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

CSL Behring

Status and phase: Completed; Phase 2; Phase 1

Conditions: Hereditary Angioedema Types I and II

Treatments: Biological: C1-esterase inhibitor - subcutaneous high dose; Biological: C1-esterase inhibitor - subcutaneous medium dose; Biological: C1-esterase inhibitor - single intravenous dose; Biological: C1-esterase inhibitor - subcutaneous low dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT01576523

CSL830_2001

2011-005013-36 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 18 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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18 participants in 6 patient groups

Low, then medium, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - subcutaneous low dose

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous medium dose

Medium, then low, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - subcutaneous low dose

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous medium dose

Medium, then high, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous high dose

Biological: C1-esterase inhibitor - subcutaneous medium dose

Low, then high, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - subcutaneous low dose

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous high dose

High, then low, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - subcutaneous low dose

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous high dose

High, then medium, C1-esterase inhibitor dose

Experimental group

Treatment:

Biological: C1-esterase inhibitor - single intravenous dose

Biological: C1-esterase inhibitor - subcutaneous high dose

Biological: C1-esterase inhibitor - subcutaneous medium dose