Clinical trial • Phase II/III • Immunology|Neurology
EFGARTIGIMOD ALFA for Generalized myasthenia gravis
Phase II/III trial of EFGARTIGIMOD ALFA for Generalized myasthenia gravis. open-label, none/not specified-controlled. 7 participants.
Overview
- Trial Therapeutic Area
- Immunology|Neurology
- Trial Disease
- Generalized myasthenia gravis
- Trial Stage
- Phase II/III
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 05-06-2024
- First CTIS Authorization Date
- 24-06-2024
Trial design
open-label, none/not specified-controlled Phase II/III trial in Spain, Belgium, Poland and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 7
Eligibility
Recruits 7 paediatric patients.
- Vulnerable Population
- The trial enrols children aged 2 to <18 years (paediatric population). Informed consent must be provided by the participant and/or his/her legally authorized representative; assent is required where applicable. Age-specific parent/guardian consent and pediatric assent forms are provided (multiple age-band assent forms available).
Inclusion criteria
- {"criterion_text":"- Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable, willingness and ability to comply with the trial protocol procedures."}
- {"criterion_text":"- Male or female participants between 2 to < 18 years of age at the time of providing informed consent/assent."}
- {"criterion_text":"- Diagnosed with gMG with confirmed documentation"}
- {"criterion_text":"- Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa."}
- {"criterion_text":"- Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or AChE inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening."}
- {"criterion_text":"- Positive serologic test for anti-AChR antibodies at screening (for younger participants (<15kg) historical values can be used)."}
Exclusion criteria
- {"criterion_text":"- Participants with MGFA class I, IVb, and V."}
- {"criterion_text":"- Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc)."}
- {"criterion_text":"- A documented lack of clinical response to plasma exchange (PLEX)."}
- {"criterion_text":"- Received a live or live-attenuated vaccine fewer than 28 days before screening. (Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.)"}
- {"criterion_text":"- Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period."}
- {"criterion_text":"- A known hypersensitivity reaction to efgartigimod or any of its excipients."}
- {"criterion_text":"- Current participation in another interventional clinical trial."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Efgartigimod concentrations as input for compartmental, model-driven analysis to determine age and size dependency of clearance and volume of distribution","definition_or_measurement_approach":"Measurement of efgartigimod serum concentrations to be used as input for compartmental, model-driven PK analysis to determine age- and size-dependency of clearance (CL) and volume of distribution (Vd)."}
- {"endpoint_text":"- PD parameters: total IgG levels and anti-acetylcholine receptors antibodies (AChR-Ab) as input for PK/PD modeling analysis","definition_or_measurement_approach":"Measurement of total IgG and anti-AChR antibody levels for PK/PD modelling (used as PD input parameters)."}
Secondary endpoints
- {"endpoint_text":"- Incidence and severity of AEs, incidence of serious AEs (SAEs), incidence of AEs of special interest (AESIs), and changes in laboratory test results, physical examination results, vital sign measurements, and electrocardiogram (ECG) (part B only for ECGs)","definition_or_measurement_approach":"Safety monitoring via AE/SAE/AESI reporting and serial laboratory tests, physical exams, vital signs and ECGs (ECGs specified for part B)."}
- {"endpoint_text":"- Efgartigimod serum concentrations","definition_or_measurement_approach":"Measurement of serum efgartigimod concentrations over time (PK sampling)."}
- {"endpoint_text":"- Levels of total IgG and AChR-Ab: absolute values, change from baseline and percent (%) change from baseline","definition_or_measurement_approach":"Quantification of total IgG and anti-AChR antibody levels with assessment of absolute values and change from baseline (including percent change)."}
- {"endpoint_text":"- Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod","definition_or_measurement_approach":"Assessment of ADA occurrence and prevalence via immunogenicity assays."}
- {"endpoint_text":"- MG-ADL total score: absolute value and change from baseline total MG-ADL score","definition_or_measurement_approach":"Clinical efficacy assessment using MG-ADL total score (absolute and change from baseline)."}
- {"endpoint_text":"- Total QMG score: absolute value and change from baseline","definition_or_measurement_approach":"Clinical efficacy assessment using quantitative myasthenia gravis (QMG) total score (absolute and change from baseline)."}
- {"endpoint_text":"- Total score EQ-5D-Y: absolute value and change from baseline","definition_or_measurement_approach":"Health-related quality of life measured by EQ-5D-Y total score (absolute and change from baseline)."}
- {"endpoint_text":"- Quality of Life in Neurological Disorders (Neuro-QoL) Pediatric Fatigue Score: values and change from baseline","definition_or_measurement_approach":"Assessment of pediatric fatigue using Neuro-QoL Pediatric Fatigue Score (values and change from baseline)."}
- {"endpoint_text":"- Change in protective antibody titers to vaccines received before or received during the trial","definition_or_measurement_approach":"Measurement of protective antibody titers to vaccines to assess changes pre- and post-treatment."}
Recruitment
- Planned Sample Size
- 7
- Recruitment Window Months
- 65
- Consent Approach
- Informed consent is required from participants and/or their legally authorized representative (parent/guardian) with written consent; assent is required where applicable. Age-specific assent and parent/guardian consent forms are provided (multiple pediatric assent forms for age bands such as 2–6, 7–9, 10–13, 14–17, and adolescent assent forms). Consent/assent documents are available in multiple languages (examples in the dossier include English, Dutch, French, German, Italian, Polish, Spanish).
Geography
- Total Number Of Sites
- 13
- Total Number Of Participants
- 12
Spain
- Earliest CTIS Part Ii Submission Date
- 23-05-2024
- Latest Decision Or Authorization Date
- 01-08-2025
- Processing Time Days
- 435
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Servicio de Neurología
- Principal Investigator Name
- Teresa Sevilla Mantecón
- Principal Investigator Email
- sevilla_ter@gva.es
- Contact Person Name
- Teresa Sevilla Mantecón
- Contact Person Email
- sevilla_ter@gva.es
- Site Name
- Sant Joan De Deu Barcelona Hospital
- Department Name
- Unidad de Enfermedades Neuromusculares
- Principal Investigator Name
- Andrés Nascimento Osorio
- Principal Investigator Email
- andres.nascimento@sjd.es
- Contact Person Name
- Andrés Nascimento Osorio
- Contact Person Email
- andres.nascimento@sjd.es
Belgium
- Earliest CTIS Part Ii Submission Date
- 16-05-2024
- Latest Decision Or Authorization Date
- 01-08-2025
- Processing Time Days
- 442
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Universitair Ziekenhuis Antwerpen
- Department Name
- Neurology
- Principal Investigator Name
- Diane Beysen
- Principal Investigator Email
- diane.beysen@uza.be
- Contact Person Name
- Diane Beysen
- Contact Person Email
- diane.beysen@uza.be
Poland
- Earliest CTIS Part Ii Submission Date
- 21-05-2024
- Latest Decision Or Authorization Date
- 05-08-2025
- Processing Time Days
- 441
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Neurolgii Rozwojowej
- Principal Investigator Name
- Maria Mazurkiewicz-Bełdzińska
- Principal Investigator Email
- mmazur@gumed.edu.pl
- Contact Person Name
- Maria Mazurkiewicz-Bełdzińska
- Contact Person Email
- mmazur@gumed.edu.pl
- Site Name
- Neurologia Śląska Centrum Medyczne
- Principal Investigator Name
- Marek Andrzej Śmiłowski
- Principal Investigator Email
- marek.smilowski@neurologiaslaska.pl
- Contact Person Name
- Marek Andrzej Śmiłowski
- Contact Person Email
- marek.smilowski@neurologiaslaska.pl
- Site Name
- Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
- Department Name
- Centralny Szpital Kliniczny, 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
Germany
- Earliest CTIS Part Ii Submission Date
- 17-05-2024
- Latest Decision Or Authorization Date
- 17-04-2026
- Processing Time Days
- 700
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Sozialpädiatrisches Zentrum (SPZ) Neuropädiatrie
- Principal Investigator Name
- Claudia Weiß
- Principal Investigator Email
- claudia.weiss@charite.de
- Contact Person Name
- Claudia Weiß
- Contact Person Email
- claudia.weiss@charite.de
France
- Earliest CTIS Part Ii Submission Date
- 15-05-2024
- Latest Decision Or Authorization Date
- 04-05-2026
- Processing Time Days
- 719
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Service de Neuropédiatrie
- Principal Investigator Name
- Isabelle Desguerre
- Principal Investigator Email
- isabelle.desguerre@aphp.fr
- Contact Person Name
- Isabelle Desguerre
- Contact Person Email
- isabelle.desguerre@aphp.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Centre Hospitalier Universitaire (CHU) Hôpital de La Timone Service de neuro-métabolisme pédiatrique
- Principal Investigator Name
- Cécile Halbert
- Principal Investigator Email
- cecile.halbert@ap-hm.fr
- Contact Person Name
- Cécile Halbert
- Contact Person Email
- cecile.halbert@ap-hm.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 23-05-2024
- Latest Decision Or Authorization Date
- 08-05-2026
- Processing Time Days
- 715
- Number Of Sites
- 3
- Number Of Participants
- 1
Sites
- Site Name
- University Of Bari Aldo Moro
- Department Name
- Neuropsichiatria Infantile
- Principal Investigator Name
- Emilia Matera
- Principal Investigator Email
- emilia.matera@uniba.it
- Contact Person Name
- Emilia Matera
- Contact Person Email
- emilia.matera@uniba.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Pediatric Neurology
- Principal Investigator Name
- Chiara Fiorillo
- Principal Investigator Email
- chiara.fiorillo@edu.unige.it
- Contact Person Name
- Chiara Fiorillo
- Contact Person Email
- chiara.fiorillo@edu.unige.it
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- Neurologia pediatrica
- Principal Investigator Name
- Renzo Guerrini
- Principal Investigator Email
- renzo.guerrini@meyer.it
- Contact Person Name
- Renzo Guerrini
- Contact Person Email
- renzo.guerrini@meyer.it
Netherlands
- Earliest CTIS Part Ii Submission Date
- 21-05-2024
- Latest Decision Or Authorization Date
- 15-05-2026
- Processing Time Days
- 724
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- Neurology
- Principal Investigator Name
- Erik H. Niks
- Principal Investigator Email
- Myasthenie@Lumc.nl
- Contact Person Name
- Erik H. Niks
- Contact Person Email
- Myasthenie@Lumc.nl
Sponsor
Primary sponsor
- Full Name
- Argenx
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- PPD Development LP
- Responsibilities
- Vendor Management; multiple operational vendor roles (codes and values listed in sponsor duties)
- Name
- IQVIA Limited
- Responsibilities
- Clinical trial support; contact eu_clinical_trials_information@iqvia.com (code 8)
- Name
- Endpoint Clinical Inc.
- Responsibilities
- Electronic data capture / clinical technology (support)
- Name
- Fisher Clinical Services GmbH
- Responsibilities
- IMP packaging, labelling, storage and distribution; QP release
- Name
- SGS Belgium
- Responsibilities
- DSMB support and multiple laboratory/operational services
Third parties
- {"country":"Switzerland","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"IMP packaging, labelling, storage and distribution","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"code 3","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"Labelling, QP release, storage and distribution","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"IQVIA Laboratories LLC","duties_or_roles":"Vaccine protective antibody titers","organisation_type":"Industry"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/review. This vendor will provide Cardiac Safety Services for PPD","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 8","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"codes 10,13,15 (DSMB support),5,6,7","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"Analysis of PK, PD and ADA; code 4","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 States","full_name":"PPD Development LP","duties_or_roles":"multiple roles including Vendor Management (codes: 1,11,12,13,15,2,5,8 as listed)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"AChR analysis","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- Vyvgart 20 mg/mL concentrate for solution for infusion
- Active Substance
- EFGARTIGIMOD ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous infusion
- Route
- Intravenous infusion
- Authorisation Status
- Marketing authorisation EU/1/22/1674/001 (authorised product referenced in dossier)
- Orphan Designation
- Yes
- Maximum Dose
- 1200 mg (max daily dose amount as listed)
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