E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Explore the effect of rC1INH in patients with HAE in preventing the occurence of acute HAE attacks. |
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MedDRA Classification |
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 occurrence of HAE attacks under prophylactic administration of rC1INH (50 U/kg, once a week) |
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E.2.2 | Secondary objectives of the trial |
To evaluate the PK parameters, safety and immunogenicity on repeated administration of rC1INH |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Aged at least 18 years • Signed written informed consent • Confirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50 % of normal, and/or proven HAE mutation in C1INH gene • The occurence of an HAE attack at least every fortnight |
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E.4 | Principal exclusion criteria |
• A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs. • A history of allergic reactions to C1INH products or rabbit protein. • Any reported SAE related to eventual previous rC1INH administration (withdrawal criterium). • Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia) • A diagnosis of acquired C1INH deficiency (exclude patients with AAE). • Women of child bearing potential, who are (or intent to become) pregnant or breast-feeding. • Participation in another clinical study in the last month prior to this study • Treatment with C1INH concentrates or any blood or plasma-derived material (e.g. fresh-frozen plasma) within 7 days prior to treatment with rC1INH. • Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis. • Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives. • Treatment with anticoagulants (e.g) heparin or warfarin) in the last 3 days preceding treatment with rC1INH. • Any changes since screening that would exclude subject based on above exclusion criteria.
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E.5 End points |
E.5.1 | Primary end point(s) |
Frequency of breakthrough attacks of HAE. |
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
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 | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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 | 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
| Yes |
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 | 5 |
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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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 | 6 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |