Hereditary Angioedema (HAE)ENABLENCT04130191
A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II
An observational study of Hereditary Angioedema (HAE), run by Shire at 19 sites in 7 countries.
Trial summary
The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started. Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Angioedema (HAE)
- How many people
- 140 participants
- Where
- 19 sitesin 7 countries
- Study dates
- 11 Dec 2019to 9 Sep 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: Participants with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling.
Who may be able to join
- Voluntarily provision of written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
- Initiation of treatment with lanadelumab in accordance with current product labelling. Decision to start treatment with lanadelumab must be made before and be independent from enrollment in the study.
- Availability of information for HAE attack-related variables assessed at study enrollment for the previous three months.
- Ability to use a smartphone for data collection in this study.
Who cannot join
- Inability to provide written, signed, and dated informed consent.
- Participation in the study not considered appropriate by the treating physician/investigator.
- Contraindication to treatment with lanadelumab according to current product labelling.
- Pregnant or breastfeeding.
- Concomitant exposure to any investigational therapy (including medications not used for HAE).
- Current or planned participation in other interventional studies.
Trial locations and contacts
19 sites in 7 countries.
19 sites in 7 countries
Germany8 sites
Charité - Universitätsmedizin Berlin
Berlin, 10117
Universitatsklinikum Dusseldorf
Düsseldorf, 40225
Klinikum der Johann-Wolfgang Goethe-Universitat
Frankfurt am Main, Hesse, 60590
Universitatsklinikum Schleswig-Holstein
Lübeck, 23538
Hämophilie Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
Klinikum rechts der Isar der Technischen Universität München
München, 81675
Universitätsklinikum Münster
Münster, 48149
Universitätsklinikum Ulm
Ulm, 89075
Italy3 sites
ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco
Milan, 20157
Azienda Ospedaliera Universitaria Federico II
Naples, Campania, 80131
Azienda Ospedale Università Padova
Padova, 35128
Israel2 sites
Bnai Zion Medical Center
Haifa, 31048
Sheba Medical Center - PPDS
Ramat Gan, 52621
Spain2 sites
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, 08035
Hospital Universitario de Bellvitge
L'Hospitalet de Llobregat, Barcelona, 08907
Switzerland2 sites
Universitätsspital Basel
Basel, 4031
Luzerner Kantonsspital LUKS
Lucerne, 6000
About this study
Official title: A Three-year, Non-interventional, Prospective, Multicenter Study to Evaluate the Long- Term Effectiveness of Lanadelumab in Real-world Clinical Practice (ENABLE)
The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started.
Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started the study before 1 March 2021) will be collected for 36 months. Participants will report information in a smartphone application at study start and for the next 3 months and then every 6 months until the study ends; data will also be collected by the study doctor during routine clinic 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 (HAE) | Participants who initiate treatment with lanadelumab according to current product labelling will be enrolled and followed for up to 24 or 36 months (depending on their enrollment date). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks
HAE attack is defined as a discrete episode during which the participant progress from no angioedema to symptoms of angioedema. Incidence rate ratio of on-treatment patient-reported HAE attacks up to three years after initiation of lanadelumab compared to the history of HAE attacks of last three months prior to lanadelumab use will be reported.
Measured: Up to 36 months
Secondary outcomes (21)
Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70
Rate of participant-reported HAE attacks during treatment with lanadelumab from day 70 will be assessed.
Measured: From Day 70 up to 36 months
Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks
The overall severity of attack is determined using following definitions: mild (Temporary or mild discomfort), moderate (Activity limited mildly or moderately. Some assistance may be needed), severe (Activity considerably limited, assistance needed).
Measured: Up to 36 months
Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location
Number of on-treatment participant-reported HAE attacks based on anatomical (peripheral, abdominal, laryngeal) location will be assessed.
Measured: Up to 36 months
Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used
Proportion of HAE attacks for which participants use on-demand therapy will be assessed.
Measured: Up to 36 months
Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used
Time to first HAE attack for which on-demand therapy is used will be assessed.
Measured: Up to 36 months
Proportion of Hereditary Angioedema (HAE) Attacks Requiring Visit to an Healthcare Provider (HCP), Access to an Emergency Room (ER), or Hospitalization
Proportion of HAE Attacks requiring visit to HCP, access to an ER, or hospitalization will be assessed.
Measured: Up to 36 months
Angioedema Quality of Life (AE-QoL)
The AE-QoL is developed to measure participant-reported health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. It is a self-administered participant related outcome (PRO) with a recall period of 4 weeks. There are 17 items across 4 domains: functioning (4 items), fatigue/mood (5 items), fears/shame (6 items), and food (2 items). Responses use a 5-point Likert scale ranging from 'never' to 'very often.' Global scores range from 0 to 100 and scores by domains range from 0 to 100, where 0 indicates highest quality of life and 100 lowest quality of life.
Measured: Up to 36 months
Fatigue Severity Scale (FSS)
Participant reported fatigue will be measured by the FSS. The FSS is a 9-item questionnaire measuring participants fatigue severity and its impact on motivation, exercise, physical functioning, and work and social life. It uses a 7-point Likert scale response (1 = strongly disagree, 7 = strongly agree) and the final score is obtained as mean of the response scores to the individual questions.
Measured: Up to 36 months
Hospital Anxiety and Depression Scale (HADS)
The HADS is a self-rating scale developed to detect the levels of depression and anxiety experienced by participants. It is a self-administered PRO composed of 14 items, of which seven relate to anxiety and seven relate to depression. Each item on the questionnaire is scored from 0 to 3, which means that a person can score between 0 and 21 for either anxiety or depression. Recommended cut-off scores are 8 to 10 for doubtful cases and greater than or equal to (> or =) 11 for definite cases.
Measured: Up to 36 months
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)
The TSQM is a generic questionnaire to measure participants satisfaction with medication using yes/no and 5- or 7-point Likert scale response options. It is a self-administered PRO instrument designed for adults aged 18 years or older with a recall period of two to three weeks, or since the last medication use. Version TSQM-9 includes three domains: effectiveness (three items), convenience (three items), and global satisfaction scale (three items).
Measured: Up to 36 months
Work Productivity and Activity Impairment: General Health (WPAI:GH)
The WPAI:GH is a generic questionnaire to measure the effect of general health and symptom severity on work productivity and regular activities during the past seven days. It can be self or interviewer-administered to adults aged 18 years or older. This six-item PRO instrument covers work (five items) and daily activities (one item) using yes/no or numerical answers (number of hours).
Measured: Up to 36 months
Adult Carer Quality of Life (AC-QoL) Questionnaire
The AC-QoL is a 40-item tool that measures the overall QoL for adult carers, and subscale scores for eight domains of QoL: support for caring, caring choice, caring stress, money matters; personal growth; sense of value, ability to care, and carer satisfaction. It is self-administered to adult carers and should take no longer than 10 minutes to complete. Scores range from 0 to 120, with higher scores indicating greater QoL.
Measured: Up to 36 months
Dose of Lanadelumab
Dose of lanadelumab used during the study will be assessed.
Measured: From start of the study up to 36 months
Frequency of Administration of Lanadelumab
Frequency of lanadelumab injections during the study will be assessed.
Measured: From start of the study up to 36 months
Participants' Adherence Rate to Treatment with Lanadelumab
Adherence is defined as the proportion of time on treatment over the study observation time period, times the proportion of actual lanadelumab doses taken per label as reported by participants through an administration diary over the total expected lanadelumab doses per label during the treatment period.
Measured: Up to 36 months
Frequency of Use of Approved Lanadelumab Dosing Regimens
Frequency of use of approved lanadelumab dosing regimens will be assessed.
Measured: Up to 36 months
Frequency of Administration Modalities of Lanadelumab
Frequency of administration modalities of lanadelumab (self-administration versus (vs). administration by a caregiver, HCP, or other) will be assessed.
Measured: Up to 36 months
Number of Administrations of Lanadelumab Before Participant Discontinuation
Number of administrations of lanadelumab before participant discontinuation from the study will be assessed.
Measured: Up to 36 months
Frequency of Reasons for Discontinuation of Treatment With Lanadelumab Reported by Participants
Frequency of reasons for discontinuation of treatment with lanadelumab reported by participants will be assessed.
Measured: Up to 36 months
Adverse Event (AE) Incidence, Type, Seriousness and Relatedness to Lanadelumab Treatment
An AE can be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product whether or not related to the medicinal product. AE incidence, type, seriousness and relatedness to lanadelumab treatment will be assessed.
Measured: From start of the study up to 36 months
Severity of Non-Serious Adverse Events (AEs)
Severity of AEs is determined by using the following definitions: Mild: A type of AE that is usually transient and may require only minimal treatment or therapeutic intervention, the event does not generally interfere with activities of daily living. Moderate: A type of AE that is usually alleviated with specific therapeutic intervention, the event interferes with usual activities of daily living, causing discomfort, but poses no significant or permanent risk of harm to the research participant. Severe: A type of AE that interrupts usual activities of daily living (ADL), or significantly affects clinical status, or may require intensive therapeutic intervention. Severity of non-serious AEs will be assessed.
Measured: From start of the study up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2019
- Study start11 Dec 2019
- Primary completion9 Sep 2024
- Study completion9 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hereditary Angioedema (HAE)Studies 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
- 140 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04130191
- Sponsor's protocol ID
- SHP643-402
- Other IDs
- TAK-743-402
- Acronym
- ENABLE
- Status last verified
- Aug 2025
- First submitted
- 3 Oct 2019
- Last updated
- 2 Sep 2025
- 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
Zanichelli A, Wuillemin WA, Aygoren-Pursun E, Banerji A, Busse PJ, Betschel SD, Cancian M, Gagnon R, Goodyear MD, Kinaciyan T, Kessel A, Magerl M, Recke A, Wedner HJ, Estepan DN, Watt M, Andresen I, Juethner S, Khutoryansky N, Martinez-Saguer I. Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence. Ann Allergy Asthma Immunol. 2025 Nov;135(5):560-569.e2. doi: 10.1016/j.anai.2025.07.025. Epub 2025 Sep 6.
PubMed 40769455Tachdjian R, Banerji A, Busse PJ, Agmon-Levin N, Anderson J, Cancian M, Spadaro G, Enciu C, Estepan DN, Khutoryansky N, Jain S, Recke A. Effective long-term prophylaxis with lanadelumab in adolescents with hereditary angioedema: EMPOWER/ENABLE. Pediatr Allergy Immunol. 2025 Apr;36(4):e70072. doi: 10.1111/pai.70072.
PubMed 40171989
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Three-year, Non-interventional, Prospective, Multicenter Study to Evaluate the Long- Term Effectiveness of Lanadelumab in Real-world Clinical Practice (ENABLE)
- ClinicalTrials.gov ID
- NCT04130191
- Acronym / sponsor ID
- ENABLE / SHP643-402
- Conditions
- Hereditary Angioedema (HAE)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 140 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Study start
- 11 Dec 2019 (actual)
- Primary completion
- 9 Sep 2024 (actual)
- Study completion
- 9 Sep 2024 (actual)
- First posted
- 17 Oct 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.
The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started.
Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started the study before 1 March 2021) will be collected for 36 months. Participants will report information in a smartphone application at study start and for the next 3 months and then every 6 months until the study ends; data will also be collected by the study doctor during routine clinic visits
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with hereditary angioedema (HAE) | – | –Participants who initiate treatment with lanadelumab according to current product labelling will be enrolled and followed for up to 24 or 36 months (depending on their enrollment date). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling.
Key inclusion criteria (4 listed)
- Voluntarily provision of written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
- Initiation of treatment with lanadelumab in accordance with current product labelling. Decision to start treatment with lanadelumab must be made before and be independent from enrollment in the study.
- Availability of information for HAE attack-related variables assessed at study enrollment for the previous three months.
- Ability to use a smartphone for data collection in this study.
Key exclusion criteria (6 listed)
- Inability to provide written, signed, and dated informed consent.
- Participation in the study not considered appropriate by the treating physician/investigator.
- Contraindication to treatment with lanadelumab according to current product labelling.
- Pregnant or breastfeeding.
- Concomitant exposure to any investigational therapy (including medications not used for HAE).
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
Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks
Time frame: Up to 36 months
HAE attack is defined as a discrete episode during which the participant progress from no angioedema to symptoms of angioedema. Incidence rate ratio of on-treatment patient-reported HAE attacks up to three years after initiation of lanadelumab compared to the history of HAE attacks of last three months prior to lanadelumab use will be reported.
21 secondary outcomes
Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70
Time frame: From Day 70 up to 36 months
Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks
Time frame: Up to 36 months
Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location
Time frame: Up to 36 months
Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used
Time frame: Up to 36 months
Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used
Time frame: Up to 36 months
Proportion of Hereditary Angioedema (HAE) Attacks Requiring Visit to an Healthcare Provider (HCP), Access to an Emergency Room (ER), or Hospitalization
Time frame: Up to 36 months
Angioedema Quality of Life (AE-QoL)
Time frame: Up to 36 months
Fatigue Severity Scale (FSS)
Time frame: Up to 36 months
Hospital Anxiety and Depression Scale (HADS)
Time frame: Up to 36 months
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)
Time frame: Up to 36 months
Work Productivity and Activity Impairment: General Health (WPAI:GH)
Time frame: Up to 36 months
Adult Carer Quality of Life (AC-QoL) Questionnaire
Time frame: Up to 36 months
Dose of Lanadelumab
Time frame: From start of the study up to 36 months
Frequency of Administration of Lanadelumab
Time frame: From start of the study up to 36 months
Participants' Adherence Rate to Treatment with Lanadelumab
Time frame: Up to 36 months
Frequency of Use of Approved Lanadelumab Dosing Regimens
Time frame: Up to 36 months
Frequency of Administration Modalities of Lanadelumab
Time frame: Up to 36 months
Number of Administrations of Lanadelumab Before Participant Discontinuation
Time frame: Up to 36 months
Frequency of Reasons for Discontinuation of Treatment With Lanadelumab Reported by Participants
Time frame: Up to 36 months
Adverse Event (AE) Incidence, Type, Seriousness and Relatedness to Lanadelumab Treatment
Time frame: From start of the study up to 36 months
Severity of Non-Serious Adverse Events (AEs)
Time frame: From start of the study up to 36 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 2 Sep 2025.
- Derived from this study: PubMed 40769455Zanichelli A, Wuillemin WA, Aygoren-Pursun E, Banerji A, Busse PJ, Betschel SD, Cancian M, Gagnon R, Goodyear MD, Kinaciyan T, Kessel A, Magerl M, Recke A, Wedner HJ, Estepan DN, Watt M, Andresen I, Juethner S, Khutoryansky N, Martinez-Saguer I. Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence. Ann Allergy Asthma Immunol. 2025 Nov;135(5):560-569.e2. doi: 10.1016/j.anai.2025.07.025. Epub 2025 Sep 6.
- Derived from this study: PubMed 40171989Tachdjian R, Banerji A, Busse PJ, Agmon-Levin N, Anderson J, Cancian M, Spadaro G, Enciu C, Estepan DN, Khutoryansky N, Jain S, Recke A. Effective long-term prophylaxis with lanadelumab in adolescents with hereditary angioedema: EMPOWER/ENABLE. Pediatr Allergy Immunol. 2025 Apr;36(4):e70072. doi: 10.1111/pai.70072.
- To obtain more information on the study, click here/on this link.Link 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
19 sites in 7 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 Shire at 19 sites in 7 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 6 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 2 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Germany, Italy, Israel, Spain, Switzerland and 2 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?
Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks (time frame: Up to 36 months). 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 NCT04130191, which also lists 2 linked publications.
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 2 Sep 2025; AzTrials last refreshed its copy on 7 Nov 2026.