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

Primary Purpose

Angioedema

Status
Completed
Phase
Phase 3
Locations
Netherlands
Study Type
Interventional
Intervention
C1 inhibitor concentrate (C1-esteraseremmer-N)
Sponsored by
Prothya Biosolutions
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Angioedema focused on measuring MeSH: angioneurotic edema, complement 1 inactivators, Hereditary angioedema type I or II, Acquired angioedema

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Academic Medical Centre
  • Academic hospital Groningen
  • Erasmus Medical Centre

Outcomes

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.

Full Information

First Posted
July 28, 2005
Last Updated
May 1, 2009
Sponsor
Prothya Biosolutions
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1. Study Identification

Unique Protocol Identification Number
NCT00125151
Brief Title
C1-Esteraseremmer-N for the Treatment of Hereditary (and Acquired) Angioedema
Official Title
Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema
Study Type
Interventional

2. Study Status

Record Verification Date
May 2009
Overall Recruitment Status
Completed
Study Start Date
February 2006 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
April 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Prothya Biosolutions

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angioedema
Keywords
MeSH: angioneurotic edema, complement 1 inactivators, Hereditary angioedema type I or II, Acquired angioedema

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
15 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
C1 inhibitor concentrate (C1-esteraseremmer-N)
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Secondary objectives are the safety of C1-esteraseremmer-N and the ability of C1-esteraseremmer-N to increase the C1 inhibitor level and activity.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
M. M. Levi, Prof. Dr.
Organizational Affiliation
Academic Medical Centre Amsterdam
Official's Role
Principal Investigator
Facility Information:
Facility Name
Academic Medical Centre
City
Amsterdam
ZIP/Postal Code
1100 DD
Country
Netherlands
Facility Name
Academic hospital Groningen
City
Groningen
ZIP/Postal Code
9700 RB
Country
Netherlands
Facility Name
Erasmus Medical Centre
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands

12. IPD Sharing Statement

Learn more about this trial

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

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