Trial record 2 of 2 for:    "Acquired angioedema"

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

This study has been completed.
Sponsor:
Information provided by:
Sanquin
ClinicalTrials.gov Identifier:
NCT00125151
First received: July 28, 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 B of the study, 15 attacks of hereditary angioedema will be treated with open-label C1-esteraseremmer-N. Attack severity and duration will be monitored by the subjective experience of the patient himself (which has been shown to be the most sensitive way of monitoring attack severity) and compared with historical (and literature) data. If possible, some attacks of acquired angioedema will also be included.


Condition Intervention Phase
Angioedema
Drug: C1 inhibitor concentrate (C1-esteraseremmer-N)
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: Treatment
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, in which patients use C1-esteraseremmer-N to treat an attack of angioedema, is to investigate the efficacy and the time-to-effect of C1-esteraseremmer-N.

Secondary Outcome Measures:
  • Secondary objectives are the safety of C1-esteraseremmer-N and the ability of C1-esteraseremmer-N to increase the C1 inhibitor level and activity.

Estimated Enrollment: 15
Study Start Date: February 2006
Study Completion Date: April 2007
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 B of the study, 15 attacks of hereditary angioedema will be treated with open-label C1-esteraseremmer-N. Attack severity and duration will be monitored by the subjective experience of the patient himself (which has been shown to be the most sensitive way of monitoring attack severity) and compared with historical (and literature) data. If possible, some attacks of acquired angioedema will also 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 HAE type I and type II 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.
  • Age ≥ 16 years
  • Evidence of a peripheral, abdominal, facial, laryngeal or genitourinary attack of angioedema of moderate to severe intensity, starting less then 5 hours before infusion. An attack is defined as moderate if it affects the normal daily activities of the patient in any way. Severe attacks are defined by the inability to perform normal daily activities.
  • 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
  • Evidence of a peripheral, abdominal, facial, laryngeal or genitourinary attack of angioedema of moderate to severe intensity, starting less then 5 hours before infusion. An attack is defined as moderate if it affects the normal daily activities of the patient in any way. Severe attacks are defined by the inability to perform normal daily activities.
  • Currently treated with C1 inhibitor concentrate to reverse angioedema.
  • Signed informed consent by patient and patient's legal representative if under 18 years old

Exclusion Criteria:

Exclusion criteria for HAE type I and type II patients:

  • Presence of clinically-relevant anti-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 Part A of this protocol.
  • Addiction to narcotic/pain medication in case of an abdominal attack
  • B-cell malignancy
  • Use of narcotic medication within 3 days prior to attack.
  • Use of heparin within the last two days prior to the study
  • Pregnancy or lactation
  • History of allergic reactions 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
  • Addiction to narcotic/pain medication in case of an abdominal attack
  • Use of narcotic medication within 3 days prior to attack.
  • Use of heparin within the last two days prior to the study
  • Pregnancy or lactation.
  • History of allergic reactions 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: NCT00125151

Locations
Netherlands
Academic Medical Centre
Amsterdam, Netherlands, 1100 DD
Academic hospital Groningen
Groningen, Netherlands, 9700 RB
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: NCT00125151     History of Changes
Other Study ID Numbers: KB2003.01B
Study First Received: July 28, 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 August 28, 2014