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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00125151
Recruitment Status : Completed
First Posted : July 29, 2005
Last Update Posted : May 4, 2009
Sponsor:
Information provided by:
Sanquin Plasma Products BV

Tracking Information
First Submitted Date  ICMJE July 28, 2005
First Posted Date  ICMJE July 29, 2005
Last Update Posted Date May 4, 2009
Study Start Date  ICMJE February 2006
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: August 10, 2005)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: July 28, 2005)
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, the time-to-effect, of C1-esteraseremmer-N.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 28, 2005)
Secondary objectives are the safety of C1-esteraseremmer-N and the ability of C1-esteraseremmer-N to increase the C1 inhibitor level and activity.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE C1-Esteraseremmer-N for the Treatment of Hereditary (and Acquired) Angioedema
Official Title  ICMJE Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema
Brief Summary

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.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Angioedema
Intervention  ICMJE Drug: C1 inhibitor concentrate (C1-esteraseremmer-N)
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: July 28, 2005)
15
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 2007
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Netherlands
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00125151
Other Study ID Numbers  ICMJE KB2003.01B
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Sanquin Plasma Products BV
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: M. M. Levi, Prof. Dr. Academic Medical Centre Amsterdam
PRS Account Sanquin Plasma Products BV
Verification Date May 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP