Hereditary AngioedemaPhase 2/3NCT00225147
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
A Phase 2/3 study testing Recombinant Human C1 Inhibitor in people with Hereditary Angioedema and Angioneurotic Edema, run by Pharming Technologies B.V. at 1 site in Netherlands.
Trial summary
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema, Angioneurotic Edema
- What is being tested?
- Recombinant Human C1 Inhibitor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 1 siteNetherlands
- Study dates
- Jul 2005to Jan 2010
- Phase
- Phase 2/3
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Main Inclusion Criteria:
- Clear clinical and laboratory diagnosis of HAE
- Plasma level of functional C1INH of less than 50% of normal
- Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack
Main Exclusion Criteria:
- Acquired angioedema
- Pregnancy or breastfeeding
- Treatment with any investigational drug within prior 30 days
- Body weight >120 kg
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
For information on sites please contact Pharming Medical Affairs Department
Leiden, 2300 AL
About this study
Official title: A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
Read the detailed description
A prospectively planned interim analysis will be performed on the double-blind data.
- Conditions
- Hereditary Angioedema, Angioneurotic Edema
- Being tested
- Recombinant Human C1 Inhibitor, placebo
- Medical subject headings
- Angioedemas, Hereditary, Angioedema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human C1 InhibitorDrug
Also called "rhC1INH", Ruconest, conestat alfa
IV
placeboDrug
Also called saline, physiological salt solution
saline solution
| Study group | Role | What they receive |
|---|---|---|
| 100 IU/kg rhC1INH | Experimental | 100 IU/kg Recombinant human C1 inhibitor Receives: Drug: Recombinant Human C1 Inhibitor |
| 50 IU/kg rhC1INH | Experimental | 50 IU/kg Recombinant human C1 inhibitor Receives: Drug: Recombinant Human C1 Inhibitor |
| Saline | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Beginning of Relief of Symptoms
The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
Measured: up to 48 hours after study drug administration
Secondary outcomes (1)
Time to Minimal Symptoms
The time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.
Measured: up to 48 hours after study drug administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2005 (estimated)
- Study startJul 2005
- Primary completionOct 2009
- Study completionJan 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 (4)Papers the sponsor has linked to this study.
- Other trials for Hereditary AngioedemaStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anurag Relan, MD, Study director, Pharming Group N.V.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00225147
- Sponsor's protocol ID
- C1 1205-01
- Status last verified
- Feb 2013
- First submitted
- 20 Sep 2005
- Last updated
- 22 Feb 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.
PubMed 20920772Riedl MA, Narsipur N, Jones D, Tachdjian R, Relan A, Kilvert H, Aiello E, Habimana K, Roskell N, Gough A, Harper JR, Li HH, Harrington A. Post Hoc Analysis of Recombinant C1 Inhibitor Clinical Data Using Contemporary Endpoints for Hereditary Angioedema. Adv Ther. 2026 Aug;43(8):3506-3520. doi: 10.1007/s12325-026-03625-0. Epub 2026 May 20.
PubMed 42159953Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.
PubMed 28284978Riedl MA, Levy RJ, Suez D, Lockey RF, Baker JW, Relan A, Zuraw BL. Efficacy and safety of recombinant C1 inhibitor for the treatment of hereditary angioedema attacks: a North American open-label study. Ann Allergy Asthma Immunol. 2013 Apr;110(4):295-9. doi: 10.1016/j.anai.2013.02.007. Epub 2013 Mar 6.
PubMed 23535096
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT00225147
- Acronym / sponsor ID
- C1 1205-01
- Conditions
- Hereditary Angioedema, Angioneurotic Edema
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Jul 2005
- Primary completion
- Oct 2009 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 23 Sep 2005 (estimated)
- Results
- Posted 31 Aug 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
Detailed description
A prospectively planned interim analysis will be performed on the double-blind data.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 100 IU/kg rhC1INH | Experimental | Drug: Recombinant Human C1 Inhibitor100 IU/kg Recombinant human C1 inhibitor |
| 50 IU/kg rhC1INH | Experimental | Drug: Recombinant Human C1 Inhibitor50 IU/kg Recombinant human C1 inhibitor |
| Saline | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Recombinant Human C1 InhibitorAlso: "rhC1INH", Ruconest, conestat alfaIV | Drug | 100 IU/kg rhC1INH, 50 IU/kg rhC1INH |
| placeboAlso: saline, physiological salt solutionsaline solution | Drug | Saline |
Primary outcome
Time to Beginning of Relief of Symptoms
Time frame: up to 48 hours after study drug administration
The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
1 secondary outcome
Time to Minimal Symptoms
Time frame: up to 48 hours after study drug administration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 20920772Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.
- Derived from this study: PubMed 42159953Riedl MA, Narsipur N, Jones D, Tachdjian R, Relan A, Kilvert H, Aiello E, Habimana K, Roskell N, Gough A, Harper JR, Li HH, Harrington A. Post Hoc Analysis of Recombinant C1 Inhibitor Clinical Data Using Contemporary Endpoints for Hereditary Angioedema. Adv Ther. 2026 Aug;43(8):3506-3520. doi: 10.1007/s12325-026-03625-0. Epub 2026 May 20.
- Derived from this study: PubMed 28284978Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.
- Derived from this study: PubMed 23535096Riedl MA, Levy RJ, Suez D, Lockey RF, Baker JW, Relan A, Zuraw BL. Efficacy and safety of recombinant C1 inhibitor for the treatment of hereditary angioedema attacks: a North American open-label study. Ann Allergy Asthma Immunol. 2013 Apr;110(4):295-9. doi: 10.1016/j.anai.2013.02.007. Epub 2013 Mar 6.
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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.
Anurag Relan, MD study director
Pharming Group N.V.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Recombinant Human C1 Inhibitor in people with Hereditary Angioedema and Angioneurotic Edema, run by Pharming Technologies B.V. at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 22 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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 31 Aug 2012). 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 Beginning of Relief of Symptoms (time frame: up to 48 hours after study drug administration). 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 NCT00225147, which also lists 4 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 22 Feb 2013.