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

January 29, 2021 updated by: CSL Behring

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

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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
18

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Berlin, Germany, 10117
        • Study Site
      • Frankfurt, Germany, 60596
        • Study Site
      • Mainz, Germany, 55101
        • Study Site
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Study Site
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • Study Site
    • Ohio
      • Cincinnati, Ohio, United States, 45231
        • Study Site
      • Toledo, Ohio, United States, 43617
        • Study Site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 19108
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Non-Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Low, then medium, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous low dose
A low dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous medium dose
A medium dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Experimental: Medium, then low, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous low dose
A low dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous medium dose
A medium dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Experimental: Medium, then high, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous medium dose
A medium dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous high dose
A high dose of C1-esterase inhibitor will administered subcutaneously twice a week for four weeks.
Experimental: Low, then high, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous low dose
A low dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous high dose
A high dose of C1-esterase inhibitor will administered subcutaneously twice a week for four weeks.
Experimental: High, then low, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous low dose
A low dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous high dose
A high dose of C1-esterase inhibitor will administered subcutaneously twice a week for four weeks.
Experimental: High, then medium, C1-esterase inhibitor dose
Biological: 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.
Biological: C1-esterase inhibitor - subcutaneous medium dose
A medium dose of C1-esterase inhibitor will be administered subcutaneously twice a week for four weeks.
Biological: C1-esterase inhibitor - subcutaneous high dose
A high dose of C1-esterase inhibitor will administered subcutaneously twice a week for four weeks.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
at the fourth week of each dosing regimen

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
during the last week of 4-week dose regimen
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
during the last week of 4-week dose regimen
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
during the last week of 4-week dose regimen
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
Baseline and during the last week of 4-week dose regimen
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
Baseline and during the last week of 4-week dose regimen
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
Baseline and during the last week of 4-week dose regimen

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CSL Behring

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Parexel

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 1, 2012

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2012

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2012

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 10, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 10, 2012

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 12, 2012

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 1, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 29, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSL830_2001
  • 2011-005013-36 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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