Clinical trial • Phase II/III • Immunology|Neurology|Rare Disease
Efgartigimod alfa for Generalized myasthenia gravis|Myasthenia gravis
Phase II/III trial of Efgartigimod alfa for Generalized myasthenia gravis|Myasthenia gravis. open-label. 12 participants.
Overview
- Trial Therapeutic Area
- Immunology|Neurology|Rare Disease
- Trial Disease
- Generalized myasthenia gravis|Myasthenia gravis
- Trial Stage
- Phase II/III
- Drug Modality
- Monoclonal antibody
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 13-12-2023
- First CTIS Authorization Date
- 31-01-2024
Trial design
open-label Phase II/III trial across 18 sites in Austria, Belgium, Netherlands and others.
- Open Label
- Yes
- Target Sample Size
- 12
Eligibility
Recruits 12 paediatric patients.
- Pregnancy Exclusion
- FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study
- Vulnerable Population
- Children (paediatric population) are included and flagged as a vulnerable population. Consent is to be provided by the participant and/or the participant's legally authorized representative (LAR); pediatric assent is required where appropriate. Multiple age-specific assent and parent/caregiver informed consent forms are provided (assent forms for 2-6, 7-9, 10-13, 14+ years, parent ICFs, caregiver ICFs and pregnancy/newborn ICFs), and documents are available in multiple languages (English, Dutch, French, German, Polish, Spanish, Italian, Czech as evidenced by ICF/assent documents listed).
Inclusion criteria
- {"criterion_text":"- The participant completed ARGX-113-2006 or ARGX-113-2207, defined as: a. The participant reached the end of the study in ARGX-113-2006 or ARGX-113-2207 and agreed to participate in the ARGX-113-2008 study. b. The participant qualifies for retreatment in ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 study's timeframe."}
- {"criterion_text":"- The participant and/or the participant's legally authorized representative (LAR) can understand the requirements of the study and provide written informed consent/assent, and demonstrates a willingness and ability to comply with the study protocol procedures."}
- {"criterion_text":"- Contraceptive use for sexually active participants of childbearing potential should be consistent with local regulations for those participating in clinical studies. A participant is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children (ie, female participants have started their menses, and male participants have reached the middle of puberty). a. Male participants: contraceptive requirements for male participants are presented in Section 10.4.2.2. b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1) must have a negative urine pregnancy test at study entry (TP1V1 or IP0V1). The contraceptive requirements for FAOCBP are described in Section 10.4.2.1."}
Exclusion criteria
- {"criterion_text":"- FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study"}
- {"criterion_text":"- Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment"}
- {"criterion_text":"- A known hypersensitivity reaction to efgartigimod or any of its excipients"}
- {"criterion_text":"- Any of the following medical conditions: a. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at study entry not sufficiently resolved in the investigtor's opinion. b. Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or put the participant at undue risk"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Incidence and severity of adverse events (AEs)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of serious adverse events (SAEs)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Incidence and severity of adverse events of special interest (AESIs)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Changes in laboratory test results, vital signs, height and weight, and electrocardiogram (ECG) results","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod and antibodies against recombinant human hyaluronidase PH20 (rHuPH20).","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 12
- Recruitment Window Months
- 85
- Consent Approach
- Informed consent is obtained from the participant and/or the participant's legally authorized representative (LAR). Pediatric assent is obtained according to age group (multiple pediatric assent forms for ages 2-6, 7-9, 10-13, 14+ are provided). Parent/guardian ICFs and caregiver ICFs are available. Documents and ICF/assent forms are provided in multiple languages (English, Dutch, French, German, Polish, Spanish, Italian, Czech) as listed in the public documents.
