Angioedemas, Hereditary
Conditions
Keywords
Lanadelumab, Post-authorization study
Brief summary
The main aim of this study is to learn about the safety profile of lanadelumab in teenagers and adults with hereditary angioedema (HAE) in Argentina as part of routine routine practice. This study is about collecting data only. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. The study will collect data from the participant's medical records. Participants do not need to visit their doctor in addition to their normal visits.
Interventions
None listed
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants (\>=12 years) with investigator confirmed diagnosis of HAE. * Have received at least one dose of lanadelumab according to approved indications. * Signed the mandatory consent that has been agreed with national regulatory authorities (ANMAT) as applicable.
Exclusion criteria
\- Hypersensitivity to the active substance or to any of the excipients.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Adverse Events of Special Interest (AESIs) | Baseline up to end of study (up to 24 months) | An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Percentage of participants with AESIs such as hypersensitivity, immunogenicity, liver toxicity, and embryo-fetal toxicity will be reported. |
| Percentage of Participants With Serious and Non-serious Adverse Events (AEs) | Baseline up to end of study (up to 24 months) | An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Serious AE is any untoward medical occurrence (whether considered to be related to study assigned treatment or not) that at any dose resulted in death, is life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or was an important medical event. AEs including both serious and non-serious adverse events will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Monthly Mean Rate of Investigator Confirmed HAE Attacks | At Weeks 12 and 24 | HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in greater than or equal to (\>=) 1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Monthly mean rate of investigator confirmed HAE attacks with assessments at Weeks 12 and 24 will be reported. |
| Monthly Mean Rate of Investigator Confirmed Moderate or Severe HAE Attacks | At Weeks 12 and 24 | The overall severity of attack will be determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Monthly mean rate of investigator confirmed moderate or severe HAE attacks with assessments at Weeks 12 and 24 will be reported. |
| Monthly Mean Rate of Investigator Confirmed HAE Attacks Requiring Acute Treatment | At Weeks 12 and 24 | HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in \>=1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Monthly mean rate of investigator confirmed HAE attacks requiring acute treatment with assessments at Weeks 12 and 24 will be reported. |
| Percentage of Participants who are Attack Free | At Weeks 12 and 24 | A participant will be considered as attack free during an efficacy evaluation period if the participant has no investigator-confirmed HAE attacks during treatment period. HAE attack is defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema based on investigator judgement. In general, the event must have symptoms or signs consistent with an attack in \>=1 of the typical locations (peripheral angioedema; abdominal angioedema; laryngeal/tongue angioedema). Percentage of participants who are attack free with assessments at 12 and 24 weeks will be reported. |
Countries
Argentina
Outcome results
None listed