Hereditary Angioedema (HAE)INTEGRATEDNCT04861090
A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis
An observational study of Hereditary Angioedema (HAE), run by Takeda at 20 sites in 4 countries.
Trial summary
The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life. This includes the number of people that are attack-free when lanadelumab is given every 2 and every 4 weeks. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- How many people
- 207 participants
- Where
- 20 sitesin 4 countries
- Study dates
- 15 Sep 2021to 30 Jun 2022
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adult or adolescent participants (aged greater than or equal to [>=] 12 years) with HAE type I or type II who initiated LTP treatment with lanadelumab within a routine clinical setting.
Who may be able to join
- Participant is aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- Participant has a physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- Participant had initiated LTP with lanadelumab during the eligibility period.
- Participant provides informed consent or assent prior to the initiation of any study procedures (where required by local regulations).
Who cannot join
- Participant was enrolled in a therapeutic investigational drug or device trial during the observation period.
- Participant without documented HAE attacks in the pre-index period and/or without available participant diary or systematic documentation of HAE attacks in the medical records during the post index period.
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
France7 sites
CHU de Grenoble
Grenoble, 38043
CHRU Lille
Lille, 59800
Groupement Hospitalier Edouard Herriot
Lyon, 69437
CHU Montpellier - Hôpital St Eloi
Montpellier, 34295
Hôtel Dieu de Paris Hospital
Paris, 75181
Hôpital Saint Antoine
Paris, 75571
Centre Hospitalier Universitaire Hopitaux de Rouen
Rouen, 76031
Germany7 sites
Charitè Campus Mitte
Berlin, 10117
Universitätsklinikum Frankfurt
Frankfurt am Main, 60596
Medizinische Hochschule Hannover
Hanover, 30625
Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
Klinikum rechts der Isa der Technischen Universitaet Muenchen
Munich, 80802
Universitatsklinikum Munster
Münster, 48149
Universitätsklinikum Ulm
Ulm, 89075
Austria3 sites
LKH-Universitätsklinikum Klinikum Graz
Graz, 8036
Kepler Universitätsklinikum Linz
Linz, 4021
Medizinische Universitat Wien (Medical University of Vienna)
Vienna, 1090
Greece3 sites
Navy Hospital of Athens
Athens, 115 21
Laiko General Hospital of Athens
Athens, 115 27
University General Hospital of Larissa
Larissa, 15125
About this study
Official title: Retrospective, Observational Chart Review Study Evaluating Clinical Effectiveness and Disease/Treatment Management Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-world Setting
The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life. This includes the number of people that are attack-free when lanadelumab is given every 2 and every 4 weeks.
This study is about collecting existing data only; participants will not receive lanadelumab as part of this study. No new information will be collected during this study. Only data already available at the participant's doctor's office will be reviewed and collected for this study.
Participants do not need to visit their doctor in addition to their normal visits.
- Conditions
- Hereditary Angioedema (HAE)
- Medical subject headings
- Angioedemas, Hereditary
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 with HAE type I or type II who had initiated long-term prophylaxis (LTP) treatment with lanadelumab which was administered every two weeks (Q2W) or every four weeks (Q4W) or every six weeks (Q6W) or every eight weeks (Q8W) in accordance to Summary of Product Characteristics (SmPC), during a routine clinical setting will be followed up to 38 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Measured: Up to Month 12
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two Weeks
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every two weeks will be reported.
Measured: Up to Month 12
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every four weeks will be reported.
Measured: Up to Month 12
Secondary outcomes (4)
Percentage of Participants With Specific HAE Attack Occurrence
Percentage of participants with specific HAE attack occurrence (e.g. attacks of specific localization, attacks requiring on-demand medication, life-threatening attacks) will be reported.
Measured: Up to Month 12
Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment
Percentage of participants who are free of HAE attacks relative to prior treatment (e.g. prior prophylaxis treatment or on-demand medications) among participants treated with lanadelumab will be reported.
Measured: Up to Month 12
Number of Participants Characterized With Every Four Weeks Adjustment
Number of participants will be characterized with every four weeks adjustment (e.g. weight, age, duration of disease, history of life-threating attacks, length of attack-free interval during lanadelumab treatment) will be reported.
Measured: Every 4 weeks from start of treatment (Up to Month 12)
Number of Participants Characterized Based on Primary Reasons for Down Titration
Number of participants will be characterized based on primary reasons for down titration will be reported.
Measured: Up to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2021
- Study start15 Sep 2021
- Primary completion30 Jun 2022
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- 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
- Retrospective
- Enrolment
- 207 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04861090
- Sponsor's protocol ID
- TAK-743-4006
- Other IDs
- MACS-2020-081002
- Acronym
- INTEGRATED
- Status last verified
- Jan 2023
- First submitted
- 19 Apr 2021
- Last updated
- 13 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Publications
Magerl M, Bouillet L, Martinez-Saguer I, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: Multicountry INTEGRATED Observational Study. J Allergy Clin Immunol Pract. 2025 Feb;13(2):378-387.e2. doi: 10.1016/j.jaip.2024.12.008. Epub 2024 Dec 17.
PubMed 39701274
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective, Observational Chart Review Study Evaluating Clinical Effectiveness and Disease/Treatment Management Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-world Setting
- ClinicalTrials.gov ID
- NCT04861090
- Acronym / sponsor ID
- INTEGRATED / TAK-743-4006
- Conditions
- Hereditary Angioedema (HAE)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 207 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 15 Sep 2021 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 27 Apr 2021 (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.
The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life. This includes the number of people that are attack-free when lanadelumab is given every 2 and every 4 weeks.
This study is about collecting existing data only; participants will not receive lanadelumab as part of this study. No new information will be collected during this study. Only data already available at the participant's doctor's office will be reviewed and collected for this study.
Participants do not need to visit their doctor in addition to their normal visits.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants With Hereditary Angioedema | – | –Participants with HAE type I or type II who had initiated long-term prophylaxis (LTP) treatment with lanadelumab which was administered every two weeks (Q2W) or every four weeks (Q4W) or every six weeks (Q6W) or every eight weeks (Q8W) in accordance to Summary of Product Characteristics (SmPC), during a routine clinical setting will be followed up to 38 months. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adult or adolescent participants (aged greater than or equal to [>=] 12 years) with HAE type I or type II who initiated LTP treatment with lanadelumab within a routine clinical setting.
Key inclusion criteria (4 listed)
- Participant is aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- Participant has a physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- Participant had initiated LTP with lanadelumab during the eligibility period.
- Participant provides informed consent or assent prior to the initiation of any study procedures (where required by local regulations).
Key exclusion criteria (2 listed)
- Participant was enrolled in a therapeutic investigational drug or device trial during the observation period.
- Participant without documented HAE attacks in the pre-index period and/or without available participant diary or systematic documentation of HAE attacks in the medical records during the post index period.
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
Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab
Time frame: Up to Month 12
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two Weeks
Time frame: Up to Month 12
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every two weeks will be reported.
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks
Time frame: Up to Month 12
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every four weeks will be reported.
4 secondary outcomes
Percentage of Participants With Specific HAE Attack Occurrence
Time frame: Up to Month 12
Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment
Time frame: Up to Month 12
Number of Participants Characterized With Every Four Weeks Adjustment
Time frame: Every 4 weeks from start of treatment (Up to Month 12)
Number of Participants Characterized Based on Primary Reasons for Down Titration
Time frame: Up to Month 12
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 13 Jan 2023.
- Derived from this study: PubMed 39701274Magerl M, Bouillet L, Martinez-Saguer I, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: Multicountry INTEGRATED Observational Study. J Allergy Clin Immunol Pract. 2025 Feb;13(2):378-387.e2. doi: 10.1016/j.jaip.2024.12.008. Epub 2024 Dec 17.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Study Director study director
Takeda
20 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema (HAE), run by Takeda at 20 sites in 4 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 4 inclusion and 2 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 13 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in France, Germany, Austria, Greece. 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?
Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab (time frame: Up to Month 12). The study lists 3 primary outcomes in all. 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 NCT04861090, 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 13 Jan 2023.