E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Myasthenia Gravis |
Miastenia Grave |
|
E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis in patients who have generalized Muscle Weakness |
Miastenia grave in pazienti con debolezza muscolare generalizzata |
|
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 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028423 |
E.1.2 | Term | Myasthenia gravis-like syndrome |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10071942 |
E.1.2 | Term | Myasthenia gravis and related conditions |
E.1.2 | System Organ Class | 100000004859 |
|
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 long-term safety and tolerability of ARGX-113 in acetylcholine receptor antibody (AChR-Ab) seropositive patients. |
Valutare la tollerabilità e la sicurezza a lungo termine di ARGX-113 nei pazienti sieropositivi agli anticorpi contro il recettore dell’acetilcolina (AChR-Ab). |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the long-term safety and tolerability of ARGX-113 in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients). |
Valutare la tollerabilità e la sicurezza a lungo termine di ARGX-113 nella popolazione complessiva (pazienti sieropositivi e pazienti sieronegativi agli AChR-Ab). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients will roll over in this trial only if they meet all of the following criteria:
1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and can comply with the trial protocol procedures. 2. Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.
Other more specific inclusion criteria are further defined in the protocol. |
I pazienti saranno sottoposti a questa sperimentazione solo se soddisfano tutti i seguenti criteri:
1. Pazienti in grado di comprendere i requisiti della sperimentazione, fornire il consenso informato scritto e rispettare le procedure del protocollo sperimentale. 2. Pazienti che hanno partecipato alla sperimentazione ARGX-113-1704 e sono idonei al roll over, come specificato nel protocollo.
Altri criteri di inclusione più specifici sono ulteriormente definiti nel protocollo. |
|
E.4 | Principal exclusion criteria |
Patients will not roll over in this trial if they meet any of the following criteria:
1. Patients who discontinued early from trial ARGX-113-1704 or patients who discontinued early from randomized treatment for rescue or pregnancy reasons or an (S)AE that might jeopardize the safety of the patient in that trial. 2. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women of childbearing potential (DEFINITION OF TERMS) should have a negative urine pregnancy test at SEB. 3. Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing. 4. Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.
Other more specific exclusion criteria are further defined in the protocol. |
I pazienti non saranno sottoposti a questa sperimentazione se soddisfano uno dei seguenti criteri:
1. Pazienti che hanno interrotto anticipatamente la sperimentazione ARGX-113-1704 o il trattamento randomizzato per interventi di soccorso, per gravidanza o per un (S)AE che avrebbe potuto pregiudicare la sicurezza del paziente in quella sperimentazione. 2. Donne in gravidanza o in allattamento o che intendono avviare una gravidanza durante la sperimentazione o entro 90 giorni dopo l’ultima somministrazione della dose. Le donne in età fertile (DEFINIZIONE DEI TERMINI) devono presentare un test di gravidanza sulle urine negativo al SEB. 3. I pazienti di sesso maschile sessualmente attivi che non intendono utilizzare metodi contraccettivi efficaci (riportati sopra) durante la sperimentazione o entro 90 giorni dalla somministrazione dell’ultima dose oppure pazienti di sesso maschile che prevedono di donare lo sperma durante la sperimentazione o entro 90 giorni dalla somministrazione dell’ultima dose. 4. Sieropositività nota del paziente al virus dell’epatite B (HBV), al virus dell’epatite C (HCV) o al virus dell’immunodeficienza umana (HIV).
Altri criteri di esclusione più specifici sono ulteriormente definiti nel protocollo. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence and severity of AEs, serious adverse events (SAEs), vital signs, electrocardiogram (ECG) and laboratory assessments over the duration of the trial in AChR-Ab seropositive patients. |
Incidenza e gravità di AE e SAE, segni vitali, elettrocardiogramma (ECG) e valutazioni di laboratorio nell’arco di tutta la durata dello studio nei pazienti sieropositivi agli AChR-Ab. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to maximum 3 years |
Massimo 3 anni |
|
E.5.2 | Secondary end point(s) |
Incidence and severity of AEs, SAEs, vital signs, ECG and laboratory assessments over the duration of the trial in the overall population (AChR-Ab seropositive patients and AChR-Ab seronegative patients). |
Incidenza e gravità di AE e SAE, segni vitali, ECG e valutazioni di laboratorio nell’arco della durata dello studio nella popolazione complessiva (pazienti sieropositivi e pazienti sieronegativi agli AChR-Ab). |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
over the duration of the trial (up to maximum 3 years) |
per la durata dello studio (al massimo 3 anni) |
|
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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
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 |
Tollerabilità |
|
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) | No |
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 | 22 |
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 |
Canada |
Japan |
Russian Federation |
Serbia |
United States |
Belgium |
Czechia |
Denmark |
France |
Germany |
Hungary |
Italy |
Netherlands |
Poland |
Sweden |
United Kingdom |
|
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
|
|
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 | 1 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |