Hereditary Angioedema Types I and IIPhase 4NCT01467947
Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert
A Phase 4 study testing Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection in people with Hereditary Angioedema Types I and II, run by CSL Behring at 4 sites in 4 countries.
Trial summary
This is a prospective, international, multi-center, non-randomized, single arm, open-label, postmarketing study to investigate the formation of inhibitory anti-C1-INH antibodies in HAE subjects treated intravenously with Berinert. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema Types I and II
- What is being tested?
- Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Nov 2011to Oct 2014
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of congenital C1-INH deficiency (HAE type I or II) and assessed by the investigator to likely require intravenous (IV) Berinert treatment during the study period.
- Male or female, ≥ 12 years of age at the time of signing informed consent.
- Written informed consent for study participation obtained before undergoing any study specific procedures.
Who cannot join
- Incurable malignancies in the last 6 months prior to study entry.
- Acquired angioedema due to C1-INH deficiency.
- All other types of angioedema not associated with C1-INH deficiency.
- Use of any C1-INH products other than Berinert within 30 days before the study, or planned use during the study.
- Immunization within 30 days prior to study entry.
- Autoimmune conditions requiring use of immunosuppressants during the study.
- Known or suspected hypersensitivity to C1-INH.
- Participation in any of the previous Berinert studies from which anti-C1-INH antibody results were submitted to the Food and Drug Administration.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
Romania1 site
Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
Târgu-Mures, Mureș County, Cod 540103
About this study
Official title: Prospective Open-label Uncontrolled Multi-center Post-marketing Study to Assess Inhibitory Antibody Formation in Subjects With Congenital C1-INH Deficiency and Acute Hereditary Angioedema (HAE) Attacks Treated With Berinert® , a C1-esterase Inhibitor
This is a prospective, international, multi-center, non-randomized, single arm, open-label, postmarketing study to investigate the formation of inhibitory anti-C1-INH antibodies in HAE subjects treated intravenously with Berinert. Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. All subjects will receive 20 IU Berinert/kg body weight per attack.
- Conditions
- Hereditary Angioedema Types I and II
- Being tested
- Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection
- Medical subject headings
- Hereditary Angioedema Types I and II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injectionBiological
Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. Each attack that occurs in this time frame will be treated with 20 IU Berinert/kg body weight.
| Study group | Role | What they receive |
|---|---|---|
| Berinert | Experimental | Not described Receives: Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies
Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.
Measured: Baseline to approximately 9 months
Secondary outcomes (1)
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies
Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.
Measured: Baseline to approximately 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2011 (estimated)
- Study startNov 2011
- Primary completionOct 2014
- Study completionOct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary Angioedema Types I and IIStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mikhail Rojavin, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01467947
- Sponsor's protocol ID
- CE1145_4001
- Other IDs
- 2010-024242-30
- Status last verified
- Oct 2015
- First submitted
- 7 Nov 2011
- Last updated
- 6 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Farkas H, Varga L, Moldovan D, Obtulowicz K, Shirov T, Machnig T, Feuersenger H, Edelman J, Williams-Herman D, Rojavin M. Assessment of inhibitory antibodies in patients with hereditary angioedema treated with plasma-derived C1 inhibitor. Ann Allergy Asthma Immunol. 2016 Nov;117(5):508-513. doi: 10.1016/j.anai.2016.08.025. Epub 2016 Oct 24.
PubMed 27788880
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Open-label Uncontrolled Multi-center Post-marketing Study to Assess Inhibitory Antibody Formation in Subjects With Congenital C1-INH Deficiency and Acute Hereditary Angioedema (HAE) Attacks Treated With Berinert® , a C1-esterase Inhibitor
- ClinicalTrials.gov ID
- NCT01467947
- Acronym / sponsor ID
- CE1145_4001
- Conditions
- Hereditary Angioedema Types I and II
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Nov 2011
- Primary completion
- Oct 2014 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 9 Nov 2011 (estimated)
- Results
- Posted 16 Nov 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, international, multi-center, non-randomized, single arm, open-label, postmarketing study to investigate the formation of inhibitory anti-C1-INH antibodies in HAE subjects treated intravenously with Berinert. Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. All subjects will receive 20 IU Berinert/kg body weight per attack.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Berinert | Experimental | Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of congenital C1-INH deficiency (HAE type I or II) and assessed by the investigator to likely require intravenous (IV) Berinert treatment during the study period.
- Male or female, ≥ 12 years of age at the time of signing informed consent.
- Written informed consent for study participation obtained before undergoing any study specific procedures.
Key exclusion criteria (8 listed)
- Incurable malignancies in the last 6 months prior to study entry.
- Acquired angioedema due to C1-INH deficiency.
- All other types of angioedema not associated with C1-INH deficiency.
- Use of any C1-INH products other than Berinert within 30 days before the study, or planned use during the study.
- Immunization within 30 days prior to study entry.
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 |
|---|---|---|
| Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injectionIndividual treatment duration per subject is 9 months, irrespective of the number of treated attacks. Each attack that occurs in this time frame will be treated with 20 IU Berinert/kg body weight. | Biological | Berinert |
Primary outcome
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies
Time frame: Baseline to approximately 9 months
Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.
1 secondary outcome
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies
Time frame: Baseline to approximately 9 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 27788880Farkas H, Varga L, Moldovan D, Obtulowicz K, Shirov T, Machnig T, Feuersenger H, Edelman J, Williams-Herman D, Rojavin M. Assessment of inhibitory antibodies in patients with hereditary angioedema treated with plasma-derived C1 inhibitor. Ann Allergy Asthma Immunol. 2016 Nov;117(5):508-513. doi: 10.1016/j.anai.2016.08.025. Epub 2016 Oct 24.
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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.
Mikhail Rojavin study director
CSL Behring
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection in people with Hereditary Angioedema Types I and II, run by CSL Behring at 4 sites in 4 countries.
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 3 inclusion and 8 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 6 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Bulgaria, Hungary, Poland, Romania. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Nov 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies (time frame: Baseline to approximately 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01467947, which also lists 1 linked publication.
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 6 Feb 2017.