E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Antiphospholipid Syndrome |
Sindrome da Anticorpi Antifosfolipidi |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002817 |
E.1.2 | Term | Antiphospholipid syndrome |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic 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 belimumab treatment in patients with APS at 24 months of treatment |
valutare la sicurezza e tollerabilità del trattamento con belimumab nei pazienti con APS a 24 mesi di trattamento |
|
E.2.2 | Secondary objectives of the trial |
changes in antibodies titre |
variazioni nel titolo anticorpale |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Positive aPL profile Clinical features attributable to aPL that are resistant to warfarin and/or heparin: o Recurrent thrombosis despite ongoing anticoagulation and/or o Persistent thrombocytopenia and/or o Persistent autoimmune hemolytic anemia and/or o Cardiac valve disease and/or o Chronic skin ulcers and/or o Renal thrombotic microangiopathy and/or o Cognitive dysfunction with/without white matter changes |
Profilo aPL positivo Caratteristiche cliniche attribuibili ad aPL resistenti a warfarin e / o eparina: o Trombosi ricorrente nonostante l'anticoagulazione in corso e / o o Trombocitopenia persistente e / o o Anemia emolitica autoimmune persistente e / o o Malattia della valvola cardiaca e / o o ulcere cutanee croniche e / o o Microangiopatia trombotica renale e / o o Disfunzione cognitiva con / senza alterazioni della sostanza bianca |
|
E.4 | Principal exclusion criteria |
• >=4/11 American College of Rheumatology Classification Criteria for SLE • Acute thrombosis (arterial or venous acute thrombosis diagnosis less than 30 days before study screening) • History of stroke Acute or chronic pancreatitis • Pregnancy • Have a history of malignant neoplasm within the last 5 years except basal cell or squamous cell carcinoma of the skin treated with local resection only or carcinoma in situ of the uterine cervix treated locally and with no evidence of metastatic disease for 3 years • Have evidence of serious suicide risk including any history of suicidal behaviour in the last 6 months and/or any suicidal ideation in the last 2 months or who in the investigator's judgment, poses a significant suicide risk • Have a history of a primary immunodeficiency • Have a significant IgG deficiency (IgG level < 400 mg/dL) • Have an IgA deficiency (IgA level < 10 mg/dL) • Known active bacterial, viral fungal mycobacterial, or other infection • Infection history: • Currently on any suppressive therapy for a chronic infection (such as tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical mycobacteria) • Hospitalization for treatment of infection within 60 days of Day 0. • Use of parenteral (IV or IM) antibiotics (anti-bacterial, antiviral, anti-fungal, or antiparasitic agents) within 60 days of Day 0 • Have current drug or alcohol abuse or dependence, or a history of drug or alcohol abuse or dependence within 365 days prior to Day 0 • Have a historically positive HIV test or test positive at screening for HIV • Hepatitis status: • Serologic evidence of current or past Hepatitis B (HB) infection based on the results of testing for HBsAg and HBcAb as follows: • Patients positive for HBsAg or HBcAb are excluded • Positive test for Hepatitis C antibody • Have a history of an anaphylactic reaction to parenteral administration of contrast agents, human or murine proteins or monoclonal antibodies • Have any other clinically significant abnormal laboratory value in the opinion of the investigator • If Women of Child-Bearing Potential (WCBP) are included, please see special instructions below. |
•> = 4/11 Criteri di classificazione dell'American College of Rheumatology per il LES • Trombosi acuta (diagnosi di trombosi acuta arteriosa o venosa inferiore a 30 giorni prima dello screening dello studio) • Storia di ictus Pancreatite acuta o cronica • Gravidanza • Avere una storia di neoplasia maligna negli ultimi 5 anni ad eccezione delle cellule basali o carcinoma a cellule squamose della pelle trattata solo con resezione locale o carcinoma in situ della cervice uterina trattata localmente e senza evidenza di malattia metastatica per 3 anni • Avere prove di un grave rischio di suicidio, inclusa qualsiasi storia di comportamento suicidario in ultimi 6 mesi e / o qualsiasi idea suicida negli ultimi 2 mesi o chi nel giudizio dell'investigatore, pone un rischio di suicidio significativo • Avere una storia di immunodeficienza primaria • Presenta una significativa carenza di IgG (livello di IgG <400 mg / dL) • Ha un deficit di IgA (livello di IgA <10 mg / dL) • Nota infezione batterica attiva, micobatterica fungina virale o altra infezione • Storia dell'infezione: • Attualmente in terapia soppressiva per un'infezione cronica (come la tubercolosi, pneumocystis, citomegalovirus, virus herpes simplex, herpes zoster e atipico micobatteri) • Ricovero per il trattamento dell'infezione entro 60 giorni dal Giorno 0. • Uso di antibiotici parenterali (EV o IM) (antibatterici, antivirali, antifungini o antiparassitari agenti) entro 60 giorni dal Giorno 0 • Avere abuso o dipendenza da droghe o alcol, o una storia di droga o alcol abuso o dipendenza entro 365 giorni prima del giorno 0 • Avere un test HIV storicamente positivo o risultare positivo allo screening per l'HIV • Stato dell'epatite: • Evidenza sierologica di infezione da epatite B (HB) attuale o passata basata su risultati dei test per HBsAg e HBcAb come segue: • I pazienti positivi per HBsAg o HBcAb sono esclusi • Test positivo per l'anticorpo dell'epatite C. • Avere una storia di reazione anafilattica alla somministrazione parenterale di contrasto agenti, proteine ¿¿umane o murine o anticorpi monoclonali • Possedere qualsiasi altro valore di laboratorio anormale clinicamente significativo secondo il parere di investigatore • Se sono incluse le donne in età fertile (WCBP), vedere l'apposito istruzioni di seguito. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of Participants Experiencing Adverse Events [Time Frame: 104 weeks] |
Numero di partecipanti che hanno manifestato eventi avversi [lasso di tempo: 104 settimane] |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Antibodies titre |
Titolo anticorpale |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | 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 | No |
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) | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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 | 2 |
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 | 2 |
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 |