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

NCT04861090 | Completed

• 2 other identifiers: TAK-743-4006MACS-2020-081002
• Study Type: observational
• Target: 207
• Locations: 4 countries
• Sites: 20

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.

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (4)

• Percentage of Participants With Specific HAE Attack Occurrence

  Up to Month 12

• Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment

  Up to Month 12

• Number of Participants Characterized With Every Four Weeks Adjustment

  Every 4 weeks from start of treatment (Up to Month 12)

• Number of Participants Characterized Based on Primary Reasons for Down Titration

  Up to Month 12

Study Arms (1)

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.

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (20)

LKH-Universitätsklinikum Klinikum Graz

Graz, 8036, Austria

Location

Kepler Universitätsklinikum Linz

Linz, 4021, Austria

Location

Medizinische Universitat Wien (Medical University of Vienna)

Vienna, 1090, Austria

Location

CHU de Grenoble

Grenoble, 38043, France

Location

CHRU Lille

Lille, 59800, France

Location

Groupement Hospitalier Edouard Herriot

Lyon, 69437, France

Location

CHU Montpellier - Hôpital St Eloi

Montpellier, 34295, France

Location

Hôtel Dieu de Paris Hospital

Paris, 75181, France

Location

Hôpital Saint Antoine

Paris, 75571, France

Location

Centre Hospitalier Universitaire Hopitaux de Rouen

Rouen, 76031, France

Location

Charitè Campus Mitte

Berlin, 10117, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt am Main, 60596, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Hämophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, 64546, Germany

Location

Klinikum rechts der Isa der Technischen Universitaet Muenchen

Munich, 80802, Germany

Location

Universitatsklinikum Munster

Münster, 48149, Germany

Location

Universitätsklinikum Ulm

Ulm, 89075, Germany

Location

Navy Hospital of Athens

Athens, 115 21, Greece

Location

Laiko General Hospital of Athens

Athens, 115 27, Greece

Location

University General Hospital of Larissa

Larissa, 15125, Greece

Location

Related Publications (1)

• Magerl M, Bouillet L, Martinez-Saguer I, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: Multicountry INTEGRATED Observational Study. J Allergy Clin Immunol Pract. 2025 Feb;13(2):378-387.e2. doi: 10.1016/j.jaip.2024.12.008. Epub 2024 Dec 17.

  PMID: 39701274 DERIVED

Related Links

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

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 19, 2021
• First Posted: April 27, 2021
• Study Start: September 15, 2021
• Primary Completion: June 30, 2022
• Study Completion: June 30, 2022
• Last Updated: January 13, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

DE (7); GR (3); FR (7); AU (3)