E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Systemic lupus erythematosis (SLE) |
Lupus Eritematoso Sistemico (LES) |
|
E.1.1.1 | Medical condition in easily understood language |
Systemic lupus erythematosis (SLE) |
Lupus Eritematoso Sistemico (LES) |
|
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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10042944 |
E.1.2 | Term | Systemic lupus erythematosis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To evaluate the efficacy of belimumab administered SC in adult subjects with SLE. • To evaluate the safety and tolerability of belimumab administered SC in adult subjects with SLE. |
• Valutare l’efficacia di belimumab somministrato per via SC in soggetti adulti affetti da LES. • Valutare la sicurezza e la tollerabilità di belimumab somministrato per via SC in soggetti adulti affetti da LES. |
|
E.2.2 | Secondary objectives of the trial |
Not applicable |
Non applicabile |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
PHARMACOGENETIC: Vers:Emedn.3 Date:2011/11/17 Title:Pharmacogenetic Research Objectives:Relationship between genetic variants and the pharmacokinetics of
belimumab.
• Relationship between genetic variants and safety and/or tolerability of
belimumab.
• Relationship between genetic variants and efficacy of belimumab.
|
FARMACOGENETICA: Vers:Emedn.3 Data:2011/11/17 Titolo:Ricerca Farmacogenetica Obiettivi:-Relazione tra le varianti genetiche e la farmacocinetica di belimumab.
-Relazione tra le varianti genetiche e la sicurezza e/o tollerabilità di belimumab.
-Relazione tra le varianti genetiche e l’efficacia di belimumab.
|
|
E.3 | Principal inclusion criteria |
1. At least 18 years of age. 2. Clinical diagnosis of Systemic Lupus Erythematosus (SLE) by ACR criteria. 3. Active SLE disease. 4. Autoantibody-positive. 5. On stable SLE treatment regimen which may include corticosteroids (for example, prednisone), antimalarial (for example, hydroxychloroquine) and/or immunosuppressants (for example, azathioprine, methotrexate, mycophenolate, etc.) |
1.Avere almeno 18 anni di età; 2.Avere una diagnosi clinica di LES secondo i criteri dell’ACR;3.Essere affetti da LES attivo; 4.Avere risultati positivi al test degli autoanticorpi. 5.Essere sotto un regime di trattamento per il LES stabile che potrebbero includere corticosteroidi (ad es. prednisone), antimalarici (ad es. idrossiclorochina) e/o immunosoppressori (ad es. metotrexato, azatioprina, micofenolato ecc.). |
|
E.4 | Principal exclusion criteria |
1. Pregnant or nursing. 2. Have received treatment with an B cell targeted therapy (for example, rituximab or belimumab). 3. Have received treatment an investigational biological agent in the past year. 4. Have received IV cyclophosphamide within 90 days of Day 0. 5. Have severe active lupus kidney disease. 6. Have severe active central nervous system (CNS) lupus. 7. Have required management of acute or chronic infections within the past 60 days. 8. Have current drug or alcohol abuse or dependence. 9. Have a positive test for HIV, hepatitis B, or hepatitis C. 10. Have a history of hypersensitivity reactions to contrast agents or biological medicines. |
1. Gravidanza e Allattamento; 2. Hanno ricevuto in qualsiasi momento una qualsiasi terapia con azione sulle cellule B (ad es. rituximab e belimumab); 3. Hanno ricevuto una terapia con agente biologico di sperimentazione nell’anno precedente; 4. Hanno ricevuto Ciclofosfamide per via endovenosa (EV) entro 90 giorni dal Giorno 0. 5. Sono affetti da patologia renale grave di lupus; 6. Sono affetti da lupus grave attivo a carico del sistema nervoso centrale (SNC); 7. Hanno avuto bisogno di un trattamento di infezioni acute o croniche entro i precedenti 60 giorni dal Giorno 0. 8. Mostrano attualmente un abuso o dipendenza da droga o alcool; 9. Hanno un test positivo per l’HIV, l’epatice B o l’epatite C. 10. Hanno una storia anamnestica di reazione anafilattica alla somministrazione parenterale di agenti di contrasto o farmaci biologici. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary Outcome Measure: SLE Responder Index (SRI) response rate; [Time Frame: Baseline, 52 Weeks]. A patient that has an SRI response has all 3 of the following: - >=4 point reduction from baseline in SELENA SLEDAI score, AND - No worsening (increase of <0.30 points from baseline) in Physician's Global Assessment (PGA), AND - No new BILAG A organ domain score or 2 new BILAG B organ domain scores compared with baseline at the time of assessment (ie, at Week 52). |
Misura dell’end-point primario: -percentuale dell’indice di risposta LSE secondo l’SRI (Periodo: Basale alla Settimana 52). -un paziente che ha una risposta SRI secondo le 3 seguenti: • >=4 punti di riduzione dal basale sul punteggio SELENA SLEDAI, E • Nessun peggioramento (aumento di < 0,30 punti dal basale) nella PGA (Physician’s Global Assessment, Valutazione Globale del Medico), E • Nessun nuovo punteggio BILAG A di dominio d’organo o 2 nuovi punteggi BILAG B di dominio d’organo in confronto al basale nel momento della valutazione (cioè alla Settimana 52). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
baseline 52 weeks |
basale alla settimana 52 |
|
E.5.2 | Secondary end point(s) |
Secondary Outcome Measures: • Time to first severe flare (SLE Flare Index); [Time Frame: Baseline to 52 weeks] • Reduction in prednisone dose. Percent of subjects whose average prednisone dose has been reduced by >= 25% from baseline to <=7.5 mg/day during Weeks 40 through 52 in subjects receiving greater than 7.5 mg/day at baseline ; [Time Frame: Baseline, Weeks 40-52] |
Misure dell’end-point secondario: 1. Tempo trascorso fino alla prima riacutizzazione grave (misurato secondo l’ SLE Flare Index))(Periodo: Basale alla Settimana 52). 2. Riduzione della dose di prednisone.Percentuale di soggetti la cui dose media di prednisone è stata ridotta del >= 25% dal basale a <= 7,5 mg/die durante le settimane 40-52 in soggetti che ricevevano quantità superiori a 7,5 mg/die al basale. (Periodo: Basale, settimane 40-52) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1.Time Frame: Baseline to 52 weeks; 2.Time Frame: Baseline, Weeks 40-52. |
1.Periodo: Basale alla Settimana 52; 2.Periodo: Basale, settimane 40-52 |
|
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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 56 |
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 | No |
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 |
Argentina |
Brazil |
Canada |
Chile |
Colombia |
India |
Malaysia |
Mexico |
Peru |
Philippines |
Russian Federation |
Singapore |
Taiwan |
Thailand |
Ukraine |
United States |
|
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 | 0 |
E.8.9.1 | In the Member State concerned months | 28 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 34 |
E.8.9.2 | In all countries concerned by the trial days | 0 |