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

NCT ID: NCT00125151

Last Updated: 2009-05-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-02-28
• Study Completion Date: 2007-04-30

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.

Conditions

Angioedema

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

C1 inhibitor concentrate (C1-esteraseremmer-N)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

• 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

• 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

• 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

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Prothya Biosolutions

INDUSTRY

Sponsor Role: lead

Principal Investigators

M. M. Levi, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

Academic Medical Centre Amsterdam

Locations

Academic Medical Centre

Amsterdam, , Netherlands

Academic hospital Groningen

Groningen, , Netherlands

Erasmus Medical Centre

Rotterdam, , Netherlands

Countries

Netherlands

Other Identifiers

KB2003.01B

Identifier Type: -

Identifier Source: org_study_id