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| ID | Type | Description | Link |
|---|---|---|---|
| TAK-743-403 | Other Identifier | Takeda Development Center Americas |
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| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
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The main aim of this study is to compare the HAE attack rate before and after lanadelumab treatment was started in persons with Hereditary Angioedeme Type I or II.
Data from participants will be collected for at least 24 months. Participants will report information in a smartphone application at study start and then every 3 months until the study ends; data will also be collected by the study doctor during routine clinic visits.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Type I or Type II HAE Participants | Participants with Type I or Type II HAE will be followed for 24 or 36 months depending upon enrollment date. Data collection will cease at the end of follow-up period, at the time of withdrawal, lost to follow-up, or death whichever comes first. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Hereditary Angioedema (HAE) Attacks | Number of HAE attacks before and after lanadelumab initiation will be reported. | From enrollment up to 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Dose of Lanadelumab Injections | Dose of lanadelumab injections used during the study will be reported. | From enrollment up to 36 months |
| Frequency of Lanadelumab Injections | Frequency of lanadelumab injections during the study will be reported. |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with a diagnosis of Type I or Type II HAE.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Research Center of Alabama | Birmingham | Alabama | 35209 | United States | ||
| Medical Research of Arizona |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41818404 | Derived | Zaragoza-Urdaz RH, Busse PJ, Fox D, Castaner DN, Khutoryansky N, Juethner S, McBayne T. Lanadelumab's Enduring Effectiveness and Safety in the Management of Hereditary Angioedema for Patients from Puerto Rico: Data from EMPOWER. Int Arch Allergy Immunol. 2026 Mar 12:1-11. doi: 10.1159/000550883. Online ahead of print. | |
| 41566486 |
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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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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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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| From enrollment up to 36 months |
| Proportion of Injections Based on the Type of Administration of Lanadelumab | Proportion of injections based on the type of administration of lanadelumab (self, caregiver, health care provider [HCP], other) will be reported. | From enrollment up to 36 months |
| Time From Diagnosis to Lanadelumab Initiation | Time from diagnosis of HAE attack(s) to lanadelumab treatment initiation will be reported. | From enrollment up to 36 months |
| Proportion of Participants who Discontinue Lanadelumab | Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Proportion of participants who discontinue lanadelumab treatment will be reported. | From enrollment up to 36 months |
| Number of Lanadelumab Injections Before Discontinuation per Reason for Injection | Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Number of injections administered by participant before discontinuation per reason for injection will be reported. | From enrollment up to 36 months |
| Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s) | Number of hospitalizations due to HAE attack(s) will be reported. | From enrollment up to 36 months |
| Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s) | Number of emergency room (ER) visits due to HAE attack(s) will be reported. | From enrollment up to 36 months |
| Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s) | Number of physician visits due to HAE attack(s) will be reported. | From enrollment up to 36 months |
| Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s) | Number, type of rescue medications taken by the participants at the time of HAE attack(s) per the type of administration (self, caregiver, HCP, other) will be reported. | From enrollment up to 36 months |
| Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT) | AECT is a questionnaire and not a validated patient-reported outcome (PRO). HAE control score is evaluated as frequency of answers [very often, often, sometimes, seldom, not at all] to the following questions at record closest to enrollment date): 1. In the last 3 months, how often have you had angioedema? 2. In the last 3 months, how much has your quality of life been affected by angioedema? 3. In the last 3 months, how much has the unpredictability of your angioedema bothered you? 4. In the last 3 months, how well has your angioedema been controlled by your therapy? | From enrollment up to 36 months (every 3 months) |
