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 | 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) |
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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 |
1. Exploratory genomic substudy
During this study, subjects will also have the option of providing
additional informed consent for exploratory DNA and RNA analyses.
Participation in this additional portion of the study is optional and does
not preclude subject's eligibility for participation in the main study.
2. PK/PD substudy
During this study, subjects will also have the option of providing
additional informed consent for a PK/PD substudy (early blood
sampling) at selected study sites. Participation in this additional portion
of the study is optional and does not preclude subject's eligibility for
participation in the main study. The additional PK/PD blood samples
must not be collected if the subject has not consented to participate in
this exploratory 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) |
|
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 |
|
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 |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |