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

This Tabular View shows the required WHO registration data elements as marked by ^†

Tracking Information

First Received Date ^†

July 29, 2005

Last Updated Date

May 1, 2009

Start Date ^†

November 2006

Current 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 ]

Original Primary Outcome Measures ^†

Same as current

Change History

Complete list of historical versions of study NCT00125541 on ClinicalTrials.gov Archive Site

Current 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 ]

Original Secondary Outcome Measures ^†

Same as current

Descriptive Information

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

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 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.

Detailed Description

C will provide data on the efficacy of C1-esteraseremmer-N.

Study Phase

Phase II, Phase III

Study Type ^†

Interventional

Study Design ^†

Prevention, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study

Condition ^†

Angioedema

Intervention ^†

Drug: C1 inhibitor concentrate (C1-esteraseremmer-N)

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^†

Completed

Estimated Enrollment ^†

Completion Date

May 2008

Primary Completion Date

April 2008 (final data collection date for primary outcome measure)

Eligibility 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

Gender

Both

Ages

16 Years and older

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

Netherlands

Expanded Access Status

Administrative Information

NCT ID ^†

NCT00125541

Responsible Party

P. Strengers, Sanquin

Secondary IDs ^††

Study Sponsor ^†

Sanquin

Collaborators ^††

Investigators ^†

Principal Investigator:

M. M. Levi, Prof. Dr.

Academic Medical Centre Amsterdam

Information Provided By

Sanquin

Verification Date

May 2009

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.