E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary immune thrombocytopenia (ITP) |
Trombocitopenia immune primaria |
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E.1.1.1 | Medical condition in easily understood language |
Primary immune thrombocytopenia (ITP) |
Trombocitopenia immune primaria |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
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 | 10043554 |
E.1.2 | Term | Thrombocytopenia |
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 UCB7665 administered by subcutaneous (sc) infusion in patients with immune thrombocytopenia (ITP) |
Valutare la sicurezza e la tollerabilit¿ di UCB7665 somministrato tramite sc nei soggetti con ITP. |
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E.2.2 | Secondary objectives of the trial |
¿ To assess the clinical efficacy of UCB7665 as measured by the change in platelet count ¿ To assess the pharmacodynamic (PD) effect of UCB7665 as measured by the change in total immunoglobulin G (IgG) concentration in serum
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- valutare l¿efficacia clinica di UCB7665 in base alla misurazione del cambiamento della conta piastrinica - valutare l¿effetto di farmacodinamica (pharmacodynamic, PD) di UCB7665 in base alla misurazione del cambiamento nelle concentrazioni di IgG totale nel siero. |
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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 |
Pharmacogenomics Version: - Date: 15/02/2017 Title: Exploratory genomic substudy Objectives: -
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Farmacogenomica Versione: - Data: 15/02/2017 Titolo: Sottostudio Genomico Esplorativo Obiettivi: -
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E.3 | Principal inclusion criteria |
• Subject has a diagnosis of primary immune thrombocytopenia for a minimum of 3 months prior to Screening Visit • Subject has a platelet count <30x10^9/L at Screening and <35x10^9/L at Baseline (Visit 2) • Subject has a current or history of a peripheral blood smear consistent with ITP • Subject has adequate peripheral venous access • Subject has responded to previous ITP therapy (according to the judgment of the investigator)
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- Il soggetto presenta una diagnosi di ITP primaria da almeno 3 anni prima della Visita di screening. - Il soggetto presenta una conta piastrinica <30x109/l allo screening e <35x109/l al basale (Visita 2). - Il soggetto presenta uno striscio di sangue periferico coerente con ITP. - Il soggetto deve avere disponibilità di un accesso venoso periferico adeguato. - Il soggetto ha risposto a una precedente terapia per l’ITP (in base al giudizio dello sperimentatore). |
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E.4 | Principal exclusion criteria |
• Subject has an immunoglobulin G (IgG) level =6g/L at Screening Visit • Subject has a partial thromboplastin time (PTT) =1.5x upper limit of normal (ULN) or International Normalized Ratio (INR) =1.5 at Screening Visit • Subject has renal and/or liver impairment • Subject has planned an elective surgical procedure in the coming 6 months • Subject has evidence of a secondary cause of immune thrombocytopenia • Subject has a history of clinically relevant ongoing chronic infections • Subject has a family history of primary immunodeficiency • Subject has a clinically relevant active infection or has had a serious infection within 6 weeks prior to the first dose of IMP • Subject has a history of known inflammatory bowel disease, diverticular disease or has a history of confirmed, duodenal, gastric or esophageal ulceration in the past 6 months • Subject has experienced a clinically symptomatic gastrointestinal bleed (positive hemoccult tests without any signs and symptoms of gastrointestinal bleedings will not be considered as "clinically symptomatic") in the last 6 months prior to Screening Visit and/or has current gastritis or esophagitis and/or has a known risk for clinical relevant gastrointestinal bleeding beyond ITP • Subject has a medical history of thrombosis within the past 5 years or a history of thrombosis with unknown cause at any time or a significant known risk for thrombosis • Subject has a history of coagulopathy disorders other than ITP • Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP • Subject has had prior treatment with rituximab in the 6 months prior to the Baseline • Subject has not completed the washout period for the immune suppressants, biologics and other therapies
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- Il soggetto presenta un livello di IgG =6 g/l alla Visita di screening. - Il soggetto presenta un tempo di tromboplastina parziale (partial thromboplastin time, PTT) =1,5x limite superiore della normalità (upper limit of normal, ULN) o un rapporto internazionale normalizzato (International Normalized Ratio, INR)=1,5 alla Visita di screening. - Il soggetto presenta disfunzione renale e/o epatica. - Il soggetto ha programmato una procedura chirurgica elettiva nei prossimi 6 mesi. -Il soggetto presenta evidenza di una causa secondaria della porpora trombocitopenica immune da pregressa anamnesi medica. -Il soggetto presenta una storia di infezioni croniche in corso clinicamente rilevanti, tra cui, ma senza limitazioni, il virus dell’immunodeficienza umana .-Il soggetto presenta una storia familiare di immunodeficienza primaria. - Il soggetto presenta un’infezione attiva clinicamente rilevante oppure presenta un’infezione grave nelle 6 settimane precedenti la prima dose dell’IMP .- Il soggetto ha una storia di nota malattia infiammatoria dell’intestino, malattia diverticolare o ha una storia di ulcera gastrica, duodenale o esofagea confermata negli ultimi 6 mesi. -Il soggetto ha manifestato un sanguinamento gastrointestinale clinicamente sintomatico (test del sangue occulto nelle feci positive senza segni e sintomi di sanguinamento gastrointestinale non sarà considerato come “clinicamente sintomatico”) nei 6 mesi precedenti la Visita di screening e/o presenta una gastrite o esofagite in corso e/o un rischio noto di sanguinamento gastrointestinale di rilevanza clinica oltre ITP - Il soggetto presenta un’anamnesi medica di trombosi negli ultimi 5 anni o una storia di trombosi con causa sconosciuta in qualsiasi momento o un rischio noto significativo di trombosi. -Il soggetto ha una storia di coagulopatie diverse da ITP. .-Il soggetto ha ricevuto un vaccino vivo nelle 8 settimane precedenti la Visita basale; oppure intende sottoporsi a un vaccino vivo durante lo studio o nelle 7 settimane successive la dose finale dell’IMP. -Il soggetto è stato sottoposto a pregresso trattamento con rituximab nei 6 mesi precedenti la Visita basale. - Il soggetto non ha completato il periodo di wash-out per gli immunosoppressori, I biologici e altre terapie.
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of Subjects experiencing at least one Treatment Emergent Adverse Event (TEAE) |
Numero di soggetti che hanno avuto esperienza di eventi avversi successivi al trattamento (TEAE) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From Visit2 (Week1) until End of Study Visit |
dalla Visita 2 (Settimana 1) fino alla Visita di Conclusione studio |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability |
Tollerabilit¿ |
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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) | No |
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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Dose differente dello stesso prodotto. |
Different dose of the same product
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E.8.2.4 | Number of treatment arms in the trial | 5 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 33 |
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 |
Australia |
Belgium |
Bulgaria |
Czechia |
France |
Georgia |
Germany |
Italy |
Lithuania |
Moldova, Republic of |
Poland |
Romania |
Spain |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | No |
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
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Last Subject Last Visit |
"LVLS" |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 7 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |