Hereditary AngioedemaHAEKANCT04091113
Hereditary Angioedema Kininogen Assay
An observational study of Hereditary Angioedema, run by CENTOGENE GmbH Rostock at 7 sites in Germany.
Trial summary
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema
- How many people
- 59 participants
- Where
- 7 sitesGermany
- Study dates
- 1 Sep 2019to 31 Dec 2021
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Hereditary Angioedema type 1/2 patients
Who may be able to join
- Informed consent is obtained from the participant
- The patient with diagnosis of Hereditary Angioedema (HAE) type 1/2 based on international guidelines
- The patient experienced ≥4 HAE attacks within last 12 month before enrolment in the study
- The participant is older than 18 years old
Who cannot join
- Inability to provide informed consent
- The patient is not diagnosed with Hereditary Angioedema (HAE) type 1/2
- The patient experienced ˂ 4 HAE attacks within last 12 month before enrolment in the study
- The participant is younger than 18 years old
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Germany7 sites
Charité - Universitätsmedizin Berlin
Berlin, 10117
Klinik für Hals-Nasen-Ohrenheilkunde, Universitätsklinikum Düsseldorf,
Düsseldorf, 40225
Klinikum der Johann-Wolfgang-Goethe-Universität
Frankfurt, 60596
Medizinische Hochschule Hannover
Hanover, 30625
Universitäts-Hautklinik Leipzig
Leipzig, 04103
Hämophilie-Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
Universitätsklinikum Ulm
Ulm, 89075
About this study
Official title: Hereditary Angioedema Kininogen Assay: A Multicenter, Epidemiological, Observational Study.
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients
Read the detailed description
Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by recurrent tissue angioedema episodes, mainly caused by mutations in the SERPING1 gene that encodes C1 inhibitor (C1-INH), a protease involved in limiting bradykinin production. Low levels of C1-INH (HAE type 1) or dysfunctional C1-INH (HAE type 2) lead to bradykinin accumulation, resulting in capillary leakage and tissue swelling.
High Molecular Weight Kininogen (HMWK) proteolysis, by active plasma kallikrein, results in bradykinin and cHMWK generation.
The goal of this study is to explore the cHMWK concentrations in HAE type 1/2 patients, as a biomarker for this disease.
The HAEKA study is performed in collaboration with Shire. Shire is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary Angioedema, Cleaved high-molecular weight kininogen, Hereditary Angioedema Type I, Hereditary Angioedema Type II
- Medical subject headings
- Angioedemas, Hereditary, Hereditary Angioedema Types I and II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Participants with Hereditary Angioedema | Participants older than 18 years that are clinically diagnosed with Hereditary Angioedema type 1/2 and experienced ≥4 HAE attacks within last 12 month before the enrollment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).
Measured: 30 months
Secondary outcomes (1)
Studying cHMWK as a biomarker in HAE type 1/2 patients with edema attack, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment collected before/during/after angioedema attacks will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).
Measured: 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2019
- Study start1 Sep 2019
- Primary completion12 Sep 2021
- Study completion31 Dec 2021
Why it stopped: we will update quickly
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
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- CENTOGENE GmbH Rostock
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter Bauer, MD, Study chair, Centogene GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04091113
- Sponsor's protocol ID
- HAEKA 01-2019
- Acronym
- HAEKA
- Status last verified
- Mar 2022
- First submitted
- 6 Sep 2019
- Last updated
- 10 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hereditary Angioedema Kininogen Assay: A Multicenter, Epidemiological, Observational Study.
- ClinicalTrials.gov ID
- NCT04091113
- Acronym / sponsor ID
- HAEKA / HAEKA 01-2019
- Conditions
- Hereditary Angioedema
- Keywords
- Hereditary Angioedema, Cleaved high-molecular weight kininogen, Hereditary Angioedema Type I, Hereditary Angioedema Type II
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: we will update quickly
- Enrolment
- 59 participants (actual)
- Lead sponsor
- CENTOGENE GmbH RostockIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2019 (actual)
- Primary completion
- 12 Sep 2021 (actual)
- Study completion
- 31 Dec 2021 (actual)
- First posted
- 16 Sep 2019 (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.
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients
Detailed description
Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by recurrent tissue angioedema episodes, mainly caused by mutations in the SERPING1 gene that encodes C1 inhibitor (C1-INH), a protease involved in limiting bradykinin production. Low levels of C1-INH (HAE type 1) or dysfunctional C1-INH (HAE type 2) lead to bradykinin accumulation, resulting in capillary leakage and tissue swelling.
High Molecular Weight Kininogen (HMWK) proteolysis, by active plasma kallikrein, results in bradykinin and cHMWK generation.
The goal of this study is to explore the cHMWK concentrations in HAE type 1/2 patients, as a biomarker for this disease.
The HAEKA study is performed in collaboration with Shire. Shire is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with Hereditary Angioedema | – | –Participants older than 18 years that are clinically diagnosed with Hereditary Angioedema type 1/2 and experienced ≥4 HAE attacks within last 12 month before the enrollment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Hereditary Angioedema type 1/2 patients
Key inclusion criteria (4 listed)
- Informed consent is obtained from the participant
- The patient with diagnosis of Hereditary Angioedema (HAE) type 1/2 based on international guidelines
- The patient experienced ≥4 HAE attacks within last 12 month before enrolment in the study
- The participant is older than 18 years old
Key exclusion criteria (4 listed)
- Inability to provide informed consent
- The patient is not diagnosed with Hereditary Angioedema (HAE) type 1/2
- The patient experienced ˂ 4 HAE attacks within last 12 month before enrolment in the study
- The participant is younger than 18 years old
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
Time frame: 30 months
Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).
1 secondary outcome
Studying cHMWK as a biomarker in HAE type 1/2 patients with edema attack, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
Time frame: 30 months
Results and safety
The study was stopped early (reason given: we will update quickly). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2022.
- Individual participant dataSharing planned: No
Related trials for Hereditary Angioedema
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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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
Enrolling by invitationPhase 3
Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
Hereditary Angioedema (HAE)
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.
Peter Bauer, MD study chair
Centogene GmbH
7 sites 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 CENTOGENE GmbH Rostock at 7 sites in Germany.
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 4 inclusion and 4 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: stopped early, as of the sponsor's last update on 10 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany. 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 was stopped early (reason given: we will update quickly). Results are not available in the source record.
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?
Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment. (time frame: 30 months). 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 10 Mar 2022.