Phase 2
N=18
A Study to Evaluate the Clinical Pharmacology and Safety of C1-esterase Inhibitor Administered by the Subcutaneous Route
Hereditary Angioedema Types I and II
Bottom Line
View on ClinicalTrials.gov: NCT01576523 ↗Enrolled (actual)
18
Serious AEs
3.7%
Results posted
Feb 2021
Primary outcome: Primary: Modeled C1-esterase Inhibitor Functional Activity Trough Level — 30.3; 45.9; 80.6 percent functional activity
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- C1-esterase inhibitor - single intravenous dose (Biological); C1-esterase inhibitor - subcutaneous low dose (Biological); C1-esterase inhibitor - subcutaneous medium dose (Biological); C1-esterase inhibitor - subcutaneous high dose (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- CSL Behring
- Primary completion
- Dec 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Modeled C1-esterase Inhibitor Functional Activity Trough Level |
30.3; 45.9; 80.6 | — |
| SECONDARY As-observed C1-esterase Inhibitor Functional Activity Trough Level |
31.7; 44.3; 80.5 | — |
| SECONDARY C1-esterase Inhibitor Concentration Trough Level |
0.06; 0.15; 0.23 | — |
| SECONDARY C4 Concentration Trough Level |
11.1; 14.1; 18.4 | — |
| SECONDARY Change From Baseline in C1-esterase Inhibitor Functional Activity |
16.4; 33.2; 63.3 | — |
| SECONDARY Change From Baseline in C1-esterase Inhibitor Concentration |
0.02; 0.05; 0.14 | — |
| SECONDARY Change From Baseline in C4 Concentration |
4.3; 5.6; 9.1 | — |
Summary
The aim of the study is to assess what happens to C1-esterase inhibitor that is administered under the skin of subjects with hereditary angioedema. Three different dosing regimens of C1-esterase inhibitor will be assessed. Each subject will be assigned to receive 2 of the 3 dosing regimens, each for 4 weeks. The activity and concentration of C1-esterase inhibitor in the blood will be measured during each 4-week period. The study will also examine how well C1-esterase inhibitor administered under the skin is tolerated by the subjects.
Eligibility Criteria
Inclusion Criteria
- Males or females aged 18 years or older.
- Laboratory-confirmed hereditary angioedema type I or II.
- Less than two hereditary angioedema attacks per month in the last three months.
- Body weight of 50.0 kg to 110.0 kg.
Exclusion Criteria
- Receiving prophylactic C1-esterase inhibitor therapy.
- Received C1-esterase inhibitor, ecallantide, icatibant or any blood products for the prevention or treatment of hereditary angioedema within 7 days before the screening visit.
- Intends to use recombinant C1-esterase inhibitor or fresh frozen plasma for the acute treatment of hereditary angioedema during the study.
- Received androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 30 days before the screening visit.
- Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.
- Known or suspected hypersensitivity to the study product, or to any excipients of the study product.
- Pregnancy or lactation.
Data sourced from ClinicalTrials.gov (NCT01576523). 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.