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A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis

Completed
Conditions
Hereditary Angioedema (HAE)
Registration Number
NCT04861090
Lead Sponsor
Takeda
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
207
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).
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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.
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Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four WeeksUp to Month 12

Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every four weeks will be reported.

Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With LanadelumabUp to Month 12

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).

Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two WeeksUp to Month 12

Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every two weeks will be reported.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With Specific HAE Attack OccurrenceUp to Month 12

Percentage of participants with specific HAE attack occurrence (e.g. attacks of specific localization, attacks requiring on-demand medication, life-threatening attacks) will be reported.

Percentage of Participants Who are Free of HAE Attacks Relative to Prior TreatmentUp to Month 12

Percentage of participants who are free of HAE attacks relative to prior treatment (e.g. prior prophylaxis treatment or on-demand medications) among participants treated with lanadelumab will be reported.

Number of Participants Characterized With Every Four Weeks AdjustmentEvery 4 weeks from start of treatment (Up to Month 12)

Number of participants will be characterized with every four weeks adjustment (e.g. weight, age, duration of disease, history of life-threating attacks, length of attack-free interval during lanadelumab treatment) will be reported.

Number of Participants Characterized Based on Primary Reasons for Down TitrationUp to Month 12

Number of participants will be characterized based on primary reasons for down titration will be reported.

Trial Locations

Locations (20)

LKH-Universitätsklinikum Klinikum Graz

🇦🇹

Graz, Austria

Kepler Universitätsklinikum Linz

🇦🇹

Linz, Austria

Medizinische Universitat Wien (Medical University of Vienna)

🇦🇹

Wien, Austria

CHU de Grenoble

🇫🇷

Grenoble, France

CHRU Lille

🇫🇷

Lille, France

Groupement Hospitalier Edouard Herriot

🇫🇷

Lyon, France

Hôpital Saint Antoine

🇫🇷

Paris, France

CHU Montpellier - Hôpital St Eloi

🇫🇷

Montpellier, France

Hôtel Dieu de Paris Hospital

🇫🇷

Paris, France

Centre Hospitalier Universitaire Hopitaux de Rouen

🇫🇷

Rouen, France

Charitè Campus Mitte

🇩🇪

Berlin, Germany

Universitätsklinikum Frankfurt

🇩🇪

Frankfurt am Main, Germany

Medizinische Hochschule Hannover

🇩🇪

Hannover, Germany

Hämophilie Zentrum Rhein Main GmbH

🇩🇪

Mörfelden-Walldorf, Germany

Universitatsklinikum Munster

🇩🇪

Münster, Germany

Universitätsklinikum Ulm

🇩🇪

Ulm, Germany

Navy Hospital of Athens

🇬🇷

Athens, Greece

Laiko General Hospital of Athens

🇬🇷

Athens, Greece

University General Hospital of Larissa

🇬🇷

Larissa, Greece

Klinikum rechts der Isa der Technischen Universitaet Muenchen

🇩🇪

Munich, Germany

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