (actual)
Sponsor and investigators
- Lead sponsor
- H. Lundbeck A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Email contact via H. Lundbeck A/S, Study director, H. Lundbeck A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06706622
- Sponsor's protocol ID
- 20432A
- Other IDs
- 2024-517169-18-00
- Acronym
- MASCOT
- Status last verified
- Oct 2026
- First submitted
- 22 Nov 2024
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No