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HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment.
The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks.
Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition.
The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants with HAE-C1INH | Paediatric participants with HAE-C1INH who initiated long-term prophylaxis (LTP) treatment with lanadelumab within a routine clinical setting will be included. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No intervention | Other | This is a non-interventional study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants who are Free of HAE Attacks | 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). | Up to 13 months |
| Measure | Description | Time Frame |
|---|---|---|
| HAE Attack Rate of Participants | Up to 13 months | |
| HAE Attack-free Rate of Participants Relative to Prior Treatment | Up to 13 months | |
| Association Between Covariates of Interest and Attack Free Rate |
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Inclusion Criteria:
Exclusion Criteria:
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Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Takeda Contact | Contact | +1-877-825-3327 | medinfoUS@takeda.com |
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital de Ninos Sor Maria Ludovica | Not yet recruiting | La Plata | Buenos Aires | B1904CSI | Argentina |
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| Label | URL |
|---|---|
| Click here for more information about this trial in easy-to-understand language. | View source |
| Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language. | 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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Association between covariates of interest (e.g., age, sex, family history) and attack-free rate will be determined. Associations between attack free rate and covariates of interest will be modelled, using generalized linear models. |
| Up to 13 months |
| Number of Participants With HAE Attack Based on Attack Severity | Number of participants with HAE attack based on attack severity (mild, moderate, severe, unknown) will be reported. | Up to 13 months |
| Number of Participants With HAE Attack Based on Location | Number of participants with HAE attack based on location (e.g. extremities, abdominal, facial, laryngeal or genital) will be reported. | Up to 13 months |
| Number of Participants with Treatment History Before use of Lanadelumab | Number of participants with treatment history (type of treatment, duration of treatment, reason for start/ discontinuation/ switch) before use of lanadelumab will be reported. | Up to 13 months |
| Average Duration of Lanadelumab Treatment | Average duration of lanadelumab treatment will be reported. | Up to 13 months |
| Number of Participants Characterized Based on Lanadelumab Treatment Patterns | Number of participants based on lanadelumab treatment patterns (e.g. setting of administration, interval of administration changes/dose modifications, reasons for initiation/discontinuation and modifications) will be reported. | Up to 13 months |
| Number of Participants With Treatments Received Following Lanadelumab Discontinuation | Number of participants with treatments (e.g. type of treatment, reason for selection) received following lanadelumab discontinuation will be evaluated. | Up to 13 months |
| Number of Participants With Healthcare Resource Utilization (HRU) Outcomes | HRU outcomes will include number of healthcare professional (HCP) visits, number of emergency department (ED) visits, number of inpatient hospitalizations, number of participants with admission to and days spent in intensive care unit related to HAE-C1INH, and number of school days missed will be reported. | Up to 13 months |
| Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations | Length of hospital stay in days related to HAE-C1INH hospitalizations will be reported. | Up to 13 months |
| Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Up to 13 months |
| Percentage of Participants With Injection Site Reactions | Percentage of participants with injection site reactions will be reported. | Up to 13 months |
| Centro de Alergia e Inmunologia Clinica Mar del Plata | Not yet recruiting | Mar del Plata | Buenos Aires | 7600 | Argentina |
|
| Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan | Not yet recruiting | Ciudad de Buenos Aires | Buenos Aires F.D. | C1249ABN | Argentina |
|
| Hospital Italiano de Buenos Aires | Recruiting | Buenos Aires | Ciudad Autonoma de BuenosAires | C1199ABB | Argentina |
|
| Sanatorio de la Canada-Cordoba | Recruiting | Córdoba | X5000BSQ | Argentina |
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| AP-HM- Hopital de La Timone | Recruiting | Marseille | Bouches-du-Rhone | 13005 | France |
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| CHU de Grenoble Alpes - Hopital Couple-Enfant | Recruiting | La Tronche | Grenoble | 38700 | France |
|
| AP-HP - Hopital Armand Trousseau | Recruiting | Paris | 75012 | France |
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| Universitatsklinikum Ulm | Not yet recruiting | Ulm | Baden-Wurttemberg | 89081 | Germany |
|
| Universitatsklinikum Frankfurt am Main | Not yet recruiting | Frankfurt am Main | Hesse | 60590 | Germany |
|
| Hamophilie Zentrum Rhein Main GmbH | Recruiting | Frankfurt am Main | Hesse | 60596 | Germany |
|
| Universitatsklinikum Munster | Recruiting | Münster | North Rhine-Westphalia | 48149 | Germany |
|
| Universitatsklinikum Leipzig | Recruiting | Leipzig | Saxony | 4103 | Germany |
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| Klinikum St. Georg Leipzig | Recruiting | Leipzig | Saxony | 4129 | Germany |
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| Universitaetsklinikum Schleswig-Holstein - Campus Luebeck | Recruiting | Lübeck | Schleswig-Holstein | 23538 | Germany |
|
| Charite Campus Virchow-Klinikum-Augustenburger Platz 1 | Recruiting | Berlin | 13353 | Germany |
|
| Hadassah Medical Center- Ein Kerem - PPDS | Recruiting | Jerusalem | 9112001 | Israel |
|
| Schneider Childrens Medical Center of Israel Petah Tikvah PIN | Recruiting | Petah Tikva | 4910000 | Israel |
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| Tel Aviv Sourasky Medical Center | Recruiting | Tel Aviv | 6423906 | Israel |
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| Childrens University Hospital | Not yet recruiting | Belgrade | 11000 | Serbia |
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| Mother and Child Health Care Institute of Serbia Dr Vukan Cupic | Recruiting | Belgrade | 11000 | Serbia |
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| Children and Youth Health Care Institute of Vojvodina | Recruiting | Novi Sad | 21000 | Serbia |
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| Royal Victoria Infirmary | Recruiting | Newcastle upon Tyne | Tyne and Wear | NE1 4LP | United Kingdom |
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| The Royal London Hospital - PPDS | Recruiting | London | E1 1FR | United Kingdom |
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| 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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