Hereditary AngioedemaPhase 3NCT00292981

C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)

A Phase 3 study testing C1 Esterase Inhibitor in people with Hereditary Angioedema, run by CSL Behring at 14 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 6 years and older
Conditions the study is for: Hereditary Angioedema
What is being tested?: C1 Esterase Inhibitor
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 57 participants
Where: 14 sitesin 2 countries
Study dates: Aug 2005to May 2010
Phase: Phase 3
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 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Documented congenital C1-INH deficiency
Acute HAE attack
Participation in base study CE1145_3001 (NCT00168103)

Who cannot join

Acquired angioedema
Treatment with any other investigational drug besides CE1145 in the last 30 days before study entry

Trial locations and contacts

14 sites in 2 countries.

Country

City, postcode or hospital

14 sites in 2 countries

United States13 sites

Contact CSL Behring for facility details
Atlanta, Georgia, 30342
Contact CSL Behring for facility details
Boston, Massachusetts, 02115
Contact CSL Behring for facility details
Chicago, Illinois, 60612-3244
Contact CSL Behring for facility details
Dallas, Texas, 75230
Contact CSL Behring for facility details
Eugene, Oregon, 97401
Contact CSL Behring for facility details
Hershey, Pennsylvania, 17033
Contact CSL Behring for facility details
Idaho Falls, Idaho, 83404
Contact CSL Behring for facility details
Omaha, Nebraska, 69131
Contact CSL Behring for facility details
Plymouth, Minnesota, 55446
Contact CSL Behring for facility details
Rapid City, South Dakota, 57702
Contact CSL Behring for facility details
Shreveport, Louisiana, 71130
Contact CSL Behring for facility details
Tulsa, Oklahoma, 74133
Contact CSL Behring for facility details
Weston, Florida, 33331

Other trials in United States

Canada1 site

Contact CSL Behring for facility details
Ottawa, Ontario, KIY 4G2

Other trials in Canada

About this study

Official title: Open-label Extension Study of CE1145 (Human Pasteurized C1 Esterase Inhibitor Concentrate) in Subjects With Congenital C1-INH Deficiency and Acute HAE Attacks

Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of involvement of the larynx.The planned extension study is designed to enrol subjects that participated in the pivotal study in order to provide them with C1-INH for treatment of acute HAE attacks for 24 months or until the licensing procedure for C1-INH is finalized, whatever comes first.

Conditions: Hereditary Angioedema
Being tested: C1 Esterase Inhibitor
Keywords: C1 Inhibitor, Hereditary angioedema, Acute HAE attack
Medical subject headings: Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

C1 Esterase InhibitorDrug
Also called Berinert P
Lyophilisate containing approximately 500 U C1-INH to be reconstituted with 10 mL water for injection; Single Dose: 20 U/kg b.w. iv

Study groups in NCT00292981
Study group	Role	What they receive
C1 Esterase Inhibitor	Experimental	Not described Receives: Drug: C1 Esterase Inhibitor

What is being measured

The primary outcome is the main question the study is designed to answer.

Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)
The start of symptom relief was determined by subject self-assessment.
Measured: Up to 24 h after start of study treatment
Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)
The start of symptom relief was determined by subject self-assessment.
Measured: Up to 24 h after start of study treatment

Secondary outcomes (2)

Time to Complete Resolution of All HAE Symptoms (ITT Subject Population)
Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.
Measured: Up to Day 9 following an attack
Time to Complete Resolution of All HAE Symptoms (ITT Attack Population)
Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.
Measured: Up to Day 9 following an attack

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2005May 2010

First posted16 Feb 2006 (estimated)
Study startAug 2005
Primary completionFeb 2010
Study completionMay 2010

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 (7)Papers the sponsor has linked to this study.
Other trials for Hereditary AngioedemaStudies 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 3
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 57 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00292981
Sponsor's protocol ID: CE1145_3003
Other IDs: 1453
Status last verified: Oct 2011
First submitted: 15 Feb 2006
Last updated: 7 May 2015
Results posted: Yes, on ClinicalTrials.gov

