E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
hereditary angioedema, HAE (in adult patients with C1-esterase-inhibitor deficiency) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019860 |
E.1.2 | Term | Hereditary angioedema |
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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 |
The primary objective is to explore clinical safety with self-treatment of acute HAE attacks with subcutaneous injections of icatibant. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are to determine local tolerability, convenience, and clinical efficacy of self-treatment of HAE attacks with subcutaneous injections of icatibant. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Each patient must meet the following criteria to be enrolled in this study: • Males and females ≥18 years of age at the time of informed consent • Documented diagnosis of HAE Type I or II based on ALL of the following criteria: • Family and/or medical history • Characteristic attack manifestations, recurrent attacks • Historical functional C1-INH <50% normal values • Women of childbearing potential must use consistently and correctly a highly effective, adequate method of birth control (failure rate less than 1% per year) - sexual abstinence or have a vasectomised partner during the duration of the study. Hormonal contraception can be continued if verified by a physician that it doesn’t affect the course of HAE attacks. • Mental and physical condition allowing patients to complete baseline assessment, to self-administer icatibant and to follow other study procedures. • Ability to provide signed written informed consent after all aspects of the study have been explained and discussed with the patient.
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria will be excluded from the study: • Participation in a clinical trial of another investigational medicinal product within the past month (except a previous icatibant study). • Diagnosis of angioedema other than Type I or Type II HAE. • Evidence of symptomatic coronary artery disease based on medical history, in particular, unstable angina pectoris or severe coronary heart disease. • Congestive heart failure (NYHA Class 3 and 4). • Stroke within the past 6 months. • Treatment with angiotensin converting enzyme (ACE) inhibitor. • Pregnancy and/or breast-feeding. • In the opinion of the investigator: mental condition rendering the patient unable to understand the nature, scope and possible consequences of the study. • In the opinion of the investigator: unlikely to comply with the protocol, for example, uncooperative attitude, inability to return for follow-up visits, or unlikely to complete the study for any reason. • In the opinion of the investigator: inability to manage study medication or selfadministration of an injection. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is to show clinical safety with self-treatment of acute HAE attacks with subcutaneous injections of icatibant. |
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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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
Convenience of Self-administration |
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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) | 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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
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 |
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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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Screening and enrollment of patients will continue until approximately 150 patients have been enrolled in the trial or until the sponsor decides to discontinue the study.
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |