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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-506159-12-00 | EU Trial (CTIS) Number |
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The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to <18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study.
The participants will be in the study for up to 14 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Efgartigimod PH20 SC | Experimental | Participants aged 12 to <18 years receiving efgartigimod PH20 SC treatment |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Biological | Subcutaneous injections |
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| Measure | Description | Time Frame |
|---|---|---|
| Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL) | Up to 12 weeks | |
| Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd) | Up to 12 weeks | |
| Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis | Up to 12 weeks | |
| Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis | Up to 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events (AEs) | Up to 14 weeks | |
| Severity of adverse events (AEs) | Up to 14 weeks | |
| Incidence of serious adverse events (SAEs) |
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Inclusion Criteria:
Exclusion Criteria:
The complete list of exclusion criteria can be found in the protocol.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sabine Coppieters, MD | Contact | 857-350-4834 | clinicaltrials@argenx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Carolinas HealthCare System Neurosciences Institute - Neurology | Recruiting | Charlotte | North Carolina | 28207 | United States |
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| Up to 14 weeks |
| Severity of serious adverse events (SAEs) | Up to 14 weeks |
| Incidence of adverse events of special interest (AESI) | Up to 14 weeks |
| Severity of adverse events of special interest (AESI) | Up to 14 weeks |
| Efgartigimod serum concentrations | Up to 12 weeks |
| Absolute values of total Immunoglobulin G (IgG) from blood samples | Up to 12 weeks |
| Change from baseline values of total Immunoglobulin G (IgG) from blood samples | Up to 12 weeks |
| Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples | Up to 12 weeks |
| Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples | Up to 12 weeks |
| Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples | Up to 12 weeks |
| Percentage change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples | Up to 12 weeks |
| Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples | Up to 12 weeks |
| Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples | Up to 12 weeks |
| Incidence of antibodies against rHuPH20 in serum samples | Up to 12 weeks |
| Prevalence of antibodies against rHuPH20 in serum samples | Up to 12 weeks |
| Absolute value of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use | Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment) | Up to 12 weeks |
| Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use | Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment) | Up to 12 weeks |
| Absolute value of Quantitative Myasthenia Gravis (QMG) score | Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment) | Up to 12 weeks |
| Change from baseline of Quantitative Myasthenia Gravis (QMG) score | Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment) | Up to 12 weeks |
| Absolute value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score | Up to 12 weeks |
| Change from baseline value of Neuro-QoL Pediatric Fatigue Score | Up to 12 weeks |
| Change from baseline value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score | Up to 12 weeks |
| Change from baseline value of Clinical Global Impression of Improvement (CGI-I) | Up to 12 weeks |
| Changes in protective antibody titers to vaccines | Up to 12 weeks |
| Neurology Rare Disease Center | Recruiting | Denton | Texas | 76208 | United States |
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| University of Virginia | Recruiting | Charlottesville | Virginia | 22903 | United States |
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| UZ Gent | Recruiting | Ghent | 9000 | Belgium |
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| Childrens Hospital of Eastern Ontario | Recruiting | Ottawa | K1H 8L1 | Canada |
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| Fakultni nemocnice Brno | Recruiting | Brno | 613 00 | Czechia |
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| Fakultni nemocnice Ostrava | Recruiting | Ostrava | 708 00 | Czechia |
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| AP-HM- Hôpital de La Timone | Recruiting | Marseille | 13385 | France |
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| Universitätsklinikum Essen | Recruiting | Essen | 45147 | Germany |
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| Hadassah Medical Center- Ein Kerem | Recruiting | Jerusalem | 9112001 | Israel |
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| Schneider Children's Medical Center of Israel | Recruiting | Petah Tikvah | 4920235 | Israel |
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| Tel Aviv Sourasky Medical Center Ichilov | Recruiting | Tel Aviv | 6423906 | Israel |
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| Istituto G Gaslini Ospedale Pediatrico IRCCS | Recruiting | Genova | 16147 | Italy |
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| Leiden University Medical Center | Recruiting | Leiden | 2333 ZA | Netherlands |
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| Uniwersyteckie Centrum Kliniczne w Gdansku | Recruiting | Gdansk | 80-211 | Poland |
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| Neurologia Śląska Centrum Medyczne | Recruiting | Katowice | 40-689 | Poland |
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| Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM | Recruiting | Warsaw | 02-097 | Poland |
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| Hospital Universitari i Politecnic La Fe de Valencia | Recruiting | Valencia | 46026 | Spain |
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| Hôpital Nestlé | Recruiting | Lausanne | 1011 | Switzerland |
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| Oxford Children's Hospital | Recruiting | Oxford | OX3 9DU | United Kingdom |
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| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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