Publications

Craig TJ, Wasserman RL, Levy RJ, Bewtra AK, Schneider L, Packer F, Yang WH, Keinecke HO, Kiessling PC. Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema. J Clin Immunol. 2010 Nov;30(6):823-9. doi: 10.1007/s10875-010-9442-1. Epub 2010 Jul 16.
PubMed 20635155
Bernstein JA, Machnig T, Keinecke HO, Whelan GJ, Craig TJ. The effect of weight on the efficacy and safety of C1 esterase inhibitor concentrate for the treatment of acute hereditary angioedema. Clin Ther. 2014 Apr 1;36(4):518-25. doi: 10.1016/j.clinthera.2014.02.005. Epub 2014 Mar 21.
PubMed 24661784
Bork K, Craig TJ, Bernstein JA, Feuersenger H, Machnig T, Staubach P. Efficacy of C1 esterase inhibitor concentrate in treatment of cutaneous attacks of hereditary angioedema. Allergy Asthma Proc. 2015 May-Jun;36(3):218-24. doi: 10.2500/aap.2015.36.3844. Epub 2015 Mar 23.
PubMed 25803207
Wasserman RL, Levy RJ, Bewtra AK, Hurewitz D, Craig TJ, Kiessling PC, Keinecke HO, Bernstein JA. Prospective study of C1 esterase inhibitor in the treatment of successive acute abdominal and facial hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2011 Jan;106(1):62-8. doi: 10.1016/j.anai.2010.10.012. Epub 2010 Nov 20.
PubMed 21195947
Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, Moy JN, Offenberger J, Jacobson KW, Yang WH, Eidelman F, Janss G, Packer FR, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks--final results of the I.M.P.A.C.T.2 study. Allergy. 2011 Dec;66(12):1604-11. doi: 10.1111/j.1398-9995.2011.02702.x. Epub 2011 Sep 2.
PubMed 21884533
Craig TJ, Rojavin MA, Machnig T, Keinecke HO, Bernstein JA. Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Sep;111(3):211-5. doi: 10.1016/j.anai.2013.06.021. Epub 2013 Jul 16.
PubMed 23987198
Craig TJ, Bewtra AK, Hurewitz D, Levy R, Janss G, Jacobson KW, Packer F, Bernstein JA, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. Treatment response after repeated administration of C1 esterase inhibitor for successive acute hereditary angioedema attacks. Allergy Asthma Proc. 2012 Jul-Aug;33(4):354-61. doi: 10.2500/aap.2012.33.3589.
PubMed 22856636

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema

All Hereditary Angioedema trials

Study at a glance

From the registry record, last updated by the sponsor on 7 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Open-label Extension Study of CE1145 (Human Pasteurized C1 Esterase Inhibitor Concentrate) in Subjects With Congenital C1-INH Deficiency and Acute HAE Attacks
ClinicalTrials.gov ID: NCT00292981
Acronym / sponsor ID: CE1145_3003
Conditions: Hereditary Angioedema
Keywords: C1 Inhibitor, Hereditary angioedema, Acute HAE attack
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 57 participants (actual)
Lead sponsor: CSL BehringIndustry (pharma, biotech, devices)
Study start: Aug 2005
Primary completion: Feb 2010 (actual)
Study completion: May 2010 (actual)
First posted: 16 Feb 2006 (estimated)
Results: Posted 23 Jun 2011

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
C1 Esterase Inhibitor	Experimental	Drug: C1 Esterase Inhibitor

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Documented congenital C1-INH deficiency
Acute HAE attack
Participation in base study CE1145_3001 (NCT00168103)

Key exclusion criteria (2 listed)

Acquired angioedema
Treatment with any other investigational drug besides CE1145 in the last 30 days before 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
C1 Esterase InhibitorAlso: Berinert PLyophilisate containing approximately 500 U C1-INH to be reconstituted with 10 mL water for injection; Single Dose: 20 U/kg b.w. iv	Drug	C1 Esterase Inhibitor

Primary outcome

Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)
Time frame: Up to 24 h after start of study treatment
The start of symptom relief was determined by subject self-assessment.
Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)
Time frame: Up to 24 h after start of study treatment
The start of symptom relief was determined by subject self-assessment.

2 secondary outcomes

Time to Complete Resolution of All HAE Symptoms (ITT Subject Population)
Time frame: Up to Day 9 following an attack
Time to Complete Resolution of All HAE Symptoms (ITT Attack Population)
Time frame: Up to Day 9 following an attack

Results and safety

Results posted to the registry on 23 Jun 2011, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
C1 Esterase Inhibitor	57	18

Primary outcome: Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)

Time frame: Up to 24 h after start of study treatment. Median (95% Confidence Interval). Unit: hours

Measure	C1 Esterase Inhibitor n = 57
Value	0.46 (0.39 to 0.53)

Population analysed: Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication.

Primary outcome: Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)

Time frame: Up to 24 h after start of study treatment. Median (95% Confidence Interval). Unit: hours

Measure	C1 Esterase Inhibitor n = 57
Value	0.37 (0.33 to 0.42)

Population analysed: ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
C1 Esterase Inhibitor	1 of 57 (1.8%)	25 of 57 (43.9%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2015.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 20635155Craig TJ, Wasserman RL, Levy RJ, Bewtra AK, Schneider L, Packer F, Yang WH, Keinecke HO, Kiessling PC. Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema. J Clin Immunol. 2010 Nov;30(6):823-9. doi: 10.1007/s10875-010-9442-1. Epub 2010 Jul 16.
Background: PubMed 24661784Bernstein JA, Machnig T, Keinecke HO, Whelan GJ, Craig TJ. The effect of weight on the efficacy and safety of C1 esterase inhibitor concentrate for the treatment of acute hereditary angioedema. Clin Ther. 2014 Apr 1;36(4):518-25. doi: 10.1016/j.clinthera.2014.02.005. Epub 2014 Mar 21.
Background: PubMed 25803207Bork K, Craig TJ, Bernstein JA, Feuersenger H, Machnig T, Staubach P. Efficacy of C1 esterase inhibitor concentrate in treatment of cutaneous attacks of hereditary angioedema. Allergy Asthma Proc. 2015 May-Jun;36(3):218-24. doi: 10.2500/aap.2015.36.3844. Epub 2015 Mar 23.
Results publication: PubMed 21195947Wasserman RL, Levy RJ, Bewtra AK, Hurewitz D, Craig TJ, Kiessling PC, Keinecke HO, Bernstein JA. Prospective study of C1 esterase inhibitor in the treatment of successive acute abdominal and facial hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2011 Jan;106(1):62-8. doi: 10.1016/j.anai.2010.10.012. Epub 2010 Nov 20.
Results publication: PubMed 21884533Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, Moy JN, Offenberger J, Jacobson KW, Yang WH, Eidelman F, Janss G, Packer FR, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks--final results of the I.M.P.A.C.T.2 study. Allergy. 2011 Dec;66(12):1604-11. doi: 10.1111/j.1398-9995.2011.02702.x. Epub 2011 Sep 2.
Derived from this study: PubMed 23987198Craig TJ, Rojavin MA, Machnig T, Keinecke HO, Bernstein JA. Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Sep;111(3):211-5. doi: 10.1016/j.anai.2013.06.021. Epub 2013 Jul 16.
Derived from this study: PubMed 22856636Craig TJ, Bewtra AK, Hurewitz D, Levy R, Janss G, Jacobson KW, Packer F, Bernstein JA, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. Treatment response after repeated administration of C1 esterase inhibitor for successive acute hereditary angioedema attacks. Allergy Asthma Proc. 2012 Jul-Aug;33(4):354-61. doi: 10.2500/aap.2012.33.3589.
United States Hereditary Angioedema AssociationLink provided by the sponsor.

Related trials for Hereditary Angioedema

RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
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RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
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HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
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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.

Program Director study director

CSL Behring

14 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing C1 Esterase Inhibitor in people with Hereditary Angioedema, run by CSL Behring at 14 sites in 2 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 2 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 7 May 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United States, Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 23 Jun 2011). 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?

Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) (time frame: Up to 24 h after start of study treatment). The study lists 2 primary outcomes in all. 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 NCT00292981, which also lists 7 linked publications.

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 7 May 2015.