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Completed

NCT Number: NCT01576523

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

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.

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Key information

Conditions

Hereditary Angioedema Types I and II Angioedema Angioedemas, Hereditary Cardiovascular Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Study Site, Berlin, Germany

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

C1-esterase inhibitor - single intravenous dose

Biological

A single intravenous dose of C1-esterase inhibitor (Berinert) at 20 units per kg body weight will be administered to all subjects prior to receiving the first dose of subcutaneous C1-esterase inhibitor.

C1-esterase inhibitor - subcutaneous low dose

Biological

A low dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.

C1-esterase inhibitor - subcutaneous medium dose

Biological

A medium dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.

C1-esterase inhibitor - subcutaneous high dose

Biological

A high dose of C1-esterase inhibitor will administered subcutaneously twice a week for four weeks.

Primary outcomes

  1. Modeled C1-esterase Inhibitor Functional Activity Trough Level

    Time frame: at the fourth week of each dosing regimen

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

Secondary outcomes

  1. As-observed C1-esterase Inhibitor Functional Activity Trough Level

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

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

  2. C1-esterase Inhibitor Concentration Trough Level

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

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

  3. C4 Concentration Trough Level

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

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

  4. Change From Baseline in C1-esterase Inhibitor Functional Activity

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

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

  5. Change From Baseline in C1-esterase Inhibitor Concentration

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

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

  6. Change From Baseline in C4 Concentration

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

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

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Collaborators

  • Parexel

Registry information

Official study title

An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjects With Hereditary Angioedema

Important dates

Study start
2012
Primary completion
2012
Study completion
2012
First posted
Apr 12, 2012
Registry last updated
Feb 1, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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