Clinical trial • Phase II • Neurology
EMPASIPRUBART for Multifocal motor neuropathy
Phase II trial of EMPASIPRUBART for Multifocal motor neuropathy.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Multifocal motor neuropathy
- Trial Stage
- Phase II
- Drug Modality
- Monoclonal antibody
Key dates
- Initial CTIS Submission Date
- 11-12-2023
- First CTIS Authorization Date
- 06-02-2024
Trial design
Randomised, open-label, placebo to argx-117 iv (placebo comparator; no dose/schedule specified in record)-controlled Phase II trial in Netherlands, Germany, Belgium and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Placebo to ARGX-117 IV (placebo comparator; no dose/schedule specified in record)
- Target Sample Size
- 17
Eligibility
Recruits 17 Vulnerable population flag is selected. Participants must be capable of providing signed informed consent and must be able to read and write. Consent is provided by the participant (no paediatric assent procedures described)..
- Pregnancy Exclusion
- 4. Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the IMP.
- Vulnerable Population
- Vulnerable population flag is selected. Participants must be capable of providing signed informed consent and must be able to read and write. Consent is provided by the participant (no paediatric assent procedures described).
Inclusion criteria
- {"criterion_text":"- Participants are eligible to be included in the trial only if all of the following criteria apply: 1. Capable of providing signed informed consent, and complying with protocol requirements. Participants must be able to read and write."}
- {"criterion_text":"- 2. Must have completed the double-blinded treatment period of the ARGX-117-2002 trial and considered to be eligible for treatment with ARGX-117"}
- {"criterion_text":"- 3a. Male participants: must use an acceptable contraceptive method that should be maintained at minimum until 15 months after last dose of Investigational Medicinal Product (IMP)."}
- {"criterion_text":"- 3b. Female participants (women) of childbearing potential must have a negative urine pregnancy test at baseline before IMP can be administered."}
Exclusion criteria
- {"criterion_text":"- Participants will be excluded from the trial if any of the following criteria apply: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection"}
- {"criterion_text":"- 2. Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition, in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk."}
- {"criterion_text":"- 3. Currently participating in another interventional clinical study"}
- {"criterion_text":"- 4. Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the IMP."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Safety outcomes based on AE monitoring and other safety assessments (clinical laboratory tests)","definition_or_measurement_approach":"Based on adverse event monitoring and other safety assessments including clinical laboratory tests."}
Secondary endpoints
- {"endpoint_text":"- 1. Modified Medical Research Council (mMRC) − Value and change from baseline in the mMRC-10 sum score − Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score − Value and change from baseline in the mMRC-14 sum score − Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score","definition_or_measurement_approach":"mMRC-10 and mMRC-14 scores: value and change from baseline; proportion with ≥2 point deterioration."}
- {"endpoint_text":"- − Value and change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score","definition_or_measurement_approach":"Average of the two most important muscle groups assessed via mMRC-14 sum score; value and change from baseline."}
- {"endpoint_text":"- 2. Grip strength (GS) − Values, change, and percent change from baseline in GS − Proportion of participants with a decline of >30% in GS","definition_or_measurement_approach":"Grip strength measured; absolute, change and percent change from baseline; proportion with >30% decline."}
- {"endpoint_text":"- 3. Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN‐RODS)","definition_or_measurement_approach":"MMN‐RODS score: value and change from baseline."}
- {"endpoint_text":"- 4. Values, change, and percentage change from baseline in the average time for the upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed pegboard test)","definition_or_measurement_approach":"9-HPT or timed pegboard: average time for upper extremity function; value, change and percent change from baseline."}
- {"endpoint_text":"- 5. Proportion of participants by level of severity on each dimension of EQ-5D-5L","definition_or_measurement_approach":"EQ-5D-5L dimensions: proportion by severity level."}
- {"endpoint_text":"- 6. Value and change from baseline in EQ-5D-5L visual analog scale (VAS)","definition_or_measurement_approach":"EQ-5D-5L VAS: value and change from baseline."}
- {"endpoint_text":"- 7. Values and change from baseline in the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI)","definition_or_measurement_approach":"CAP-PRI: value and change from baseline (patient-reported)."}
- {"endpoint_text":"- 8. Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)","definition_or_measurement_approach":"FSS (9-item): value and change from baseline."}
- {"endpoint_text":"- 9. Values of the Patient Global Impression of Change (PGIC) scale","definition_or_measurement_approach":"PGIC scores at visits."}
