C1-Esteraseremmer-N for the Treatment of Hereditary (and Acquired) Angioedema

This study has been completed.
Sponsor:
Information provided by:
Sanquin
ClinicalTrials.gov Identifier:
NCT00125541
First received: July 29, 2005
Last updated: May 1, 2009
Last verified: May 2009
  Purpose

A multicentre study to investigate the 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). Parts B + C will provide data on the efficacy of C1-esteraseremmer-N.

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

In part C of the study, a number of HAE patients will be treated prophylactically with open-label C1-esteraseremmer-N for a period of 16 weeks. The number of attacks occurring will be compared with historical data. If possible, also some patients treated prophylactically with C1 inhibitor for acquired angioedema will be included.


Condition Intervention Phase
Angioedema
Drug: C1 inhibitor concentrate (C1-esteraseremmer-N)
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
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:
  • The primary objective in this clinical study is to evaluate the prevention of angioedema attacks in patients prophylactically treated with C1-esteraseremmer-N. [ Time Frame: 16 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The secondary objective of this study is to evaluate the safety and clinical activity of C1-esteraseremmer-N. [ Time Frame: 16 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 6
Study Start Date: November 2006
Study Completion Date: May 2008
Primary Completion Date: April 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: C1 inhibitor concentrate (C1-esteraseremmer-N)
    every 5-7 days
Detailed Description:

A multicentre study to investigate the 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). Parts B + C will provide data on the efficacy of C1-esteraseremmer-N.

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

In part C of the study, a number of HAE patients will be treated prophylactically with open-label C1-esteraseremmer-N for a period of 16 weeks. The number of attacks occurring will be compared with historical data. If possible, also some patients treated prophylactically with C1 inhibitor for acquired angioedema will be included.

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Inclusion criteria for hereditary angioedema patients:

  • Established diagnosis of hereditary angioedema type I or II: markedly decreased C1 inhibitor activity; decreased (type I), normal or elevated (type II) level of C1 inhibitor antigen; decreased level of C4.
  • Patients already using C1 inhibitor concentrate for prophylaxis of angioedema attacks.
  • Age ≥ 16 years
  • Signed informed consent by patient and patient's legal representative if under 18 years old

Inclusion criteria for acquired angioedema patients:

  • Established diagnosis of acquired angioedema: recurrent attacks of angioedema without urticaria; no family history; decreased functional C1 inhibitor; decreased level of C4.
  • Autoantibodies to C1 inhibitor or decreased C1q or onset after the third decade of life.
  • Age ≥ 16 years
  • Patient already using C1 inhibitor concentrate for prophylaxis of angioedema attacks
  • Signed informed consent by patient and patient's legal representative if under 18 years old

Exclusion Criteria:

Exclusion criteria for hereditary angioedema patients:

  • Use of angioedema prophylactic medication during the study, other than C1-esteraseremmer-N.
  • Change in oral contraceptives starting from the last two months before the start of the trial until the end of the study period.
  • Presence of clinically relevant C1 inhibitor auto antibodies
  • Participation in another pharmaceutical clinical study, which can interfere with this study, in the last 3 months prior to the study, other than in part A of this protocol.
  • Usage of heparin starting from the last two days prior to the study until the end of the study period.
  • B-cell malignancy
  • Pregnancy or lactation
  • History of allergic reaction to C1 inhibitor concentrate or other blood products

Exclusion criteria for acquired angioedema patients:

  • Participation in another pharmaceutical clinical study, which can interfere with this study, in the last 3 months prior to the study
  • Usage of heparin within the last two days prior to the study
  • Pregnancy or lactation
  • 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: NCT00125541

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

No publications provided

Responsible Party: P. Strengers, Sanquin
ClinicalTrials.gov Identifier: NCT00125541     History of Changes
Other Study ID Numbers: KB2003.01C
Study First Received: July 29, 2005
Last Updated: May 1, 2009
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Sanquin:
MeSH: angioneurotic edema, complement 1 inactivators
Hereditary Angioedema Type I or II, Acquired Angioedema

Additional relevant MeSH terms:
Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases

ClinicalTrials.gov processed this record on September 18, 2014