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

Hereditary Angioedema (HAE) Clinical Trial

— INTEGRATED  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04861090
• Other study ID #: TAK-743-4006
• Secondary ID: MACS-2020-081002
• Status: Completed
• Start date: September 15, 2021
• Est. completion date: June 30, 2022

Study information

• Verified date: January 2023
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 207
• Est. completion date: June 30, 2022
• Est. primary completion date: June 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Locations

Country	Name	City	State
Austria	LKH-Universitätsklinikum Klinikum Graz	Graz
Austria	Kepler Universitätsklinikum Linz	Linz
Austria	Medizinische Universitat Wien (Medical University of Vienna)	Wien
France	CHU de Grenoble	Grenoble
France	CHRU Lille	Lille
France	Groupement Hospitalier Edouard Herriot	Lyon
France	CHU Montpellier - Hôpital St Eloi	Montpellier
France	Hôpital Saint Antoine	Paris
France	Hôtel Dieu de Paris Hospital	Paris
France	Centre Hospitalier Universitaire Hopitaux de Rouen	Rouen
Germany	Charitè Campus Mitte	Berlin
Germany	Universitätsklinikum Frankfurt	Frankfurt am Main
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Hämophilie Zentrum Rhein Main GmbH	Mörfelden-Walldorf
Germany	Klinikum rechts der Isa der Technischen Universitaet Muenchen	Munich
Germany	Universitatsklinikum Munster	Münster
Germany	Universitätsklinikum Ulm	Ulm
Greece	Laiko General Hospital of Athens	Athens
Greece	Navy Hospital of Athens	Athens
Greece	University General Hospital of Larissa	Larissa

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

Austria,  France,  Germany,  Greece, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab	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 Month 12
Primary	Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two Weeks	Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every two weeks will be reported.	Up to Month 12
Primary	Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks	Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every four weeks will be reported.	Up to Month 12
Secondary	Percentage of Participants With Specific HAE Attack Occurrence	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.	Up to Month 12
Secondary	Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment	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.	Up to Month 12
Secondary	Number of Participants Characterized With Every Four Weeks Adjustment	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.	Every 4 weeks from start of treatment (Up to Month 12)
Secondary	Number of Participants Characterized Based on Primary Reasons for Down Titration	Number of participants will be characterized based on primary reasons for down titration will be reported.	Up to Month 12

External Links

•   To obtain more information on the study, click here/on this link