- {"endpoint_text":"- 10. Proportion of participants by level of severity of MMN as assessed by the Patient Global Impression of Severity (PGIS)","definition_or_measurement_approach":"PGIS: proportion by severity level."}
- {"endpoint_text":"- 11. Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ) at each visit: − Hours lost because of absenteeism − Hours lost because of presenteeism","definition_or_measurement_approach":"HRPQ: hours lost due to absenteeism and presenteeism at each visit."}
- {"endpoint_text":"- − Total hours lost − Percent of scheduled hours lost because of absenteeism − Percent of scheduled hours lost because of presenteeism − Percent of scheduled hours lost in total","definition_or_measurement_approach":"Work productivity endpoints: total hours lost and percent of scheduled hours lost (absenteeism, presenteeism, total)."}
- {"endpoint_text":"- 12. Serum concentrations and PK parameters for ARGX-117","definition_or_measurement_approach":"Serum concentration measurements and pharmacokinetic parameter calculation for ARGX-117."}
- {"endpoint_text":"- 13.Values and change from baseline in free C2, total C2, and functional complement activity (CH50) over time","definition_or_measurement_approach":"Laboratory PD measurements of free C2, total C2, and CH50 over time; values and change from baseline."}
- {"endpoint_text":"- 14. Incidence and prevalence of antidrug antibodies (ADA) against ARGX-117","definition_or_measurement_approach":"Immunogenicity testing: incidence and prevalence of ADA to ARGX-117."}
- {"endpoint_text":"- 15. Values and change from baseline in free C2, total C2, and functional complement activity (CH50) over time","definition_or_measurement_approach":"Duplicate entry in source: laboratory PD measurements (as above)."}
- {"endpoint_text":"- 16. Incidence and prevalence of antidrug antibodies (ADA) against ARGX-117","definition_or_measurement_approach":"Duplicate entry in source: ADA incidence and prevalence (as above)."}
Recruitment
- Planned Sample Size
- 17
- Recruitment Window Months
- 51
- Consent Approach
- Participants must provide signed informed consent and be capable of doing so; participants must be able to read and write. Subject information and informed consent forms are provided in country-specific languages (examples in documents: Dutch, German, English, French, Spanish, Polish, Italian). No paediatric assent procedures are described (adults only).
Geography
- Total Number Of Sites
- 17
- Total Number Of Participants
- 34
Netherlands
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 01-07-2025
- Processing Time Days
- 537
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Neurology and Neurosurgery
- Principal Investigator Name
- Ludo Van der Pol
- Principal Investigator Email
- W.L.vanderPol@umcutrecht.nl
- Contact Person Name
- Ludo Van der Pol
- Contact Person Email
- W.L.vanderPol@umcutrecht.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 01-07-2025
- Processing Time Days
- 537
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Universitätsklinikum Essen
- Department Name
- Klinik für Neurologie
- Principal Investigator Name
- Mark Stettner
- Principal Investigator Email
- mark.stettner@uk-essen.de
- Contact Person Name
- Mark Stettner
- Contact Person Email
- mark.stettner@uk-essen.de
- Site Name
- Universitätsmedizin Göttingen
- Department Name
- Klinik für Neurologie
- Principal Investigator Name
- Jana Zschüntzsch
- Principal Investigator Email
- j.zschuentzsch@med.uni-goettingen.de
- Contact Person Name
- Jana Zschüntzsch
- Contact Person Email
- j.zschuentzsch@med.uni-goettingen.de
Belgium
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 02-07-2025
- Processing Time Days
- 538
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- AZ Sint-Lucas & Volkskliniek
- Department Name
- Neurology
- Principal Investigator Name
- Jan De Bleecker
- Principal Investigator Email
- jan.debleecker@azstlucas.be
- Contact Person Name
- Jan De Bleecker
- Contact Person Email
- jan.debleecker@azstlucas.be
Spain
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 04-07-2025
- Processing Time Days
- 540
- Number Of Sites
- 3
- Number Of Participants
- 8
Sites
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Unidad de Enfermedades Neuromusculares
- Principal Investigator Name
- María Teresa Sevilla Mantecón
- Principal Investigator Email
- sevilla_ter@gva.es
- Contact Person Name
- María Teresa Sevilla Mantecón
- Contact Person Email
- sevilla_ter@gva.es
- Site Name
- Hospital De La Santa Creu I Sant Pau
- Department Name
- Servicio de Neurologia
- Principal Investigator Name
- Luis Antonio Querol Gutiérrez
- Principal Investigator Email
- lquerol@santpau.cat
- Contact Person Name
- Luis Antonio Querol Gutiérrez
- Contact Person Email
- lquerol@santpau.cat
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Unidad de Enfermedades Neuromusculares y Raras
- Principal Investigator Name
- Raúl Juntas Morales
- Principal Investigator Email
- raul.juntas@vallhebron.cat
- Contact Person Name
- Raúl Juntas Morales
- Contact Person Email
- raul.juntas@vallhebron.cat
Poland
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 07-07-2025
- Processing Time Days
- 543
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Medical University Of Warsaw
- Department Name
- Klinika Neurologii
- Principal Investigator Name
- Anna Kostera-Pruszczyk
- Principal Investigator Email
- anna.kostera-pruszczyk@wum.edu.pl
- Contact Person Name
- Anna Kostera-Pruszczyk
- Contact Person Email
