Kinetics, Efficacy and Safety of C1-Esteraseremmer-N

May 1, 2009 updated by: Prothya Biosolutions

Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema

A multicentre study to investigate pharmacokinetics, clinical efficacy and safety of nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the treatment of hereditary angioedema (HAE) will be performed. This study KB2003.01 consists of three parts, part A pharmacokinetics (phase II), part B treatment of attacks of angioedema (phase III) and part C prophylactic use of C1 inhibitor (phase III). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.

The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.

In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

A multicentre study to investigate pharmacokinetics, clinical efficacy and safety of nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the treatment of hereditary angioedema (HAE) will be performed. This study KB2003.01 consists of three parts, part A pharmacokinetics (phase II), part B treatment of attacks of angioedema (phase III) and part C prophylactic use of C1 inhibitor (phase III). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.

The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.

In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N. Twelve HAE patients without signs of an attack will receive an administration of 1,000 U, 1,500 U or 2,000 U of C1-esteraseremmer-N or Cetor® and later on the same dose of the other product. Both antigenic and functional C1 inhibitor levels will be determined. Laboratory safety parameters and adverse events will be monitored as well

Study Type

Interventional

Enrollment (Anticipated)

12

Phase

  • Phase 2

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

  • Netherlands
      • Amsterdam, Netherlands, 1100 DD
        • Academic Medical Centre
      • Rotterdam, Netherlands, 3015 GD
        • Erasmus Medical Centre

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Established diagnosis of hereditary angioedema type I: i.e. markedly decreased C1 inhibitor activity, decreased level of C1 inhibitor antigen and a decreased level of C4.
  • Age ≥ 18 years
  • Body weight between 40 and 100 kg.
  • Signed Informed consent

Exclusion Criteria:

  • C1 inhibitor infusion within the last 7 days
  • Signs of any attack
  • Angioedema attack within 7 days before actual infusion of study medication
  • Change in the dosage of androgens in the last 14 days before the study
  • Change in oral anticonceptive medication in the last two months before the study
  • Pregnancy or lactation.
  • B-cell malignancy
  • Participation in another pharmaceutical clinical study, which can interfere with this study, in the last 3 months prior to the study
  • History of clinically relevant antibody development to C1 inhibitor
  • Use of oral anticoagulant medication in the last 14 days
  • Use of heparin within the last two days prior to the study
  • History of allergic reaction to C1 inhibitor concentrate or other blood products

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: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Pharmacokinetics of C1-esteraseremmer-N versus Cetor.

Secondary Outcome Measures

Outcome Measure
Laboratory and clinical safety as well as clinical tolerance of C1-esteraseremmer-N versus current Cetor.

Collaborators and Investigators

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

Sponsor

Prothya Biosolutions

Investigators

  • Principal Investigator: M. M. Levi, Prof. Dr., Academic Medical Centre Amsterdam

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

September 1, 2005

Study Completion (Actual)

March 1, 2006

Study Registration Dates

First Submitted

July 4, 2005

First Submitted That Met QC Criteria

July 4, 2005

First Posted (Estimate)

July 13, 2005

Study Record Updates

Last Update Posted (Estimate)

May 4, 2009

Last Update Submitted That Met QC Criteria

May 1, 2009

Last Verified

May 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KB2003.01A

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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