Geography
- Total Number Of Sites
- 18
- Total Number Of Participants
- 19
Austria
- Latest Decision Or Authorization Date
- 27-02-2024
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Pediatrics and Adolescent Medicine
- Contact Person Name
- Sandy Siegert
- Contact Person Email
- sandy.siegert@meduniwien.ac.at
Belgium
- Latest Decision Or Authorization Date
- 02-02-2024
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Neurology
- Contact Person Name
- Nicolas Deconinck
- Contact Person Email
- nicolas.deconinck@huderf.be
- Site Name
- Antwerp University Hospital
- Department Name
- Pediatrics
- Contact Person Name
- Diane Beysen
- Contact Person Email
- diane.beysen@uza.be
Netherlands
- Latest Decision Or Authorization Date
- 05-02-2024
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Academisch Ziekenhuis Leiden
- Department Name
- Neurology
- Contact Person Name
- Erik H. Niks
- Contact Person Email
- e.h.niks@lumc.nl
France
- Latest Decision Or Authorization Date
- 31-01-2024
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Service de neuro-métabolisme pédiatrique
- Contact Person Name
- Cecile HALBERT
- Contact Person Email
- cecile.halbert@ap-hm.fr
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Service de Neuropédiatrie
- Contact Person Name
- Isabelle Desguerre
- Contact Person Email
- isabelle.desguerre@aphp.fr
Germany
- Latest Decision Or Authorization Date
- 21-11-2025
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Sozialpädiatrisches Zentrum (SPZ) Neuropädiatrie
- Contact Person Name
- Claudia Weiß
- Contact Person Email
- claudia.weiss@charite.de
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Kinderklinik I, Neuropädiatrie, Sozialpaediatrisches Zentrum
- Contact Person Name
- Adela Della Marina
- Contact Person Email
- adela.dellamarina@uk-essen.de
Spain
- Latest Decision Or Authorization Date
- 24-11-2025
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Sant Joan De Deu Barcelona Hospital
- Department Name
- Unidad de Enfermedades Neuromusculares
- Contact Person Name
- Andrés Nascimento Osorio
- Contact Person Email
- andres.nascimento@sjd.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Servicio de Neurología
- Contact Person Name
- Teresa Sevilla Mantecón
- Contact Person Email
- sevilla_ter@gva.es
Czechia
- Latest Decision Or Authorization Date
- 12-06-2025
- Number Of Sites
- 2
- Number Of Participants
- 1
Sites
- Site Name
- Fakultni Nemocnice Ostrava
- Department Name
- Oddělení dětské neurologie
- Contact Person Name
- Hana Medřická
- Contact Person Email
- hana.medricka@fno.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Dětská nemocnice, Klinika dětské neurologie
- Contact Person Name
- Ondřej Havlín
- Contact Person Email
- havlin.ondrej@fnbrno.cz
Poland
- Latest Decision Or Authorization Date
- 20-05-2024
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Neurologia Śląska Centrum Medyczne
- Contact Person Name
- Marek Śmiłowski
- Contact Person Email
- marek.smilowski2@gmail.com
- Site Name
- Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
- Department Name
- Klinika Neurologii Centralny Szpital Kliniczny
- Contact Person Name
- Anna Kostera-Pruszczyk
- Contact Person Email
- anna.kostera-pruszczyk@wum.edu.pl
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Neurologii Rozwojowej
- Contact Person Name
- Maria Mazurkiewicz-Bełdzińska
- Contact Person Email
- mmazur@gumed.edu.pl
Italy
- Latest Decision Or Authorization Date
- 05-02-2024
- Number Of Sites
- 3
- Number Of Participants
- 2
Sites
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Pediatric Neurology
- Contact Person Name
- Chiara Fiorillo
- Contact Person Email
- chiara.fiorillo@edu.unige.it
- Site Name
- University Of Bari Aldo Moro
- Department Name
- Neuropsichiatria Infantile
- Contact Person Name
- Emilia Matera
- Contact Person Email
- emilia.matera@uniba.it
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- Pediatric Neurology
- Contact Person Name
- Renzo Guerrini
- Contact Person Email
- renzo.guerrini@meyer.it
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 and multiple operational trial support activities (codes listed: 1,11,12,13,15 etc.)
- Name
- IQVIA Limited
- Responsibilities
- Clinical trial operational support (code 8)
Third parties
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Codes: 1,11,12,13,15 (Vendor Management),2,5,8","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"IMP packaging, labelling, storage and distribution (code 15)","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"BioStatistics, Medical Data Review, and DSMB (code 15), other duties codes: 10,6","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Long term storage of study samples (code 15)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/review; Cardiac Safety Services for PPD (code 15)","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Central lab services (code 4)","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"efgartigimod immunogenicity (ADA and Nab) analysis (code 15), code 4","organisation_type":"Industry"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Bioanalytical Lab for PH20 immunogenicity analysis (ADA and NAb) (code 15)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Clinical trial support (code 8)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"Electronic clinical outcomes systems (code 3)","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH (Weil Am Rhein)","duties_or_roles":"Labelling, QP release, storage and distribution (code 15)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ARGX-113
- Active Substance
- Efgartigimod alfa
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous infusion
- Route
- Intravenous infusion
- Authorisation Status
- Not authorised
- Orphan Designation
- Yes
- Frequency
- 4 once-weekly infusions per treatment period (as described in protocol)
- Maximum Dose
- max daily dose 1200 mg
- Investigational Product Name
- Vyvgart 20 mg/mL concentrate for solution for infusion
- Active Substance
- Efgartigimod alfa
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous infusion
- Route
- Intravenous infusion
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Frequency
- 4 once-weekly infusions per treatment period (as described in protocol)
- Maximum Dose
- max daily dose 1200 mg
- Investigational Product Name
- Vyvgart 1 000 mg solution for injection
- Active Substance
- Efgartigimod alfa
- Modality
- Monoclonal antibody
- Routes Of Administration
- Subcutaneous injection (with rHuPH20 in some presentations)
- Route
- Subcutaneous injection
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Frequency
- Injections (per protocol: 4 once-weekly injections per treatment period)
- Maximum Dose
- max daily dose 1000 mg
- Combination Treatment
- Yes
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