Hereditary AngioedemaNCT01397864
C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
An observational study of Hereditary Angioedema, run by Pharming Technologies B.V. at 17 sites in 13 countries.
Trial summary
This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- rhC1INH or pdC1INH
- How many people
- 181 participants
- Where
- 17 sitesin 13 countries
- Study dates
- Jul 2011to 10 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The aim is to recruit 300 patients treated with Ruconest. Additionally, the study will continue until 100 patients have been exposed to Ruconest for at least 3 attacks. Enrolment in the pdC1INH arm will be unrestricted.
Who may be able to join
- Decision to treat the HAE patient with C1 inhibitor (either Ruconest or pdC1INH)
- Patients must give written informed consent
Who cannot join
- A diagnosis of acquired C1INH deficiency (AAE)
Trial locations and contacts
17 sites in 13 countries.
17 sites in 13 countries
Croatia3 sites
General Hospital Sibenik
Šibenik
KBC Split
Split
CHC Sestre milosrdnice
Zagreb
Slovakia2 sites
Bratislava University Hospital
Bratislava
University Hospital Martin
Martin
Hungary1 site
Semmelweis University, 3rd Department of Internal Medicine
Budapest
About this study
Official title: C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks
This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.
Read the detailed description
see below
- Conditions
- Hereditary Angioedema
- Being tested
- rhC1INH or pdC1INH
- Keywords
- Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH, registry
- 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.
rhC1INH or pdC1INHDrug
Also called Ruconest
C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest)
| Study group | Role | What they receive |
|---|---|---|
| Hereditary Angioedema | Not described Receives: Drug: rhC1INH or pdC1INH |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks
Measured: December 2019
Secondary outcomes (1)
To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)
Measured: December 2019
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2011 (estimated)
- Study startJul 2011
- Primary completion18 Oct 2024
- Study completion10 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 181 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 Technologies BV
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01397864
- Sponsor's protocol ID
- C1 1412
- Status last verified
- Apr 2025
- First submitted
- 18 Jul 2011
- Last updated
- 8 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks
- ClinicalTrials.gov ID
- NCT01397864
- Acronym / sponsor ID
- C1 1412
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary Angioedema, HAE, Angioedema, Recombinant C1 Inhibitor, rhC1INH, registry
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 181 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Study start
- Jul 2011 (actual)
- Primary completion
- 18 Oct 2024 (actual)
- Study completion
- 10 Dec 2024 (actual)
- First posted
- 20 Jul 2011 (estimated)
- Results
- Not posted in the source record
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 non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.
Detailed description
see below
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hereditary Angioedema | – | Drug: rhC1INH or pdC1INH |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The aim is to recruit 300 patients treated with Ruconest. Additionally, the study will continue until 100 patients have been exposed to Ruconest for at least 3 attacks. Enrolment in the pdC1INH arm will be unrestricted.
Key inclusion criteria (2 listed)
- Decision to treat the HAE patient with C1 inhibitor (either Ruconest or pdC1INH)
- Patients must give written informed consent
Key exclusion criteria (1 listed)
- A diagnosis of acquired C1INH deficiency (AAE)
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 |
|---|---|---|
| rhC1INH or pdC1INHAlso: RuconestC1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest) | Drug | Hereditary Angioedema |
Primary outcome
The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks
Time frame: December 2019
1 secondary outcome
To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)
Time frame: December 2019
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2025.
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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 Technologies BV
17 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema, run by Pharming Technologies B.V. at 17 sites in 13 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 2 inclusion and 1 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 8 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Croatia, Norway, Slovakia, Bulgaria, Czechia and 8 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks (time frame: December 2019). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Apr 2025.