Clinical trial NCT04861090: A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis
Official title: Retrospective, Observational Chart Review Study Evaluating Clinical Effectiveness and Disease/Treatment Management Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-world Setting
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-04-27
Enrollment: 207
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult or adolescent participants (aged greater than or equal to \[\>=\] 12 years) with HAE type I or type II who initiated LTP treatment with lanadelumab within a routine clinical setting.

Brief summary: The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life. This includes the number of people that are attack-free when lanadelumab is given every 2 and every 4 weeks.

This study is about collecting existing data only; participants will not receive lanadelumab as part of this study. No new information will be collected during this study. Only data already available at the participant's doctor's office will be reviewed and collected for this study.

Participants do not need to visit their doctor in addition to their normal visits.


Eligibility criteria: Inclusion Criteria:

* Participant is aged \>= 12 years at the time of the last documented HAE attack in the eligibility period.
* Participant has a physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
* Participant had initiated LTP with lanadelumab during the eligibility period.
* Participant provides informed consent or assent prior to the initiation of any study procedures (where required by local regulations).

Exclusion Criteria:

* Participant was enrolled in a therapeutic investigational drug or device trial during the observation period.
* Participant without documented HAE attacks in the pre-index period and/or without available participant diary or systematic documentation of HAE attacks in the medical records during the post index period.

Primary outcomes:
measure: Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab
timeFrame: Up to Month 12
measure: Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two Weeks
timeFrame: Up to Month 12
measure: Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks
timeFrame: Up to Month 12
Secondary outcomes:
measure: Percentage of Participants With Specific HAE Attack Occurrence
timeFrame: Up to Month 12
measure: Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment
timeFrame: Up to Month 12
measure: Number of Participants Characterized With Every Four Weeks Adjustment
timeFrame: Every 4 weeks from start of treatment (Up to Month 12)
measure: Number of Participants Characterized Based on Primary Reasons for Down Titration
timeFrame: Up to Month 12
Other outcomes:
Locations:
city: Graz
country: Austria
facility: LKH-Universitätsklinikum Klinikum Graz
city: Linz
country: Austria
facility: Kepler Universitätsklinikum Linz
city: Wien
country: Austria
facility: Medizinische Universitat Wien (Medical University of Vienna)
city: Grenoble
country: France
facility: CHU de Grenoble
city: Lille
country: France
facility: CHRU Lille
city: Lyon
country: France
facility: Groupement Hospitalier Edouard Herriot
city: Montpellier
country: France
facility: CHU Montpellier - Hôpital St Eloi
city: Paris
country: France
facility: Hôtel Dieu de Paris Hospital
city: Paris
country: France
facility: Hôpital Saint Antoine
city: Rouen
country: France
facility: Centre Hospitalier Universitaire Hopitaux de Rouen
city: Berlin
country: Germany
facility: Charitè Campus Mitte
city: Frankfurt am Main
country: Germany
facility: Universitätsklinikum Frankfurt
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Munich
country: Germany
facility: Klinikum rechts der Isa der Technischen Universitaet Muenchen
city: Mörfelden-Walldorf
country: Germany
facility: Hämophilie Zentrum Rhein Main GmbH
city: Münster
country: Germany
facility: Universitatsklinikum Munster
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm
city: Athens
country: Greece
facility: Navy Hospital of Athens
city: Athens
country: Greece
facility: Laiko General Hospital of Athens
city: Larissa
country: Greece
facility: University General Hospital of Larissa
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: