Phase 3
Completed N=75
Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Hereditary Angioedema (HAE)
Source: ClinicalTrials.gov NCT02584959 ↗
Enrolled (actual)
75
Serious AEs
3.9%
Results posted
Nov 2018
Primary outcomePrimary: Time-Normalized Number of Attacks (NNA) for Participants During a Treatment Period — 1.611; 3.931 Number of attacks — p=<0.0001
◆ Published Evidence
Established
24citations · ~6 / year
Interventions for the long-term prevention of hereditary angioedema attacks.
Summary
The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.
Linked Publications (2)
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Interventions for the long-term prevention of hereditary angioedema attacks.
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Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH-HAE: SAHARA Randomized Study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time-Normalized Number of Attacks (NNA) for Participants During a Treatment Period |
1.611; 3.931 | <0.0001 sig |
| SECONDARY Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period. |
38 | — |
| SECONDARY Time-Normalized Number of Attacks (NNA) for Participants During Each Treatment Period Excluding the First 2 Weeks. |
1.524; 3.847 | <0.0001 sig |
| SECONDARY Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Placebo Period Excluding the First 2 Weeks of Each Treatment Period. |
36 | — |
| SECONDARY Proportion of Participants Meeting at Least a 50% Reduction in NNA (Normalized Number of Angioedema Attacks) During the Experimental Injection Treatment Period Relative to the Pre-treatment Assessment. |
41; 13 | — |
| SECONDARY Cumulative Attack Severity |
3.159; 8.041 | <0.0001 sig |
| SECONDARY Number of Attack-free Days |
26.788; 21.353 | <0.0001 sig |
| SECONDARY Number of Angioedema Attacks Requiring Acute Treatment |
1.454; 3.628 | <0.0001 sig |
| SECONDARY Response to Icatibant When Administered for an Acute Attack |
129; 306; 38; 89; 13; 30 | — |
| SECONDARY Number of Patients With Adverse Events (AEs) |
42; 32; 2; 3; 4; 3 | — |
| SECONDARY Number of Participants With Injection Site Reactions |
42; 15; 2; 0; 42; 15 | — |
| SECONDARY Number of Patients With Positive Anti-C1 INH Antibodies |
0; 0; 0; 0 | — |
| SECONDARY PK Parameters: AUC (0-96) and AUC (0-t) for Functional C1 INH Binding Activity |
31070; 13860; 31190; 13860 | — |
| SECONDARY PK Parameters: AUC (0-96) and AUC (0-t) for C1 INH Antigen Concentrations |
6882; 1849; 6902; 1849 | — |
| SECONDARY PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treamtment C1 INH) |
16690; 16780 | — |
| SECONDARY PK Parameters: AUC (0-96) and AUC (0-t) for Complement C4 Concentrations (Treatment Placebo) |
8840; 2150; 8840; 2150 | — |
| SECONDARY PK Parameters: Cmax and Cmin for Functional C1 INH Binding Activity |
396.20; 159.50; 258.15; 125.90 | — |
| SECONDARY PK Parameters: Cmax and Cmin for C1 INH Antigen Concentrations |
77.680; 21.257; 65.562; 17.913 | — |
| SECONDARY PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment C1 INH) |
200; 158 | — |
| SECONDARY PK Parameters: Cmax and Cmin for Complement C4 Concentrations (Treatment Placebo) |
120; 27; 82; 18 | — |
| SECONDARY PK Parameters: Tmax |
31.597; 55.689; 31.656; 47.578; 33.417 | — |
| SECONDARY PK Parameters: Tmax for Complement C4 Concentrations (Placebo Group) |
94.62; 48.12 | — |
| SECONDARY Assess Disease Activity as Measured by the Angioedema Activity Score (AAS) Normalized Per Month |
25.433; 57.168 | 0.0001 sig |
| SECONDARY Participant Experience With Self-administration: Overall Experience With the Syringe |
48; 36; 11; 4; 0; 0 | — |
| SECONDARY Participant Experience With Self-administration: How Many Visits for Confidence With Self-administration |
1.8; 2.0; 1.8 | — |
| SECONDARY Participant Experience With Self-administration: Better Long-term Option and Preferred Administration |
57; 39; 2; 1; 56; 40 | — |
| SECONDARY Mean Change in Angioedema Quality of Life Questionnaire Scores From Baseline to Week 13 |
-10.35; 4.77; -6.86; -12.10; -11.65; -0.52 | — |
Eligibility Criteria
The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (Treatment Period 1 and Treatment Period 2). After completing the 2 treatment periods, patients will enter a 1-month follow-up period.
Data sourced from ClinicalTrials.gov (NCT02584959) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.