Clinical trial • Phase II • Neurology

EMRUSOLMIN for Multiple system atrophy

Phase II trial of EMRUSOLMIN for Multiple system atrophy.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Multiple system atrophy
Trial Stage
Phase II
Drug Modality
Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
10-01-2024
First CTIS Authorization Date
16-04-2024

Trial design

Randomised, placebo to match imp tev-56286 (matching placebo with excipients only). dose and schedule not specified in the available part i documentation.-controlled Phase II trial in Germany, Italy, Spain and others.

Randomised
Yes
Comparator
Placebo to match IMP TEV-56286 (matching placebo with excipients only). Dose and schedule not specified in the available Part I documentation.
Target Sample Size
154
Trial Duration For Participant
392

Eligibility

Recruits 154 Vulnerable population selected. The trial includes specific subject information and informed consent forms for caregivers, legal representatives and pregnant partners (e.g. documents titled L1_SIS-ICF_Caregiver_FP, L1_SIS-ICF_Legal Representative PGx_FP, L1_SIS-ICF_Preg Participant_FP, L1_SIS-ICF_Preg Partner_FP, and country-specific legal representative ICFs such as L1_FR_SIS-ICF_Main Legal Representative_French_redacted). Consent is obtained via site ICFs; where applicable a legal representative / caregiver ICF is provided as indicated by the available documents..

Vulnerable Population
Vulnerable population selected. The trial includes specific subject information and informed consent forms for caregivers, legal representatives and pregnant partners (e.g. documents titled L1_SIS-ICF_Caregiver_FP, L1_SIS-ICF_Legal Representative PGx_FP, L1_SIS-ICF_Preg Participant_FP, L1_SIS-ICF_Preg Partner_FP, and country-specific legal representative ICFs such as L1_FR_SIS-ICF_Main Legal Representative_French_redacted). Consent is obtained via site ICFs; where applicable a legal representative / caregiver ICF is provided as indicated by the available documents.

Inclusion criteria

  • {"criterion_text":"- Is a male or female aged ≥30 to ≤75 years old at screening"}
  • {"criterion_text":"- Is considered to be “clinically possible” or “clinically probable” MSA as determined by the Gilman criteria."}
  • {"criterion_text":"- Is able to ambulate at least 10 meters without the assistance of another person. Use of an assistive device (cane only, not walker) is allowed during this assessment. Apart from the 10-meter walk at screening, use of any assistive device is allowed during the trial."}

Exclusion criteria

  • {"criterion_text":"- Has any clinically significant uncontrolled medical or psychiatric condition (treated or untreated), or any findings from vital signs, imaging, physical examination, electrocardiogram (QTcF >450 msec), or clinical laboratory, other than MSA related, that could jeopardize or compromise the participant’s safety, ability to participate, or integrity of the trial. The investigator should consult with the medical monitor or trial physician as needed."}
  • {"criterion_text":"- Is severely affected with a UMSARS part IV score of > 3"}
  • {"criterion_text":"- Is suspected of having a neurodegenerative disease other than MSA"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The primary efficacy endpoint (TEV-56286 versus placebo, change from baseline to week 48) is as follows: •\tFor non-EU: modified UMSARS part I score (excluding item 11, item scoring rescaled 0-3) •\tFor EU: total UMSARS score (part I and part II combined)","definition_or_measurement_approach":"Change from baseline to week 48; for non-EU sites the modified UMSARS part I score (excluding item 11, item scoring rescaled 0-3) is used; for EU sites the total UMSARS score (parts I and II combined) is used."}

Secondary endpoints

  • {"endpoint_text":"- The key secondary endpoints (TEV-56286 versus placebo, change from baseline to week 48) are as follows:•For non-EU:total UMSARS score(part I and part II combined)•For EU:modified UMSARS part I score(excluding item 11, item scoring rescaled 0-3)•UMSARS part I score•Lateral ventricle volume measured by MRI•CGI-S•NfL concentrations in CSF•PGI-S","definition_or_measurement_approach":"Change from baseline to week 48; includes UMSARS scores (total or modified), MRI lateral ventricle volume, Clinical Global Impression-Severity (CGI-S), neurofilament light (NfL) concentrations in CSF, and Patient Global Impression-Severity (PGI-S)."}
  • {"endpoint_text":"- Safety endpoints include the following: 1. number (%) of participants per adverse event from baseline","definition_or_measurement_approach":"Count and percentage of participants experiencing each adverse event from baseline."}
  • {"endpoint_text":"- Safety endpoints include the following: 2. number (%) of participants who withdraw from the trial due to an adverse event from baseline","definition_or_measurement_approach":"Count and percentage of participants who withdraw due to an adverse event from baseline."}
  • {"endpoint_text":"- Safety endpoints include the following: 4. number (%) of participants with potentially clinically significant vital sign values from baseline","definition_or_measurement_approach":"Count and percentage of participants with potentially clinically significant changes in vital signs from baseline."}
  • {"endpoint_text":"- Safety endpoints include the following: 5. number (%) of participants with potentially clinically significant laboratory tests values (hematology and chemistry) from baseline","definition_or_measurement_approach":"Count and percentage of participants with potentially clinically significant hematology and chemistry results from baseline."}
  • {"endpoint_text":"- Safety endpoints include the following: 6. number (%) of participants with potentially clinically significant 12-lead ECG measurements from baseline","definition_or_measurement_approach":"Count and percentage of participants with potentially clinically significant 12-lead ECG changes from baseline."}
  • {"endpoint_text":"- Safety endpoints include the following: 3. number (%) of participants who withdraw from treatment due to an adverse event from baseline","definition_or_measurement_approach":"Count and percentage of participants who discontinue treatment due to an adverse event from baseline."}
  • {"endpoint_text":"- The other secondary efficacy endpoints (TEV-56286 versus placebo, change from baseline to week 48) are as follows: •\tPons volume measured by MRI •\tCerebellar volume measured by MRI •\tUMSARS part II score •\tUMSARS part IV score •\tTwo-minute walk test as part of gait assessment •\tMSA-QoL","definition_or_measurement_approach":"Change from baseline to week 48 measured by MRI-derived volumes (pons, cerebellum), UMSARS part II and IV scores, two-minute walk test distance/time, and MSA-specific quality of life questionnaire (MSA-QoL)."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
154
Recruitment Window Months
32
Consent Approach
Informed consent obtained via site subject information and informed consent forms. Multiple ICFs and supporting documents exist including main ICFs, caregiver ICFs, legal representative ICFs, pregnant participant/partner ICFs, optional genetic research ICFs and privacy/reimbursement forms (documents include L1_SIS-ICF_Main redacted and localized versions, L1_SIS-ICF_Caregiver_FP, L1_SIS-ICF_Legal Representative PGx_FP, L1_SIS-ICF_Preg Participant_FP, etc.). ICFs are available in multiple local languages as indicated by country-specific redacted ICF documents (German, Italian, Spanish, French and English-language patient-facing documents). Consent is provided by the participant; where participants are vulnerable, a legal representative or caregiver ICF is provided as indicated.

Methods

  • Online posting and digital outreach (documents: K2_Online Posting_FP, K2_Patient Recruitment Digital Outreach_FP, K2_Part Recruit Digital Outreach_FP, K2_Participant_Recruit_Digital_Outreach_FP) targeted to patients
  • Social media advertising (Facebook Ad documents: K2_Facebook Ad_FP and localized Facebook Ad files) targeting potential participants
  • Healthcare professional engagement (HCP letters and HCP factsheets: K2_HCP Letter_FP, K2_HCP_Factsheet_FP) to recruit via clinicians
  • Patient-facing printed materials and localized brochures/advocacy factsheets (e.g. K2_DE_Recruitment Material_Brochure_German, K2_DE_Recruitment Material_Advocacy Factsheet_German, K2_IT_Recruitment Material_Study Brochure_Italian, K2_ES_Recruitment Material_Study Brochure_Spanish, K2_FR_Recruitment Material_Study Brochure_French) targeted by country
  • Study-specific flyers (e.g. K2_Lumbar Puncture Flyer_FP) and participant materials (subject/patient cards, tote bags) provided at sites

Geography

Total Number Of Sites
33
Total Number Of Participants
196

Germany

Earliest CTIS Part Ii Submission Date
26-03-2024
Latest Decision Or Authorization Date
08-05-2026
Processing Time Days
774
Number Of Sites
10
Number Of Participants
64

Sites

Site Name
Paracelsus-Kliniken Deutschland GmbH & Co. KGaA
Contact Person Name
Brit Mollenhauer
Site Name
Kliniken Beelitz GmbH
Department Name
Neurologisches Fachkrankenhaus für Bewergungsstörungen / Parkinson
Contact Person Name
Katharina Rukavina
Contact Person Email
rukavina@kliniken-beelitz.de
Site Name
Medical Center - University Of Freiburg
Department Name
Neurozentrum Klinik für Neurologie und Neurophysiologie
Contact Person Name
Michel Rijntjes
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Klinik für Neurologie
Contact Person Name
Georg Bernhard Landwehrmeyer
Site Name
Westfaelische Wilhelms-Universitaet Muenster
Department Name
Klinik für Neurologie mit Institut für Translationale Neurologie
Contact Person Name
Inga Claus
Contact Person Email
inga.claus@ukmuenster.de
Site Name
Universitaet Leipzig
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Jost-Julian Rumpf
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Neurologische Klinik und Poliklinik Klinikum Großhadern
Contact Person Name
Günter Höglinger
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
Klinik für Neurologie Inst für Klin Neurowiss und Medizin Psych Zentr für Bewegungsstör und Neuromod
Contact Person Name
Alfons Schnitzler
Site Name
Technische Universitat Dresden
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Björn Falkenburger
Contact Person Email
Bjoern.Falkenburger@ukdd.de
Site Name
Philipps-Universitaet Marburg
Department Name
Klinik für Neurologie
Contact Person Name
Karla Maria Eggert
Contact Person Email
KarlaMaria.Eggert@uk-gm.de

Italy

Earliest CTIS Part Ii Submission Date
29-01-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
833
Number Of Sites
7
Number Of Participants
48

Sites

Site Name
Asst Centro Specialistico Ortopedico Traumatologico Gaetano Pini Cto
Department Name
Parkinson Institute Milan
Contact Person Name
Luca Magistrelli
Site Name
Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania
Department Name
Clinica Neurologica
Contact Person Name
Alessandra Nicoletti
Contact Person Email
anicolet@unict.it
Site Name
Azienda Ospedale-Universita Padova
Department Name
U.O.C. Clinica Neurologica
Contact Person Name
Angelo Antonini
Contact Person Email
angelo.antonini@aopd.veneto.it
Site Name
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona
Department Name
Clinica Neurologica
Contact Person Name
Maria Teresa Pellecchia
Contact Person Email
mpellecchia@unisa.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Dipartimento Neurologico
Contact Person Name
Maria Antonietta Volontè
Contact Person Email
volonte.mariaantonietta@hsr.it
Site Name
Azienda Unita Sanitaria Locale Di Bologna
Department Name
UOC Clinica Neurologica Neuromet - IRCCS Istituto Delle Scienze Neurologiche di Bologna
Contact Person Name
Giovanna Calandra Buonaura
Contact Person Email
giovanna.calandra@unibo.it
Site Name
Irccs San Raffaele Roma S.r.l.
Department Name
IRCCS San Raffaele - Department of Neurology - Via di Val Cannuta 250, Rome, 00166
Contact Person Name
Fabrizio Stocchi

Spain

Earliest CTIS Part Ii Submission Date
20-02-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
807
Number Of Sites
11
Number Of Participants
59

Sites

Site Name
Clinica Universidad De Navarra
Department Name
Neurology service
Contact Person Name
Maria Rosario Luquin Piudo
Contact Person Email
rluquin@unav.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology service
Contact Person Name
Jorge Hernandez-Vara
Contact Person Email
jorge.hernandez@vallhebron.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Neurology Service
Contact Person Name
Jose Luis Lopez-Sendón Moreno
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neurology service
Contact Person Name
Javier Pagonabarraga Mora
Contact Person Email
jpagonabarraga@santpau.cat
Site Name
Hospital Universitario De La Princesa
Department Name
Neurology Service
Contact Person Name
Lydia López Manzanares
Contact Person Email
lydia.lopez@salud.madrid.org
Site Name
Hospital General Universitario De Elche
Department Name
Neurology Service
Contact Person Name
Eric Alejandro Freire Álvarez
Contact Person Email
dr.freyre@gmail.com
Site Name
Hospital Universitario De Cruces
Department Name
Neurology Service
Contact Person Name
Juan Carlos Gómez Esteban
Site Name
Hospital Clinic De Barcelona
Department Name
Neurology service
Contact Person Name
Celia Painous Marti
Contact Person Email
painous@clinic.cat
Site Name
Hospital Universitario 12 De Octubre
Department Name
Neurology Service
Contact Person Name
Alvaro Sanchez Ferro
Contact Person Email
asferro@salud.madrid.org
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Neurology service
Contact Person Name
Pablo Mir Rivera
Contact Person Email
pmir@us.es
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology service
Contact Person Name
Irene Martinez Torres
Contact Person Email
martinez_ire@gva.es

France

Earliest CTIS Part Ii Submission Date
01-04-2024
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
767
Number Of Sites
5
Number Of Participants
25

Sites

Site Name
Centre Hospitalier Universitaire De Caen Normandie
Department Name
Neurology
Contact Person Name
Claire Thiriez
Contact Person Email
thiriez-c@chu-caen.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Department of Neurology for Neurodegenerative Diseases
Contact Person Name
Wassilios Meissner
Site Name
Assistance Publique Hopitaux De Marseille
Department Name
Service de Neurologie
Contact Person Name
Alexandre Eusebio
Contact Person Email
Alexandre.eusebio@ap-hm.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Neurology
Contact Person Name
David Grabli
Contact Person Email
david.grabli@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
centre expert Parkinson
Contact Person Name
Margherita Fabbri
Contact Person Email
fabbri.m@chu-toulouse.fr

Sponsor

Primary sponsor

Full Name
Teva Branded Pharmaceutical Products R&D LLC
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Icon Clinical Research Limited
Responsibilities
Multiple trial operational responsibilities including in-home services and other vendor roles (sponsorDuties codes listed in CTIS entry).
Name
Eresearchtechnology Inc.
Responsibilities
Cardiac Safety and Precision Motion service
Name
Bioclinica Inc.
Responsibilities
MRI Imaging
Name
Perceptive Eclinical Limited
Responsibilities
Clinical data services (sponsorDuties code 3)
Name
Medidata Solutions Inc.
Responsibilities
Electronic data capture (EDC)

Third parties

  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Safety, and Precision Motion service","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Codes: 1,12,13,14,15 (includes In-Home Services)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Activinsights Limited","duties_or_roles":"Companion Device (Wearable Biomarkers)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Perceptive Eclinical Limited","duties_or_roles":"Code: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"MRI Imaging","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Retention","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"sample storage; Code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Sweden","full_name":"Olink Proteomics AB","duties_or_roles":"Biomarker","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Watson Pharma Private Limited","duties_or_roles":"PK and CSF in Plasma","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"EDC","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Genewiz LLC","duties_or_roles":"Pharmacogenomics","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Emrusolmin
Active Substance
EMRUSOLMIN
Modality
Small molecule
Routes Of Administration
Oral
Route
ORAL USE
Authorisation Status
prodAuthStatus:1
Orphan Designation
Yes
Maximum Dose
300 mg per day (maxDailyDoseAmount 300 mg)
Investigational Product Name
Placebo to match IMP TEV-56286
Modality
Other

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