| Angioedema Quality of Life (AE-QoL) Score | The AE-QoL is developed to measure health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. It is a self-administered PRO designed for adults aged 18 years and older 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. | From enrollment up to 36 months (every 3 months) |
| Work Productivity and Activity Impairment: General Health (WPAI:GH) Score | 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 7 days. It can be self- or interviewer-administered to adults aged 18 years or older. This 6-item PRO covers work (5 items) and daily activities (1 item) using yes/no or numerical answers (number of hours). WPAI:GH outcomes are expressed as impairment percentages. An overall work productivity score (health or symptom) [%0WP], is calculated by multiplying the percentage of work time spent working (health or symptom) [% WTW] by the percentage productivity at work (health or symptom) [%PW]: %0WP = %WTW * %PW. | From enrollment up to 36 months (every 3 months) |
| Treatment Satisfaction (TSQM-9) Score | 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 designed for adults aged 18 years or older with a recall period of 2 to 3 weeks, or since the last medication use. Version TSQM-9 includes 3 domains: effectiveness (3 items), convenience (3 items), and global satisfaction scale (3 items). Scores range from 0 to 100. | From enrollment up to 36 months (every 3 months) |
| Scottsdale |
| Arizona |
| 85248 |
| United States |
| University of California San Diego | San Diego | California | 92122 | United States |
| AIRE Medical of Los Angeles | Santa Monica | California | 90404 | United States |
| Asthma and Allergy Associates, PC | Colorado Springs | Colorado | 80907 | United States |
| Yale University School of Medicine | New Haven | Connecticut | 06519 | United States |
| University of South Florida Asthma, Allergy & Immunology | Tampa | Florida | 33613 | United States |
| University of Kansas Medical Center Research Institute, Inc. | Kansas City | Kansas | 66160 | United States |
| Institute for Asthma & Allergy - Chevy Chase | Chevy Chase | Maryland | 20815 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114-2752 | United States |
| University of Michigan | Ann Arbor | Michigan | 48106 | United States |
| Mid West Immunology Clinic | Plymouth | Minnesota | 55446 | United States |
| Washington University | St Louis | Missouri | 63141 | United States |
| Riverside Medical Group, Belleville | Belleville | New Jersey | 07109 | United States |
| Jay M Kashkin, MD Allergy, Asthma and Immunology | Fair Lawn | New Jersey | 07410 | United States |
| Mount Sinai | New York | New York | 10029 | United States |
| Clinical Research of Charlotte | Charlotte | North Carolina | 28277 | United States |
| Duke Asthma, Allergy & Airway Center | Durham | North Carolina | 27704 | United States |
| Bernstein Clinical Research Center, LLC | Cincinnati | Ohio | 45231 | United States |
| Portland Clinical Research/AAIM Care | Happy Valley | Oregon | 97086 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| AARA Research Center | Dallas | Texas | 75231 | United States |
| Seattle Allergy & Asthma Research Institute | Seattle | Washington | 98115 | United States |
| University of Calgary - Heritage Medical Research Clinic | Calgary | Alberta | T2N 2T9 | Canada |
| McMaster University Health Sciences Center | Hamilton | Ontario | L8S 4K1 | Canada |
| St. Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| CHUM Hôtel-Dieu | Montreal | Quebec | H2X 0A9 | Canada |
| Clinique Specialisée en Allergie de la Capitale | Québec | G1V 4W2 | Canada |
| Rafael H Zaragoza-Urdaz | San Juan | 00918 | Puerto Rico |
| Betschel SD, Chapdelaine H, Gagnon R, Goodyear MD, Keith PK, El-Zoeiby A, Khutoryansky N, Castaner DN. Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study. Allergy Asthma Clin Immunol. 2026 Jan 21;22(1):11. doi: 10.1186/s13223-025-01007-9. |
| 40769455 | Derived | 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. |
| 40504359 | Derived | Bernstein JA, Betschel SD, Busse PJ, Banerji A, Wedner HJ, Manning M, Zaragoza-Urdaz RH, Anderson J, Gagnon R, Baptist AP, Soteres D, Lumry WR, Craig T, Petroni D, Hsu FI, Nova Estepan D, Juethner S, Watt M, Khutoryansky N, Zuraw BL; EMPOWER Investigators. Sustained Effectiveness, Tolerability, and Safety of Long-Term Prophylaxis with Lanadelumab in Hereditary Angioedema: The Prospective, Phase 4, Noninterventional EMPOWER Real-World Study. Adv Ther. 2025 Aug;42(8):3882-3901. doi: 10.1007/s12325-025-03226-3. Epub 2025 Jun 12. |
| 40171989 | Derived | Tachdjian 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. |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| ID | Term |
|---|---|
| D000799 | Angioedema |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007153 | Immunologic Deficiency Syndromes |
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