E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
Miastenia Gravis Generalizzata |
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E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis in patients aged 2 to 18 years who have generalized Muscle Weakness |
Miastenia gravis in pazienti di età compresa tra 2 e 18 anni che presentano debolezza muscolare generalizzata |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of efgartigimod e.v. |
Valutare la sicurezza e la tollerabilità di efgartigimod IV |
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E.2.2 | Secondary objectives of the trial |
To evaluate the immunogenicity of efgartigimod IV |
valutare la immunogenicità di efgartigimod e.v. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The participant completed ARGX-113-2006, defined as: a. The participant reached EoT in trial ARGX-113-2006 and agreed to participate in the ARGX-113-2008 trial. b. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 trial's timeframe (ie, between TPnV1 to IPnV4). 2. Either the participant or the participant's legally acceptable representative (LAR) can understand the requirements of the trial and provide written informed consent/assent (including consent/assent for the use and disclosure of research-related health information), and demonstrates a willingness and ability to comply with the trial protocol procedures (including attending the required trial visits). 3. Contraceptive use by males and females must be consistent with local regulations for those participating in clinical trials and: a. Male participants agree not to donate sperm from the time the informed consent form (ICF) is signed until the end of the trial. b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1 of the protocol ) must have a negative urine pregnancy test at trial entry (TP1V1 or IP0V1). The contraceptive requirements for male participants are described in Section 10.4.2.2. The contraceptive requirements for FAOCBP are described in Section 10.4.2.1. |
1. Il partecipante ha completato ARGX-113-2006, definito come: a. Il partecipante ha raggiunto la fine della sperimentazione (End of Trial, [EoT]) nella sperimentazione ARGX-113-2006 e ha acconsentito a partecipare alla sperimentazione ARGX-113-2008. b. Il partecipante è idoneo al ritrattamento nella sperimentazione ARGX-113-2006, ma non può completare un periodo di trattamento (Treatment Period, [TP]) e le visite dell’IP richieste entro il periodo di tempo della sperimentazione ARGX-113-2006 (ovvero, tra TPnV1 e IPnV4). 2. Il partecipante o il rappresentante legale (Legally Acceptable Representative, [LAR]) del partecipante è in grado di comprendere i requisiti della sperimentazione e fornire un consenso/assenso informato scritto (compreso il consenso/l’assenso per l’utilizzo e la divulgazione di informazioni sanitarie correlate alla ricerca), e dimostra una volontà e capacità di attenersi alle procedure del protocollo di sperimentazione (tra cui presentarsi alle visite previste della sperimentazione). 3. L’uso di contraccettivi da parte di uomini e donne deve essere coerente con le normative locali per coloro che partecipano a sperimentazioni cliniche e: a. I partecipanti di sesso maschile acconsentono a non donare sperma dal momento in cui viene firmato il modulo di consenso informato (Informed Consent Form, [ICF]) fino alla fine della sperimentazione. b. Le adolescenti di sesso femminile in età fertile (Female adolescents of childbearing potential, [FAOCBP]; definite nella Sezione 10.4.1.1 del protocollo) devono presentare un test di gravidanza sulle urine negativo all’ingresso nella sperimentazione (TP1V1 o IP0V1). I requisiti contraccettivi per i partecipanti di sesso maschile sono descritti nella Sezione 10.4.2.2. I requisiti contraccettivi per le FAOCBP sono descritti nella Sezione 10.4.2.1. |
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E.4 | Principal exclusion criteria |
Participants are excluded from the trial if any of the following criteria apply: 1. FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of investigational medicinal product (IMP) 2. Discontinued early from ARGX-113-2006 treatment due to: pregnancy, receiving prohibited medication, participating in another trial with an investigational product, or the occurrence of a life-threatening or an investigational medicinal product-related AE, as assessed by the investigator 3. A known hypersensitivity reaction to efgartigimod or any of its excipients 4. Received a live-attenuated vaccine fewer than 4 weeks before trial entry. 5. Any of the following medical conditions: a. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at trial entry b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of gMG or put the participant at undue risk |
I partecipanti saranno esclusi dalla sperimentazione se rientreranno in uno qualsiasi dei seguenti criteri: 1. FAOCBP: Gravidanza o allattamento, oppure la partecipante intende rimanere incinta durante la sperimentazione o entro 90 giorni dopo l’ultima dose del medicinale sperimentale (Investigational Medicinal Product, [IMP]) 2. Ritirati anticipatamente dal trattamento previsto nella sperimentazione ARGX-113-2006 a causa di: gravidanza, assunzione di farmaci proibiti, partecipazione a un’altra sperimentazione con un prodotto sperimentale o insorgenza di un EA potenzialmente letale o correlato a un medicinale sperimentale, valutato dallo sperimentatore 3. Una nota reazione di ipersensibilità a efgartigimod o a uno qualsiasi dei suoi eccipienti 4. Hanno ricevuto un vaccino vivo attenuato meno di 4 settimane prima dell’ingresso nella sperimentazione 5. Qualsiasi delle seguenti condizioni mediche: a. Infezione batterica, virale o fungina non controllata, cronica, clinicamente significativa all’ingresso nella sperimentazione b. Qualsiasi altra malattia autoimmune nota che, a giudizio dello sperimentatore, interferirebbe con una valutazione accurata dei sintomi clinici di miastenia gravis generalizzata (Generalized Myasthenia Gravis, [gMG]) o esporrebbe il partecipante a un rischio inopportuno |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Incidence and severity of adverse events (AEs) - Incidence and severity of serious adverse events (SAEs) - Incidence and severity of adverse events of special interest (AESIs) - Changes in laboratory test results, vital signs, height and weight, and electrocardiogram (ECG) results |
- Incidenza e gravità degli eventi avversi (EA) - Incidenza e gravità degli eventi avversi seri (Serious Adverse Event, [SAE]) - Incidenza e gravità di eventi avversi di particolare interesse (Adverse Events of Special Interest, [AESI]) - Variazioni nei risultati dei test di laboratorio, nei segni vitali, nell’altezza e nel peso e nei risultati dell’elettrocardiogramma (ECG) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Once a month |
Una volta al mese |
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E.5.2 | Secondary end point(s) |
Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod |
Incidenza e prevalenza di anticorpi anti-farmaco (Anti-Drug Antibody, [ADA]) contro efgartigimod |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
No specific timepoints, during the entire study |
Nessun momento definito specifico, durante l’intero studio |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
- Tolerability - Immunogenicity |
- Tollerabilità - Immunogenicità |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Austria |
Belgium |
Canada |
France |
Georgia |
Germany |
Italy |
Netherlands |
Poland |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
The end of the trial is defined as the date of the last visit of the last participant in the ARGX-113-2008 trial. A participant is considered to have completed the trial if he/she completed the end of trial (EoT) visit and safety follow-up (SFU) visit |
La fine della sperimentazione è definita come la data dell’ultima visita dell’ultimo partecipante alla sperimentazione ARGX-113-2008. Si ritiene che un partecipante abbia completato la sperimentazione se ha completato la visita di fine sperimentazione (EoT) e la visita di follow-up di sicurezza (Safety Follow-Up, [SFU]) |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |