E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Hereditary angioedema (HAE) is a condition characterized by painful, recurring attacks of swelling in parts of the body including the face, throat, hands, feet, abdomen.
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10019860 |
E.1.2 | Term | Hereditary angioedema |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 |
The main objective is to evaluate the efficacy of sucutaneous administration of CSL312 as prophylaxis to prevent hereditary angioedema attacks in subjects with hereditry angioedema
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are: 1. To characterize the clinical efficacy of subcutaneous CSL312 in the prophylactic treatment of hereditary angioedema 2. to evaluate the safety of subcutaneous CSL312 in the prophylactic treatment of hereditary angioedema
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Male or female ≥ 12 years of age; diagnosed with clinically confirmed C1-INH hereditry angioedema; experience ≥ 3 attacks during the 3 months before Screening
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E.4 | Principal exclusion criteria |
Concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticarial or hereditary angioedema type 3
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E.5 End points |
E.5.1 | Primary end point(s) |
Time-normalized number of hereditary angioedema (HAE) attacks during treatment period
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Reduction in the HAE attack rate during the treatment period compared to the run-in period 2. Time-normalized number of attacks requiring on-demand Treatment 3. Time-normalized number of moderate and/or severe HAE attacks 4. Time-normalized number of HAE attacks at various timepoints during the treatment period 5. Percent reduction in the time-normalized number of HAE attacks between CSL312 and Placebo 6. Subject's Global Assessment of Response to Therapy (SGART) 7. Number of subjects with adverse events, adverse events of special interest, serious adverse events, CSL312-induced anti-CSL312 antibodies, clinically significant abnormalities in laboratory assessments 8. Percent of subjects with Adverse events, adverse events of special interest, serious adverse events, CSL312-induced anti-CSL312 antibodies, clinically significant abnormalities in laboratory assessments
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
to 1: up to 6 months to 2: 6 months, and first 3 months and second 3 months to 3: 6 months, and first 3 months and second 3 months to 4: First 3 months and second 3 months and 6 months to 5: 6 months, and first 3 months and second 3 months to 6: Up to 6 months to 7 and 8: Up to 8 months
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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 | 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 | 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 | 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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 16 |
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 |
Canada |
Germany |
Hungary |
Israel |
Italy |
Netherlands |
Spain |
United States |
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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
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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 | |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 19 |