Hereditary AngioedemaBOISTERNNCT04957641
A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema
An observational study of Hereditary Angioedema, run by Takeda at 36 sites in 22 countries.
Trial summary
This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema
- How many people
- 221 participants
- Where
- 36 sitesin 22 countries
- Study dates
- 21 Apr 2022to 13 Jan 2023
- 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: Participants aged greater than or equal to (>=) 12 years with HAE type I or type II suffering from recurrent attacks will be enrolled in this study.
Who may be able to join
- Participant aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- Physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- Participant had at least one documented HAE attack during the eligibility period.
- Participant is not adequately or sub-optimally controlled according to treating physician.
- Participant (or parent/legal guardian) is willing and able to comply with the study requirements and provides informed consent or assent.
Who cannot join
- Participant enrolled in a therapeutic investigational drug or device trial during the observation period.
- Participant initiated long-term prophylaxis with Takhzyro® at any time since diagnosis.
Trial locations and contacts
36 sites in 22 countries.
36 sites in 22 countries
Israel4 sites
Barzilai Medical Center
Ashkelon
Bnai Zion Medical Center
Haifa
Sheba Medical Center - PPDS
Ramat Gan
Tel Aviv Sourasky Medical Center PPDS
Tel Aviv
Greece3 sites
Navy Hospital of Athens
Athens, 115 21
Laiko General Hospital of Athens
Athens, 115 27
University General Hospital of Larissa
Larissa, 412 21
Portugal3 sites
Centro Hospitalar E Universitário de Coimbra EPE
Coimbra, 3004-561
Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria
Lisbon, 1649-028
Centro Hospitalar de São João, E.P.E.
Porto, 4200-319
Spain3 sites
Hospital Universitario Cruces
Barakaldo, 48903
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, 08035
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
Canada2 sites
McMaster University Medical Centre
Hamilton, ON L8N 3Z5
Vancouver Allergy Clinic
Vancouver, British Columbia, BCV5Y1K2
Croatia2 sites
General Hospital Sibenik
Šibenik, 22000
Klinicki bolnicki centar Zagreb
Zagreb, 10000
Czechia2 sites
Fakultni nemocnice Hradec Kralove
Hradec Králové, 500 05
Fakultni nemocnice v Motole
Prague, 150 06
Latvia2 sites
Pauls Stradins Clinical University Hospital
Riga, LV-1002
Centre of Investigation and Treatment of Allergic Diseases
Riga, LV-1003
Lithuania2 sites
Hospital of Lithuanian University of Health Sciences Kauno klinikos
Kaunas, 50161
Vilnius University Hospital Santaros Klinikos
Vilnius, 08410
Austria1 site
Medizinische Universitat Wien (Medical University of Vienna)
Vienna, 1090
Bulgaria1 site
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, 1431
North Macedonia1 site
Medical Faculty of Ss. Cyril and Methodius Univers
Skopje, 1000
Slovenia1 site
Bolnisnica Golnik - Klinicni Oddelek za Pljucne Bolezni In Alergijo
Golnik, 4204
About this study
Official title: A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II
This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost.
The main aims of this study are as follows:
Read more
- to learn how often, how severe and where on the body HAE attacks occur.
- to describe how HAE prophylaxis and on-demand medicines are prescribed and used. (Prophylaxis medicines prevent a bleed from happening and on-demand medicines treat a bleed when it occurs.)
This study is about collecting data only; participants will not receive treatment as part of this study. Existing data available in the participant's medical records will be collected. Participants will be asked to complete an electronic questionnaire either on a website or by using an app on their mobile phone; data from this questionnaire will also be collected.
Participants do not need to visit their doctor in addition to their normal visits.
- Conditions
- Hereditary Angioedema
- 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 HAE | Participants' data will be collected retrospectively from electronic medical records using chart review of de-identified data on participant demographics, HAE medical history and information on diagnostics, treatments and disease course assessments that are routinely performed in accordance with current guidelines and/or local standard of care which are entered into an Electronic data capture (EDC) system over multiple data collection waves up to 6 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With HAE Attacks Occurrence During Study Period
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). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) during study period will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period
HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening will be reported.
Measured: Throughout the study period (up to 6 months)
Time Interval Between HAE Attacks During Study Period
Time interval between HAE attacks during the study period will be reported.
Measured: Throughout the study period (up to 6 months)
Duration of Each HAE Attacks During Study Period
Duration of each HAE attacks during study period will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Based on Type of HAE Prophylactic Treatments
Number of participants based on type of HAE prophylactic treatment will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment
Number of participants based on long-term or short-term HAE prophylactic treatment will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation
Number of participants categorized based on reasons for HAE prophylactic treatment initiation will be reported.
Measured: Throughout the study period (up to 6 months)
Duration of HAE Prophylactic Treatment
Duration of HAE prophylactic treatment includes treatment start and end dates will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment
Number of participants categorized based on dose and route of administration for HAE prophylactic treatment will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation
Number of participants categorized based on reasons for HAE prophylactic treatment discontinuation will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Based on Type of HAE On-demand Treatment
Number of participants based on type of HAE on-demand treatment will be reported.
Measured: Throughout the study period (up to 6 months)
Duration of HAE On-demand Treatment
Duration of HAE on-demand treatment includes treatment start and end dates will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment
Number of participants categorized based on dose, route of administration and setting of administration for HAE on-demand treatment will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Associated Between Prescriptions Patterns and HAE Attacks
Number of participants with association between prescriptions patterns and HAE attacks related to frequency or severity. Generalized linear models (GLM) will be used to evaluate the association of outcomes of interest with relevant covariates between prescription pattern, HAE attacks, frequency and severity of HAE attacks will be reported.
Measured: Throughout the study period (up to 6 months)
Secondary outcomes (11)
Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)
HRU is measured by the number of visits to HCP will be reported.
Measured: Throughout the study period (up to 6 months)
HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks
Number of participants with ER/ED visits due to HAE attacks will be reported.
Measured: Throughout the study period (up to 6 months)
HRU: Number of Participants With Hospitalizations due to HAE Attacks
Number of participants with hospitalizations due to HAE attacks will be reported.
Measured: Throughout the study period (up to 6 months)
Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire
The AE-QoL was developed to measure HRQoL in participants with recurrent angioedema. It is a self-administered patient-reported outcome instrument with a recall period of four weeks. There are 17 items across four domains: functioning (four items), fatigue/mood (five items), fears/shame (six items), and food (two items). Responses use a 5-point Likert scale ranging from 1 (Never) to 5 (Very Often). Global scores range from 0 to 100 and scores by domains range from 0 to 100. An overall score is calculated and a higher score indicates lower quality of life.
Measured: Throughout the study period (up to 6 months)
HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire
The EQ-5D-5L descriptive system assesses health in five dimensions (Mobility, Self-Care, Usual Activities, Pain / Discomfort, Anxiety / Depression), each of which has five levels of response (no problems, slight problems, moderate problems, severe problems, extreme problems/unable to). This part of the EQ-5D questionnaire provides a descriptive profile that can be used to generate a health state profile. Health state index scores generally range from less than 0 (where 0 is the value of a health state equivalent to dead; negative values representing values as worse than dead) to 1 (the value of full health), with higher scores indicating higher health utility. The second part of the questionnaire consists of a visual analogue scale (VAS) on which the participant rates his/her perceived health from 0 (the worst imaginable health) to 100 (the best imaginable health).
Measured: Throughout the study period (up to 6 months)
HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)
The EQ-5D-Y is composed of two sections. The first section contains one question for each of the five dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. Participants select from three response levels (no problems, some problems, a lot of problems) for each dimension. A health state profile score can be calculated from the responses on these five questions. The second section includes the same visual analogue scale as the EQ-5D-5L. The scale is scored from 0-100. The reference to a high score indicates a better outcome of quality of life.
Measured: Throughout the study period (up to 6 months)
Number of Participants With HAE Attacks Occurrence in Relevant Subgroups
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). Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) in relevant subgroups will be reported.
Measured: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups
HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).
Measured: Throughout the study period (up to 6 months)
Time Interval Between HAE Attacks in Relevant Subgroups
Time interval between HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).
Measured: Throughout the study period (up to 6 months)
Duration of Each HAE Attacks in Relevant Subgroups
Duration of each HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).
Measured: Throughout the study period (up to 6 months)
HAE Attacks Prescription Pattern in Relevant Subgroups
Prescription pattern of HAE attacks medication by drug class in relevant participant subgroups during study period. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).
Measured: Throughout the study period (up to 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2021
- Study start21 Apr 2022
- Primary completion13 Jan 2023
- Study completion13 Jan 2023
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 AngioedemaStudies 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
- 221 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
- NCT04957641
- Sponsor's protocol ID
- TAK-743-4009
- Other IDs
- MACS-2020-080401
- Acronym
- BOISTERN
- Status last verified
- Jun 2023
- First submitted
- 5 Jul 2021
- Last updated
- 26 Jun 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
Farkas H, Aygoren-Pursun E, Ebo DG, Bara N, Psarros F, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema. World Allergy Organ J. 2025 Sep 1;18(9):101100. doi: 10.1016/j.waojou.2025.101100. eCollection 2025 Sep.
PubMed 40949786
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II
- ClinicalTrials.gov ID
- NCT04957641
- Acronym / sponsor ID
- BOISTERN / TAK-743-4009
- Conditions
- Hereditary Angioedema
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 221 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 21 Apr 2022 (actual)
- Primary completion
- 13 Jan 2023 (actual)
- Study completion
- 13 Jan 2023 (actual)
- First posted
- 12 Jul 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.
This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost.
The main aims of this study are as follows:
- to learn how often, how severe and where on the body HAE attacks occur.
- to describe how HAE prophylaxis and on-demand medicines are prescribed and used. (Prophylaxis medicines prevent a bleed from happening and on-demand medicines treat a bleed when it occurs.)
This study is about collecting data only; participants will not receive treatment as part of this study. Existing data available in the participant's medical records will be collected. Participants will be asked to complete an electronic questionnaire either on a website or by using an app on their mobile phone; data from this questionnaire will also be collected.
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 HAE | – | –Participants' data will be collected retrospectively from electronic medical records using chart review of de-identified data on participant demographics, HAE medical history and information on diagnostics, treatments and disease course assessments that are routinely performed in accordance with current guidelines and/or local standard of care which are entered into an Electronic data capture (EDC) system over multiple data collection waves up to 6 months. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants aged greater than or equal to (>=) 12 years with HAE type I or type II suffering from recurrent attacks will be enrolled in this study.
Key inclusion criteria (5 listed)
- Participant aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- Physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- Participant had at least one documented HAE attack during the eligibility period.
- Participant is not adequately or sub-optimally controlled according to treating physician.
- Participant (or parent/legal guardian) is willing and able to comply with the study requirements and provides informed consent or assent.
Key exclusion criteria (2 listed)
- Participant enrolled in a therapeutic investigational drug or device trial during the observation period.
- Participant initiated long-term prophylaxis with Takhzyro® at any time since diagnosis.
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
Number of Participants With HAE Attacks Occurrence During Study Period
Time frame: Throughout the study period (up to 6 months)
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). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) during study period will be reported.
Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period
Time frame: Throughout the study period (up to 6 months)
HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening will be reported.
Time Interval Between HAE Attacks During Study Period
Time frame: Throughout the study period (up to 6 months)
Time interval between HAE attacks during the study period will be reported.
Duration of Each HAE Attacks During Study Period
Time frame: Throughout the study period (up to 6 months)
Duration of each HAE attacks during study period will be reported.
Number of Participants Based on Type of HAE Prophylactic Treatments
Time frame: Throughout the study period (up to 6 months)
Number of participants based on type of HAE prophylactic treatment will be reported.
Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment
Time frame: Throughout the study period (up to 6 months)
Number of participants based on long-term or short-term HAE prophylactic treatment will be reported.
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation
Time frame: Throughout the study period (up to 6 months)
Number of participants categorized based on reasons for HAE prophylactic treatment initiation will be reported.
Duration of HAE Prophylactic Treatment
Time frame: Throughout the study period (up to 6 months)
Duration of HAE prophylactic treatment includes treatment start and end dates will be reported.
Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment
Time frame: Throughout the study period (up to 6 months)
Number of participants categorized based on dose and route of administration for HAE prophylactic treatment will be reported.
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation
Time frame: Throughout the study period (up to 6 months)
Number of participants categorized based on reasons for HAE prophylactic treatment discontinuation will be reported.
Number of Participants Based on Type of HAE On-demand Treatment
Time frame: Throughout the study period (up to 6 months)
Number of participants based on type of HAE on-demand treatment will be reported.
Duration of HAE On-demand Treatment
Time frame: Throughout the study period (up to 6 months)
Duration of HAE on-demand treatment includes treatment start and end dates will be reported.
Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment
Time frame: Throughout the study period (up to 6 months)
Number of participants categorized based on dose, route of administration and setting of administration for HAE on-demand treatment will be reported.
Number of Participants Associated Between Prescriptions Patterns and HAE Attacks
Time frame: Throughout the study period (up to 6 months)
Number of participants with association between prescriptions patterns and HAE attacks related to frequency or severity. Generalized linear models (GLM) will be used to evaluate the association of outcomes of interest with relevant covariates between prescription pattern, HAE attacks, frequency and severity of HAE attacks will be reported.
11 secondary outcomes
Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)
Time frame: Throughout the study period (up to 6 months)
HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks
Time frame: Throughout the study period (up to 6 months)
HRU: Number of Participants With Hospitalizations due to HAE Attacks
Time frame: Throughout the study period (up to 6 months)
Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire
Time frame: Throughout the study period (up to 6 months)
HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire
Time frame: Throughout the study period (up to 6 months)
HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)
Time frame: Throughout the study period (up to 6 months)
Number of Participants With HAE Attacks Occurrence in Relevant Subgroups
Time frame: Throughout the study period (up to 6 months)
Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups
Time frame: Throughout the study period (up to 6 months)
Time Interval Between HAE Attacks in Relevant Subgroups
Time frame: Throughout the study period (up to 6 months)
Duration of Each HAE Attacks in Relevant Subgroups
Time frame: Throughout the study period (up to 6 months)
HAE Attacks Prescription Pattern in Relevant Subgroups
Time frame: Throughout the study period (up to 6 months)
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 26 Jun 2023.
- Derived from this study: PubMed 40949786Farkas H, Aygoren-Pursun E, Ebo DG, Bara N, Psarros F, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema. World Allergy Organ J. 2025 Sep 1;18(9):101100. doi: 10.1016/j.waojou.2025.101100. eCollection 2025 Sep.
- 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
36 sites in 22 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 Takeda at 36 sites in 22 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 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 26 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in Israel, Greece, Portugal, Spain, Canada and 17 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?
Number of Participants With HAE Attacks Occurrence During Study Period (time frame: Throughout the study period (up to 6 months)). The study lists 14 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 NCT04957641, 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 26 Jun 2023.