Hereditary Angioedema Type IPhase 2NCT00119431
Kinetics, Efficacy and Safety of C1-Esteraseremmer-N
A Phase 2 study testing C1 inhibitor concentrate in people with Hereditary Angioedema Type I and Angioneurotic Edema, run by Prothya Biosolutions at 2 sites in Netherlands.
Trial summary
A multicentre study to investigate 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema Type I, Angioneurotic Edema
- What is being tested?
- C1 inhibitor concentrate
- How many people
- 12 participants
- Where
- 2 sitesNetherlands
- Study dates
- Sep 2005to Mar 2006
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Established diagnosis of hereditary angioedema type I: i.e. markedly decreased C1 inhibitor activity, decreased level of C1 inhibitor antigen and a decreased level of C4.
- Age ≥ 18 years
- Body weight between 40 and 100 kg.
- Signed Informed consent
Who cannot join
- C1 inhibitor infusion within the last 7 days
- Signs of any attack
- Angioedema attack within 7 days before actual infusion of study medication
- Change in the dosage of androgens in the last 14 days before the study
- Change in oral anticonceptive medication in the last two months before the study
- Pregnancy or lactation.
Show all 12Show fewer
- B-cell malignancy
- Participation in another pharmaceutical clinical study, which can interfere with this study, in the last 3 months prior to the study
- History of clinically relevant antibody development to C1 inhibitor
- Use of oral anticoagulant medication in the last 14 days
- Use of heparin within the last two days prior to the study
- History of allergic reaction to C1 inhibitor concentrate or other blood products
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
Academic Medical Centre
Amsterdam, 1100 DD
Erasmus Medical Centre
Rotterdam, 3015 GD
About this study
Official title: Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema
A multicentre study to investigate 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). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N.
Read the detailed description
A multicentre study to investigate 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). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N. Twelve HAE patients without signs of an attack will receive an administration of 1,000 U, 1,500 U or 2,000 U of C1-esteraseremmer-N or Cetor® and later on the same dose of the other product. Both antigenic and functional C1 inhibitor levels will be determined. Laboratory safety parameters and adverse events will be monitored as well
- Conditions
- Hereditary Angioedema Type I, Angioneurotic Edema
- Being tested
- C1 inhibitor concentrate
- Keywords
- Hereditary angioedema type I (MeSH: angioneurotic edema, complement 1 inactivators)
- Medical subject headings
- Hereditary Angioedema Types I and II, Angioedema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
C1 inhibitor concentrateDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics of C1-esteraseremmer-N versus Cetor.
Secondary outcomes (1)
Laboratory and clinical safety as well as clinical tolerance of C1-esteraseremmer-N versus current Cetor.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2005 (estimated)
- Study startSep 2005
- Study completionMar 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema Type IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Prothya Biosolutions
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- M. M. Levi, Prof. Dr., Principal investigator, Academic Medical Centre Amsterdam
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00119431
- Sponsor's protocol ID
- KB2003.01A
- Status last verified
- May 2009
- First submitted
- 4 Jul 2005
- Last updated
- 4 May 2009
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema
- ClinicalTrials.gov ID
- NCT00119431
- Acronym / sponsor ID
- KB2003.01A
- Conditions
- Hereditary Angioedema Type I, Angioneurotic Edema
- Keywords
- Hereditary angioedema type I (MeSH: angioneurotic edema, complement 1 inactivators)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Prothya BiosolutionsIndustry (pharma, biotech, devices)
- Study start
- Sep 2005
- Study completion
- Mar 2006 (actual)
- First posted
- 13 Jul 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A multicentre study to investigate 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). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N.
Detailed description
A multicentre study to investigate 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). Part B + C will provide data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary angioedema will be compared with the current registered product, Cetor®, in a randomised, blinded cross-over design. This study has to provide evidence that changes in the manufacturing process have not affected pharmacokinetics. In addition, this study provides data on safety of C1-esteraseremmer-N. Twelve HAE patients without signs of an attack will receive an administration of 1,000 U, 1,500 U or 2,000 U of C1-esteraseremmer-N or Cetor® and later on the same dose of the other product. Both antigenic and functional C1 inhibitor levels will be determined. Laboratory safety parameters and adverse events will be monitored as well
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Established diagnosis of hereditary angioedema type I: i.e. markedly decreased C1 inhibitor activity, decreased level of C1 inhibitor antigen and a decreased level of C4.
- Age ≥ 18 years
- Body weight between 40 and 100 kg.
- Signed Informed consent
Key exclusion criteria (12 listed)
- C1 inhibitor infusion within the last 7 days
- Signs of any attack
- Angioedema attack within 7 days before actual infusion of study medication
- Change in the dosage of androgens in the last 14 days before the study
- Change in oral anticonceptive medication in the last two months before the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| C1 inhibitor concentrate | Drug | – |
Primary outcome
Pharmacokinetics of C1-esteraseremmer-N versus Cetor.
1 secondary outcome
Laboratory and clinical safety as well as clinical tolerance of C1-esteraseremmer-N versus current Cetor.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2009.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
M. M. Levi, Prof. Dr. principal investigator
Academic Medical Centre Amsterdam
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing C1 inhibitor concentrate in people with Hereditary Angioedema Type I and Angioneurotic Edema, run by Prothya Biosolutions at 2 sites in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 May 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetics of C1-esteraseremmer-N versus Cetor.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 May 2009.