E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
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 assess the safety and efficacy of DX-88 in the treatment of acute attacks of Hereditary Angioedema | |
E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria | 1.Age 10 years or older 2.Documented diagnosis of HAE, Type I or II Diagnosis must meet the following criteria: •Documented clinical history consistent with HAE, •Documented reduction of either functional or antigenic C1-INH (below the lower limit of normal as defined by the laboratory performing the test), •Documented reduction of C4 (below the lower limit of normal as defined by the laboratory performing the test), and •Age of reported onset of first symptoms ≤ 25 years OR documented C1q level at or above the lower limit of normal (as defined by the laboratory performing the test) 3.Executed informed consent 4.Enrollment Visit: An acute attack of HAE having at least one symptom complex with a severity assessment of moderate or severe 5.Enrollment Visit: Presentation for treatment at the site within 8 hours of patient recognition of moderate to severe symptoms in an acute attack of HAE | |
E.4 | Principal exclusion criteria | 1.Receipt of an investigational drug or device, other than DX-88, within 30 days prior to study enrollment 2.Treatment with non-investigational C1-INH within 7 days prior to study enrollment 3.Diagnosis of acquired angioedema AAE, estrogen dependent angioedema and/or drug induced angioedema (including angiotensin-converting enzyme inhibitor induced angioedema) 4.Pregnancy or breast feeding 5.Patients who have received DX-88 (in EDEMA2 or any other studies with DX-88) within 7 days of presentation for dosing in the Double-blind Phase | |
E.5 End points |
E.5.1 | Primary end point(s) | Treatment Outcome Score (TOS) at 4 hours based on the severity assessment of symptom complex(es) as determined by patient | |
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 | Information not present in EudraCT |
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) | 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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description | first phase is double blind, second phase is open label | |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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 | |
E.8.7 | Trial has a data monitoring committee | Yes |
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 | Last patient last visit (21 treatments + 90 days follow-up) | |
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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |