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

NCT ID: NCT01576523

Last Updated: 2021-02-01

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-04-30

Study Completion Date

2012-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hereditary Angioedema Types I and II

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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Low, then medium, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous medium dose

Intervention Type BIOLOGICAL

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

Medium, then low, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous medium dose

Intervention Type BIOLOGICAL

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

Medium, then high, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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 medium dose

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous high dose

Intervention Type BIOLOGICAL

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

Low, then high, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous high dose

Intervention Type BIOLOGICAL

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

High, then low, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous high dose

Intervention Type BIOLOGICAL

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

High, then medium, C1-esterase inhibitor dose

Group Type EXPERIMENTAL

C1-esterase inhibitor - single intravenous dose

Intervention Type 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 medium dose

Intervention Type BIOLOGICAL

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

C1-esterase inhibitor - subcutaneous high dose

Intervention Type BIOLOGICAL

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

Interventions

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C1-esterase inhibitor - single intravenous dose

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.

Intervention Type BIOLOGICAL

C1-esterase inhibitor - subcutaneous low dose

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

Intervention Type BIOLOGICAL

C1-esterase inhibitor - subcutaneous medium dose

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

Intervention Type BIOLOGICAL

C1-esterase inhibitor - subcutaneous high dose

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Parexel

INDUSTRY

Sponsor Role collaborator

CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

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Study Site

Atlanta, Georgia, United States

Site Status

Study Site

Chevy Chase, Maryland, United States

Site Status

Study Site

Cincinnati, Ohio, United States

Site Status

Study Site

Toledo, Ohio, United States

Site Status

Study Site

Hershey, Pennsylvania, United States

Site Status

Study Site

Berlin, , Germany

Site Status

Study Site

Frankfurt, , Germany

Site Status

Study Site

Mainz, , Germany

Site Status

Countries

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United States Germany

References

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Zuraw BL, Cicardi M, Longhurst HJ, Bernstein JA, Li HH, Magerl M, Martinez-Saguer I, Rehman SM, Staubach P, Feuersenger H, Parasrampuria R, Sidhu J, Edelman J, Craig T. Phase II study results of a replacement therapy for hereditary angioedema with subcutaneous C1-inhibitor concentrate. Allergy. 2015 Oct;70(10):1319-28. doi: 10.1111/all.12658. Epub 2015 Aug 11.

Reference Type RESULT
PMID: 26016741 (View on PubMed)

Other Identifiers

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2011-005013-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL830_2001

Identifier Type: -

Identifier Source: org_study_id

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