Hereditary AngioedemaNCT03697187
Patient Registry to Evaluate the Real-world Safety of Ruconest®
An observational study of Hereditary Angioedema, run by Pharming Technologies B.V. at 1 site in United States.
Trial summary
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE. 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
- How many people
- 152 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jun 2018to 31 Jul 2021
- How long will I take part?
- 3 Years
- 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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Eligible patients will be 13 years of age and older, have a current diagnosis of HAE for which they have been prescribed and are currently being treated with Ruconest.
Who may be able to join
- Patient provides informed consent as documented on the Institutional Review Board (IRB) approved informed consent document (ICF). For patients aged between 13 and 17 years old, the method of consent with or without assent will be determined by the IRB.
- Patient is male or female and at least 13 years of age at the time of providing consent / assent.
- Patient has been prescribed Ruconest for HAE.
Who cannot join
- 1. Patient is receiving HAE therapy as part of a clinical trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
The US Hereditary Angioedema Association
Fairfax, Virginia, 22030
About this study
Official title: An Observational Patient Registry to Evaluate the Real-world Safety of Ruconest® (C1 Esterase Inhibitor [Recombinant]) for the Treatment of Hereditary Angioedema
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
Read the detailed description
See below.
- Conditions
- Hereditary Angioedema
- Being tested
- rhC1INH
- Keywords
- Hereditary Angioedema, HAE, Ruconest, rhC1INH, Registry, Pharming
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhC1INHDrug
Also called Ruconest
Recombinant human C1 inhibitor
| Study group | Role | What they receive |
|---|---|---|
| Hereditary Angioedema | Patients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH). Receives: Drug: rhC1INH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest.
In addition, extent of exposure to Ruconest and concomitant medications taken for HAE will be summarized. Concomitant medications will be limited to those specifically indicated for treatment AEs or treatment of symptoms of HAE, for example C1-inhibitors, epinephrine, IV fluids, etc. AEs will be summarized as incidence per 10,000 person-days where each patient counts only once for multiple events of the same System Organ Class and preferred terms within the 30-day evaluation period. The person-time duration for each AE for each patient is the time period (in days) between the dose of Ruconest and AE onset.
Measured: 3 years
Secondary outcomes (1)
AEs occurring during pregnancy or lactation and for breastfed infants will be summarized separately using the same methods as described in the primary outcome measure.
Any pregnant woman who treats with Ruconest while on the Registry will be followed up to twelve weeks post-delivery or termination. Those treated with Ruconest while actively nursing will be followed until end of lactation.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2018
- Study start30 Jun 2018
- Primary completion30 Jun 2021
- Study completion31 Jul 2021
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
- 152 participants (actual)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- US Hereditary Angioedema Association
- Responsible party
- Sponsor
- Investigators
- Anurag Relan, MD, Study director, Pharming Technologies BV
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03697187
- Sponsor's protocol ID
- C1 1414
- Status last verified
- Mar 2020
- First submitted
- 9 Aug 2018
- Last updated
- 5 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Patient Registry to Evaluate the Real-world Safety of Ruconest® (C1 Esterase Inhibitor [Recombinant]) for the Treatment of Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT03697187
- Acronym / sponsor ID
- C1 1414
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary Angioedema, HAE, Ruconest, rhC1INH, Registry, Pharming
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 152 participants (actual)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Collaborators
- US Hereditary Angioedema Association
- Study start
- 30 Jun 2018 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 31 Jul 2021 (actual)
- First posted
- 5 Oct 2018 (actual)
- 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 prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
Detailed description
See below.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hereditary Angioedema | – | Drug: rhC1INHPatients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH). |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Eligible patients will be 13 years of age and older, have a current diagnosis of HAE for which they have been prescribed and are currently being treated with Ruconest.
Key inclusion criteria (3 listed)
- Patient provides informed consent as documented on the Institutional Review Board (IRB) approved informed consent document (ICF). For patients aged between 13 and 17 years old, the method of consent with or without assent will be determined by the IRB.
- Patient is male or female and at least 13 years of age at the time of providing consent / assent.
- Patient has been prescribed Ruconest for HAE.
Key exclusion criteria (1 listed)
- 1. Patient is receiving HAE therapy as part of a clinical trial.
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 |
|---|---|---|
| rhC1INHAlso: RuconestRecombinant human C1 inhibitor | Drug | Hereditary Angioedema |
Primary outcome
Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest.
Time frame: 3 years
In addition, extent of exposure to Ruconest and concomitant medications taken for HAE will be summarized. Concomitant medications will be limited to those specifically indicated for treatment AEs or treatment of symptoms of HAE, for example C1-inhibitors, epinephrine, IV fluids, etc. AEs will be summarized as incidence per 10,000 person-days where each patient counts only once for multiple events of the same System Organ Class and preferred terms within the 30-day evaluation period. The person-time duration for each AE for each patient is the time period (in days) between the dose of Ruconest and AE onset.
1 secondary outcome
AEs occurring during pregnancy or lactation and for breastfed infants will be summarized separately using the same methods as described in the primary outcome measure.
Time frame: 3 years
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 5 Apr 2024.
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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
1 site in 1 country: 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 1 site in United States.
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 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 5 Apr 2024. 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 United States. 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?
Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest. (time frame: 3 years). 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 5 Apr 2024.