Kinetics, Efficacy and Safety of C1-Esteraseremmer-N

This study has been completed.
Sponsor:
Information provided by:
Sanquin
ClinicalTrials.gov Identifier:
NCT00119431
First received: July 4, 2005
Last updated: May 1, 2009
Last verified: May 2009
  Purpose

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.


Condition Intervention Phase
Hereditary Angioedema Type I
Angioneurotic Edema
Drug: C1 inhibitor concentrate
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Crossover Assignment
Masking: Double-Blind
Primary Purpose: Prevention
Official Title: Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema

Resource links provided by NLM:


Further study details as provided by Sanquin:

Primary Outcome Measures:
  • Pharmacokinetics of C1-esteraseremmer-N versus Cetor.

Secondary Outcome Measures:
  • Laboratory and clinical safety as well as clinical tolerance of C1-esteraseremmer-N versus current Cetor.

Estimated Enrollment: 12
Study Start Date: September 2005
Study Completion Date: March 2006
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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00119431

Locations
Netherlands
Academic Medical Centre
Amsterdam, Netherlands, 1100 DD
Erasmus Medical Centre
Rotterdam, Netherlands, 3015 GD
Sponsors and Collaborators
Sanquin
Investigators
Principal Investigator: M. M. Levi, Prof. Dr. Academic Medical Centre Amsterdam
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00119431     History of Changes
Other Study ID Numbers: KB2003.01A
Study First Received: July 4, 2005
Last Updated: May 1, 2009
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Sanquin:
Hereditary angioedema type I (MeSH: angioneurotic edema, complement 1 inactivators)

Additional relevant MeSH terms:
Angioedema
Edema
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Signs and Symptoms
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 29, 2014