A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China

The Real-World Effectiveness and Safety of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) for Hereditary Angioedema: An Observational Study in China

The Chinese health authority has approved lanadelumab to prevent Hereditary Angioedema (HAE) attacks in persons of 12 years and older. It has also approved icatibant to treat acute HAE attacks in persons 2 years and older.

One of the main aims of this study is to learn about the number of HAE attacks in 1 month in Chinese persons with HAE during their treatment with lanadelumab. The other main aim is to learn how much time is needed to resolve acute HAE attacks when treated with icatibant. Other aims of this study are to learn more about side effects of lanadelumab and icatibant treatment as well as to collect additional information on the treatment with lanadelumab, such as the dose and how often it needs to be given, reasons for stopping the treatment with lanadelumab and how long it was taken until stopping. Participants will be treated by their doctors according to routine medical practice. Only data already available in the medical records of the participants will be reviewed and collected during this study.

Study Overview

• Status: Completed
• Conditions: Hereditary Angioedema (HAE)
• Intervention / Treatment: Other: No intervention

• Study Type: Observational
• Enrollment (Actual): 115

Contacts and Locations

Study Locations

• China
  • Fujan
    • Fuzhou, Fujan, China, 350005
      • The First Affiliated Hospital Of Fujian Medical University
  • Guangdong
    • Guangzhou, Guangdong, China, 510260
      • The Second Affiliated Hospital of Guangzhou Medical University
    • Guangzhou, Guangdong, China, 510080
      • The First Affiliated Hospital ,Sun Yat-sen University
  • Henan
    • Zhengzhou, Henan, China, 450003
      • Henan Provincial People's Hospital
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital,Tongji Medical College,Huazhong University of Science & Technology
  • Liaoning
    • Shenyang, Liaoning, China, 110001
      • The First Hospital of China Medical University
  • Shandong
    • Jinan, Shandong, China, 250014
      • Qianfo Mountain Hospital, Shandong Province
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200011
      • Renji Hospital Affiliated to Shanghai Jiaotong University School of Medicine
  • Shanxi
    • Taiyuan, Shanxi, China, 030032
      • Shanxi Bethune Hospital
    • Xian, Shanxi, China, 710000
      • The Second Affiliated Hospital of Xian Jiaotong University
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • West China Hospital of Sichuan University
  • Yunnan
    • Kunming, Yunnan, China, 650032
      • First Affiliated Hospital of Kunming Medical University
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310009
      • The Second Affiliated Hospital Zhejiang University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Chinese participants who have received at least one dose of lanadelumab or icatibant from the lanadelumab national medical products administration (NMPA) approval date (02-Dec-2020) to the date of the day before the 1st site initiation date.

Description

Inclusion criteria:

• Chinese participants who have received at least one dose of lanadelumab or icatibant from the lanadelumab NMPA approval date (02-Dec-2020) to the date of the day before the 1st site initiation date.
• Informed consent will be obtained from the participant (and/or a legally authorized representative) prior to including that participant as a study participant and accessing that participant's data, except for cases in which the informed consent can be waived by the site.

Exclusion criteria:

• Participation in other interventional studies involving lanadelumab, icatibant, or any other HAE drug or investigational product on or after the date of the participant's lanadelumab or icatibant initiation.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group L: Lanadelumab; Participants with HAE who have used only lanadelumab during the study-level retrospective observation period will be observed retrospectively from the start date of the participant-level overall baseline period until the end of participant-level retrospective observation period.	Other: No intervention; This is a non-interventional study.
Group I: Icatibant; Participants with HAE who have used only icatibant during the study-level retrospective observation period will be observed retrospectively from the start date of the participant-level overall baseline period until the end of participant-level retrospective observation period.	Other: No intervention; This is a non-interventional study.
Group B: Lanadelumab + Icatibant; Participants with HAE who have used both lanadelumab and icatibant during the study-level retrospective observation period will be observed retrospectively from the start date of the participant-level overall baseline period until the end of participant-level retrospective observation period.	Other: No intervention; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Monthly Rate of HAE Attacks for Each Lanadelumab-treated Participant During the Lanadelumab Exposure Period	The HAE monthly attack rate is defined as the total number of attacks that occurred when the participant was in a lanadelumab exposure period, divided by the total lanadelumab exposure time (in days), multiplied by 28 days.	From the first dose of study drug up to approximately 3 years 8 months
Median Time to Complete Attack Resolution for Icatibant Treated Participants	Time to complete attack resolution among icatibant-treated participants during the retrospective observation period is defined as the time between icatibant first injection and the earliest time of complete resolution of symptoms for each attack during the retrospective observation period that was treated with icatibant.	From the first dose of study drug up to approximately 3 years 8 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) During Lanadelumab and Icatibant Exposure Period	An AE is any untoward medical occurrence in a participant administered a studied drug and which does not necessarily have to have a causal relationship with the studied drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a studied drug, whether or not the event is considered causally related to the use of the product. An SAE is any untoward medical occurrence that at any dose: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event in the opinion of the healthcare provider, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.	From the first dose of study drug up to approximately 3 years 8 months
Dosage of Lanadelumab Among Lanadelumab-treated Participant During the Lanadelumab Exposure Period	Dosage of lanadelumab used during the lanadelumab exposure period will be assessed.	From the first dose of study drug up to approximately 3 years 8 months
Frequency Administration of Lanadelumab Among Lanadelumab-treated Participant During the Lanadelumab Exposure Period	Frequency administration of lanadelumab among lanadelumab-treated participant during the lanadelumab exposure period will be assessed.	From the first dose of study drug up to approximately 3 years 8 months
Number of Participants with Reasons of Discontinuation of Lanadelumab During the Lanadelumab Exposure Period	Number of participants with reasons of discontinuation of lanadelumab during the lanadelumab exposure period will be assessed.	From the first dose of study drug up to approximately 3 years 8 months
Time to Lanadelumab Discontinuation Among Lanadelumab-treated Participant During the Lanadelumab Exposure Period	The time to lanadelumab discontinuation is defined as the time from lanadelumab initiation to the first lanadelumab discontinuation.	From the first dose of study drug up to approximately 3 years 8 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• Click here for more information about this trial in easy-to-understand language.

Study record dates

Study Major Dates

• Study Start (Actual): July 20, 2024
• Primary Completion (Actual): July 31, 2025
• Study Completion (Actual): July 31, 2025

Study Registration Dates

• First Submitted: March 29, 2024
• First Submitted That Met QC Criteria: March 29, 2024
• First Posted (Actual): April 4, 2024

Study Record Updates

• Last Update Posted (Actual): January 21, 2026
• Last Update Submitted That Met QC Criteria: January 16, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immunologic Deficiency Syndromes; Skin Diseases; Urticaria; Skin Diseases, Vascular; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-743-4012

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; SAP; 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