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CompletedNCT01467947Updated Feb 6, 2017Results posted

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

A Phase 4 interventional study of Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection in Hereditary Angioedema Types I and II, sponsored by CSL Behring. Completed at 4 sites in 4 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2017-02-06.

Sponsored by CSL Behring (part of CSL Limited) · Phase 4, Interventional, and Treatment

Phase
Phase 4
Study type
Interventional
Enrollment
46
Allocation
Not applicable
Ages
12 Years and older
Sex
All
01

Study 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. 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.

02

Conditions studied

  • Hereditary Angioedema Types I and II
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In context

Lead sponsor

CSL Behring is the lead sponsor of 145 studies on the registry; 17 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.

First submitted after CSL Behring became part of CSL Limited.

Browse CSL Behring studies →All CSL Limited studies →

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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.

Exclusion criteria

Exclusion Criteria:

  • 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.
05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
46 participants (actual)

Study arms

  • Experimental
    Berinert

    Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection

Interventions

  • BiologicalBerinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection

    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.

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: 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.

    Time frame: Baseline to approximately 9 months

07

Results

Posted Nov 16, 2015

Participant flow

Participant flow — Overall Study
MilestoneC1 Esterase Inhibitor
Started46
Completed46
Not completed0

Outcome measures

PrimaryNumber 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.

Time frame:
Baseline to approximately 9 months
Reported as:
Number · subjects
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies
subjectsC1 Esterase Inhibitor
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies0
SecondaryNumber 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.

Time frame:
Baseline to approximately 9 months
Reported as:
Number · Subjects
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies
SubjectsC1 Esterase Inhibitor
Antibodies detected on Day 1 (Baseline)9
Antibodies detected post-baseline10
Antibodies first detected at Month 32
Antibodies first detected at Month 61
Antibodies first detected at Month 91

Adverse events

Collected over Baseline to approximately 9 months.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
C1 Esterase Inhibitor—2/46 (4.3%)7/46 (15.2%)
Most frequent serious events
Most frequent serious events
EventC1 Esterase Inhibitor
Hereditary angioedemaCongenital, familial and genetic disorders1/46
Abortion spontaneousPregnancy, puerperium and perinatal conditions1/46
Most frequent other events
Most frequent other events
EventC1 Esterase Inhibitor
HeadacheNervous system disorders6/46
HypotensionVascular disorders3/46

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)C1 Esterase Inhibitor
<=18 years2
Between 18 and 65 years42
>=65 years2
Age, Continuous
Age, Continuous(years)C1 Esterase Inhibitor
Mean38.9 ± 14.42
Gender
Gender(Participants)C1 Esterase Inhibitor
Female32
Male14
Region of Enrollment
Region of Enrollment(participants)C1 Esterase Inhibitor
Romania15
Hungary8
Poland13
Bulgaria10
08

Study locations

4 sites
  • MHAT "Tsaritsa Yoanna"
    Sofia, 1504, Bulgaria
  • Semmelweis University
    Budapest, 1125, Hungary
  • Jagiellonian University
    Krakow, Krakow 31-531, Poland
  • Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
    Târgu-Mures, Mureș County Cod 540103, Romania
09

References and documents

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 ↗
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Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Nov 2011, a primary completion of Oct 2014 and a completion of Oct 2014. It does not say whether these dates are actual or estimated.

Results were first posted in Nov 2015, 1 year 1 month after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 7, 2011
First posted
Nov 9, 2011
Start
Nov 2011
Primary completionActual
Oct 2014
CompletionActual
Oct 2014
Results first posted
Nov 16, 2015
Last update
Feb 6, 2017
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Feb 6, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01467947
Lead sponsor
CSL Behring
Responsible party
Sponsor
First posted
Nov 9, 2011
Start date
Nov 2011
Primary completion
Oct 2014
Completion
Oct 2014
Results posted
Nov 16, 2015
Last update
Feb 6, 2017

Study contacts

Mikhail Rojavin
study director · CSL Behring
View the source record on ClinicalTrials.gov ↗

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