- anna.kostera-pruszczyk@wum.edu.pl
Austria
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 07-07-2025
- Processing Time Days
- 543
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Neurology
- Principal Investigator Name
- Jakob Rath
- Principal Investigator Email
- jakob.rath@meduniwien.ac.at
- Contact Person Name
- Jakob Rath
- Contact Person Email
- jakob.rath@meduniwien.ac.at
France
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 30-06-2025
- Processing Time Days
- 536
- Number Of Sites
- 4
- Number Of Participants
- 4
Sites
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- Service de Neurologie / Centre de Référence des Maladies Neuromusculaires et SLA
- Principal Investigator Name
- Sabrina Sacconi
- Principal Investigator Email
- sacconi.s@chu-nice.fr
- Contact Person Name
- Sabrina Sacconi
- Contact Person Email
- sacconi.s@chu-nice.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Service de Neurologie et Maladies Neuromusculaires
- Principal Investigator Name
- Gwendal Le Masson
- Principal Investigator Email
- gwendal.le-masson@chu-bordeaux.fr
- Contact Person Name
- Gwendal Le Masson
- Contact Person Email
- gwendal.le-masson@chu-bordeaux.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Service de Neurophysiologie Clinique
- Principal Investigator Name
- Karine Viala
- Principal Investigator Email
- karine.viala@aphp.fr
- Contact Person Name
- Karine Viala
- Contact Person Email
- karine.viala@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Hôpital Roger Salengro Service de Neurologie A
- Principal Investigator Name
- Céline Tard
- Principal Investigator Email
- celine.tard@chru-lille.fr
- Contact Person Name
- Céline Tard
- Contact Person Email
- celine.tard@chru-lille.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 11-01-2024
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 778
- Number Of Sites
- 4
- Number Of Participants
- 6
Sites
- Site Name
- Azienda Ospedaliero Universitaria Pisana
- Department Name
- UOC Neurologia - Ambulatorio Malattie Neuromuscolari
- Principal Investigator Name
- Michelangelo Mancuso
- Principal Investigator Email
- michelangelo.mancuso@unipi.it
- Contact Person Name
- Michelangelo Mancuso
- Contact Person Email
- michelangelo.mancuso@unipi.it
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Dipartimento di Neurologia IRCCS Ospedale San Raffaele
- Principal Investigator Name
- Stefano Carlo Previtali
- Principal Investigator Email
- previtali.stefano@hsr.it
- Contact Person Name
- Stefano Carlo Previtali
- Contact Person Email
- previtali.stefano@hsr.it
- Site Name
- Humanitas Research Hospital
- Department Name
- U.O. Neuroimmunologia e Malattie Neuromuscolari
- Principal Investigator Name
- Eduardo Nobile Orazio
- Principal Investigator Email
- eduardo.nobile@unimi.it
- Contact Person Name
- Eduardo Nobile Orazio
- Contact Person Email
- eduardo.nobile@unimi.it
- Site Name
- Azienda Ospedaliero-Universitaria Sant Andre
- Department Name
- U.O.S Malattie Neuromuscolari
- Principal Investigator Name
- Stefania Morino
- Principal Investigator Email
- stefania.morino@ospedalesantandrea.it
- Contact Person Name
- Stefania Morino
- Contact Person Email
- stefania.morino@ospedalesantandrea.it
Sponsor
Primary sponsor
- Full Name
- Argenx
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- Cytel Inc.
- Responsibilities
- Biostats, IDMC
- Name
- Endpoint Clinical Inc.
- Name
- Eresearchtechnology Inc.
- Responsibilities
- ECG analysis/ review eCOA/ePRO
- Name
- IQVIA Limited
- Responsibilities
- Pharmacovigilance
- Name
- Cerba Research
- Responsibilities
- Primary/ surrogate endpoint test
- Name
- WCG Clinical Inc.
- Responsibilities
- Training portal for investigational sites; safety reporting to sites (Safety Vigilance).
- Name
- Parexel International (IRL) Limited
- Name
- PPD Development LP
- Responsibilities
- Vendor Management
- Name
- Celerion Switzerland AG
- Responsibilities
- PK and immunogenicity (ADA, Nab) analysis
- Name
- Sanquin Diagnostiek B.V.
- Responsibilities
- PD analysis (total C2, CH50)
- Name
- SYRINX Bioanalytics Oy
- Responsibilities
- PD analysis (free C2)
Third parties
- {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"Biostats, IDMC","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/ review eCOA/ePRO","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Long term storage of study samples","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"Cerba Research","duties_or_roles":"Primary/ surrogate endpoint test","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Training portal for investigational sites; safety reporting to sites (Safety Vigilance).","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"SYRINX Bioanalytics Oy","duties_or_roles":"PD analysis (free C2)","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Sanquin Diagnostiek B.V.","duties_or_roles":"PD analysis (total C2, CH50)","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Switzerland","full_name":"Celerion Switzerland AG","duties_or_roles":"PK and immunogenicity (ADA, Nab) analysis","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Vendor Management","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ARGX-117
- Active Substance
- EMPASIPRUBART
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- Authorised
- Investigational Product Name
- Placebo to ARGX-117 IV
- Modality
- Other
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