A Study to Evaluate the Clinical Pharmacology and Safety of C1-esterase Inhibitor Administered by the Subcutaneous Route

CSL Behring18 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Hereditary Angioedema Types I and II

Interventions

C1-esterase inhibitor - single intravenous dose

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (8)

Study Site

Atlanta, Georgia, United States

Study Site

Chevy Chase, Maryland, United States

Study Site

Cincinnati, Ohio, United States

Study Site

Toledo, Ohio, United States

Study Site

Hershey, Pennsylvania, United States

Study Site

Berlin, Germany

Study Site

Frankfurt, Germany

Study Site

Mainz, Germany

Outcomes

Primary Outcomes

Modeled C1-esterase Inhibitor Functional Activity Trough Level

Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens, based on modeling and simulation

Time frame: at the fourth week of each dosing regimen

Secondary Outcomes

As-observed C1-esterase Inhibitor Functional Activity Trough Level

Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens

Time frame: during the last week of 4-week dose regimen

C1-esterase Inhibitor Concentration Trough Level

Mean trough C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens

Time frame: during the last week of 4-week dose regimen

C4 Concentration Trough Level

Mean trough C4 concentration of the low, medium and high subcutaneous dose regimens

Time frame: during the last week of 4-week dose regimen

Change From Baseline in C1-esterase Inhibitor Functional Activity

Mean change from baseline of C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens

Time frame: Baseline and during the last week of 4-week dose regimen

Change From Baseline in C1-esterase Inhibitor Concentration

Mean change from baseline of C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens

Time frame: Baseline and during the last week of 4-week dose regimen

Change From Baseline in C4 Concentration

Mean change from baseline of C4 concentration of the low, medium and high subcutaneous dose regimens

Time frame: Baseline and during the last week of 4-week dose regimen