Hereditary AngioedemaPhase 3NCT01188564
Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
A Phase 3 study testing rhC1INH in people with Hereditary Angioedema, run by Pharming Technologies B.V. at 27 sites in 11 countries.
Trial summary
This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- rhC1INH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 27 sitesin 11 countries
- Study dates
- Jan 2011to Mar 2013
- 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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged at least 13 years
- Signed written informed consent
- Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
- Willingness and ability to comply with all protocol procedures
- Clinical symptoms of an eligible HAE attack with onset less than 5 hours before the time of initial evaluation
Who cannot join
- Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
- A diagnosis of acquired C1INH deficiency (AAE)
- Pregnancy, or breastfeeding, or current intention to become pregnant
- Treatment with any investigational drug in the past 30 days
- Known or suspected addiction to drug and/or alcohol abuse
- Suspicion for an alternate explanation of the symptoms other than acute HAE attack
Trial locations and contacts
27 sites in 11 countries.
27 sites in 11 countries
United States15 sites
Family Allergy and Asthma Center
Atlanta, Georgia, 30342
Institute for Asthma and Allergy, P.C.
Chevy Chase, Maryland, 20815
University of Cincinnati Physicians, Inc.
Cincinnati, Ohio, 45267
Optimed Research, LTD
Columbus, Ohio, 43235
AARA Research Center
Dallas, Texas, 75231
University of Texas - Medical Branch
Galveston, Texas, 77555
Allergy and Asthma Institute of the Valley
Granada Hills, California, 91344
Pennsylvania State- Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Allergy, Asthma & Immunology Clinic, P.A.
Irving, Texas, 75063
Baker Allergy, Asthma and Dermatology Research Center, LLC
Lake Oswego, Oregon, 97035
UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine
Los Angeles, California, 90095
Allergy, Asthma & Immunology, Assoc, Ltd.
Scottsdale, Arizona, 85251
Marycliff Allergy Specialists
Spokane, Washington, 99204
Asthma & Allergy Center - Washington University School of Medicine
St Louis, Missouri, 63141
USF Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613
Israel2 sites
Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division
Haifa, 31048
Allergy, Immunology & Angioedema Center,
Tel HaShomer, Ramat Gan, 52621
South Africa2 sites
Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute
Mowbray, 7700
Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre
Parktown, 2193
Bulgaria1 site
UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases
Sofia, 1527
Hungary1 site
Semmelweis University Faculty of Medicine, III Department of Internal Medicine
Budapest, H-1125
Italy1 site
Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna
Milan, 20157
North Macedonia1 site
P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology
Skopje, 1000
Poland1 site
Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna
Krakow, 31-531
Romania1 site
Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie
Târgu Mureş, 540103
About this study
Official title: A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE
This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.
Read the detailed description
HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.
rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.
Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.
Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.
- Conditions
- Hereditary Angioedema
- Being tested
- rhC1INH, Placebo (Saline)
- Keywords
- Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH
- 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.
rhC1INHDrug
One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater.
Placebo (Saline)Drug
One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
| Study group | Role | What they receive |
|---|---|---|
| rhC1INH | Experimental | Not described Receives: Drug: rhC1INH |
| Placebo (Saline) | Placebo comparator | Not described Receives: Drug: Placebo (Saline) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Beginning of Relief of Symptoms
Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which
- The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and;
- The patient reports the following answer for TEQ question 2: "Yes"; and,
- There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.
Measured: Patients observed for 24 hours
Secondary outcomes (1)
Time to Minimal Symptoms
The key secondary efficacy endpoint was the time to minimal symptoms at all locations. The time to achieving minimal symptoms was defined as an answer of "Yes" to TEQ question 3.
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2010 (estimated)
- Study startJan 2011
- Primary completionDec 2012
- Study completionMar 2013
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 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01188564
- Sponsor's protocol ID
- C1 1310
- Status last verified
- Jul 2015
- First submitted
- 24 Aug 2010
- Last updated
- 7 Aug 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Riedl 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 42159953
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE
- ClinicalTrials.gov ID
- NCT01188564
- Acronym / sponsor ID
- C1 1310
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Jan 2011
- Primary completion
- Dec 2012 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 25 Aug 2010 (estimated)
- Results
- Posted 7 Aug 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.
Detailed description
HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.
rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.
Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.
Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rhC1INH | Experimental | Drug: rhC1INH |
| Placebo (Saline) | Placebo comparator | Drug: Placebo (Saline) |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Aged at least 13 years
- Signed written informed consent
- Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
- Willingness and ability to comply with all protocol procedures
- Clinical symptoms of an eligible HAE attack with onset less than 5 hours before the time of initial evaluation
Key exclusion criteria (6 listed)
- Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
- A diagnosis of acquired C1INH deficiency (AAE)
- Pregnancy, or breastfeeding, or current intention to become pregnant
- Treatment with any investigational drug in the past 30 days
- Known or suspected addiction to drug and/or alcohol abuse
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 |
|---|---|---|
| rhC1INHOne i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. | Drug | rhC1INH |
| Placebo (Saline)One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment | Drug | Placebo (Saline) |
Primary outcome
Time to Beginning of Relief of Symptoms
Time frame: Patients observed for 24 hours
Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which * The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and; * The patient reports the following answer for TEQ question 2: "Yes"; and, * There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.
1 secondary outcome
Time to Minimal Symptoms
Time frame: 24 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
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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.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rhC1INH in people with Hereditary Angioedema, run by Pharming Technologies B.V. at 27 sites in 11 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 5 inclusion and 6 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 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Israel, South Africa, Bulgaria, Canada and 6 more countries. 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 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 7 Aug 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?
Time to Beginning of Relief of Symptoms (time frame: Patients observed for 24 hours). 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 NCT01188564, 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 7 Aug 2015.