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

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

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Angioedema (HAE)

• Study Type: Observational
• Enrollment (Actual): 207

Contacts and Locations

Study Locations

• Austria
  • Graz, Austria, 8036
    • LKH-Universitätsklinikum Klinikum Graz
  • Linz, Austria, 4021
    • Kepler Universitätsklinikum Linz
  • Wien, Austria, 1090
    • Medizinische Universität Wien (Medical University of Vienna)
• France
  • Grenoble, France, 38043
    • CHU de Grenoble
  • Lille, France, 59800
    • CHRU LILLE
  • Lyon, France, 69437
    • Groupement Hospitalier Edouard Herriot
  • Montpellier, France, 34295
    • CHU Montpellier - Hôpital St Eloi
  • Paris, France, 75571
    • Hôpital Saint Antoine
  • Paris, France, 75181
    • Hôtel Dieu de Paris Hospital
  • Rouen, France, 76031
    • Centre Hospitalier Universitaire Hôpitaux de Rouen
• Germany
  • Berlin, Germany, 10117
    • Charite Campus Mitte
  • Frankfurt am Main, Germany, 60596
    • Universitätsklinikum Frankfurt
  • Hannover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Munich, Germany, 80802
    • Klinikum rechts der Isa der Technischen Universitaet Muenchen
  • Mörfelden-Walldorf, Germany, 64546
    • Hamophilie Zentrum Rhein Main GmbH
  • Münster, Germany, 48149
    • Universitätsklinikum Münster
  • Ulm, Germany, 89075
    • Universitätsklinikum Ulm
• Greece
  • Athens, Greece, 115 27
    • Laiko General Hospital of Athens
  • Athens, Greece, 115 21
    • Navy Hospital of Athens
  • Larissa, Greece, 15125
    • University General Hospital of Larissa

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Participants With Hereditary Angioedema; Participants with HAE type I or type II who had initiated long-term prophylaxis (LTP) treatment with lanadelumab which was administered every two weeks (Q2W) or every four weeks (Q4W) or every six weeks (Q6W) or every eight weeks (Q8W) in accordance to Summary of Product Characteristics (SmPC), during a routine clinical setting will be followed up to 38 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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)
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

Collaborators and Investigators

• Sponsor: Takeda

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): September 15, 2021
• Primary Completion (Actual): June 30, 2022
• Study Completion (Actual): June 30, 2022

Study Registration Dates

• First Submitted: April 19, 2021
• First Submitted That Met QC Criteria: April 26, 2021
• First Posted (Actual): April 27, 2021

Study Record Updates

• Last Update Posted (Estimate): January 13, 2023
• Last Update Submitted That Met QC Criteria: January 12, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-743-4006; MACS-2020-081002 (Other Identifier: Takeda)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: 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.
• IPD Sharing Access Criteria: 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.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No