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 |
Primary immune thrombocytopenia (ITP) |
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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 | 19.1 |
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) |
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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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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 |
Exploratory genomic substudy |
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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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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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E.5 End points |
E.5.1 | Primary end point(s) |
Number of Subjects experiencing at least one Treatment Emergent Adverse Event (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 |
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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 |
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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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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 | 2 |
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 | 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 |
Australia |
Belgium |
Bulgaria |
Czech Republic |
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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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 |