A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland

NCT05147181 | Completed

• 2 other identifiers: TAK-743-4003MACS-2020-081003
• Study Type: observational
• Target: 48
• Locations: 1 country
• Sites: 14

Brief Summary

The main aim of this study is to learn about how many persons with HAE type I or type II are attack-free when treated with lanadelumab in real life, how many attacks occur and how many of these attacks need rescue treatment and about the nature of HAE attacks. Participants will need to visit their doctor 5 times in total as part of this study. The visits are planned every 6 months. Participants will also be asked to fill out questionnaires as part of this study.

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (12)

• Change From Baseline in Total Number of HAE Attacks to 3 Months on Lanadelumab Therapy

  Change from baseline (3 months period prior to qualification to NDP) to 3 months on lanadelumab therapy in the total number of HAE attacks will be analyzed using either a Poisson or negative binomial mixed effect model with period (pre or post qualification to NDP) as fixed effect and participant as a random effect. Choice of modelling method will depend on the degree of overdispersion in number of HAE attacks.

  Baseline up to 3 months

• Percentage of Participants Without HAE Attacks at Month 6

  Percentage of participants without HAE attacks will be analyzed and reported at Month 6.

  At Month 6

• Percentage of Participants Without HAE Attacks at Month 12

  Percentage of participants without HAE attacks will be analyzed and reported at Month 12.

  At Month 12

• Percentage of Participants Without HAE Attacks at Month 18

  Percentage of participants without HAE attacks will be analyzed and reported at Month 18.

  At Month 18

• Percentage of Participants Without HAE Attacks at Month 24

  Percentage of participants without HAE attacks will be analyzed and reported at Month 24.

  At Month 24

• Number of Participants Based on Type of Rescue Treatment Received

  Number of participants based on type of rescue treatment received will be analyzed and reported.

  Up to 24 months

• Percentage of Participants With HAE Attacks Who Received Rescue Treatment

  Percentage of participants with HAE attacks who received rescue treatment will be analyzed and reported.

  Up to 24 months

• Number of Participants Based on Severity of HAE Attacks

  Number of Participants based on severity of HAE Attacks i.e mild, moderate and severe will be analyzed. Mild means temporary or mild discomfort, moderate means activity limited mildly or moderately and some assistance may be needed and severe means activity considerably limited, assistance needed.

  Up to 24 months

• Number of Participants Based on Anatomical Location of HAE Attacks

  Number of participants based on anatomical location of HAE attacks per body part(s) affected, such as peripheral (e.g., skin), abdominal, upper airway (e.g., larynx), other organs, duration of symptoms/ number of days with angioedema symptoms will be analyzed and reported.

  Up to 24 months

• Time to First HAE Attack Requiring Rescue Treatment

  Kaplan-Meier analysis will be prepared for time to first HAE attack for which rescue treatment was used and time to first HAE attack after lanadelumab treatment discontinuation.

  Up to 24 months

• Percentage of Participants With HAE Attacks Based on Visit to Healthcare Professional (HCP), Electronic Record (ER) or Hospitalizations

  Percentage of participants with HAE Attacks will be analyzed and reported based on Visit to HCP, access to an ER or hospitalizations.

  Up to 24 months

• Duration of HAE Attack

  Duration of HAE Attack will be analyzed and reported.

  Up to 24 months

Secondary Outcomes (5)

• Number of Participants Categorized Based on Lanadelumab Treatments Patterns

  Up to 24 months

• Number of Participants Who Received Lanadelumab Treatment Prior to Discontinuation

  Up to 24 months

• Number of Participants Based on Reason for Lanadelumab Treatment Discontinuation

  Up to 24 months

• Change From Baseline in Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH) at End of Landelumab Therapy

  Up to 24 months

• Change From Baseline in Angioedema Quality of Life (AE-QoL) at End of Landelumab Therapy

  Up to 24 months

Study Arms (1)

Participants With HAE

Participants with type 1 or type 2 HAE when treated with lanadelumab in real life in accordance with Summary of Product Characteristics (SmPC) and NDP requirements will be observed in this prospective observational study for 36 months

Eligibility Criteria

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

Study Population

Participants with HAE are included in this study.

You may qualify if:

• Participant eligibility is determined according to the following criteria prior to entry into the study:
• In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements.
• The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
• HAE participant (aged greater than or equal to \[\>=\] 12 years old.) qualified to treatment with lanadelumab in the NDP and receiving treatment according to the Summary of Product Characteristics for Takhzyro.
• Male or female participants, aged \>=12 years old.

You may not qualify if:

• Any participant who meets any of the following criteria will not qualify for entry into the study:
• Currently participates or plans to participate in any interventional clinical trial.
• Any other reason that, in the Investigator's opinion, makes the participant unsuitable to participate in this study.
• Participants should be included in the study only once. Data erroneously collected from participants for which written consent is not available, will not be included in or will be deleted from the database.

Sponsors & Collaborators

• Takeda: lead

Study Sites (14)

Wielkopolskie Centrum Pulmonologii i Torakochirurgii im. Eugenii i Janusza Zeylandów/Szpital w Poznaniu

Poznan, Greater Poland Voivodeship, 60-569, Poland

Location

Szpital Uniwersytecki nr 2

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-168, Poland

Location

Szpital Uniwersytecki w Krakowie

Krakow, Lesser Poland Voivodeship, 31-501, Poland

Location

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu

Wroclaw, Lower Silesian Voivodeship, 50-566, Poland

Location

Wojewódzki Szpital Specjalistyczny im. Stefana Kardynała Wyszyńskiego

Lublin, Lublin Voivodeship, 20-718, Poland

Location

Wojskowy Instytut Medyczny

Warsaw, Masovian Voivodeship, 04-141, Poland

Location

Kliniczny Szpital Wojewódzki nr 1 im. Fryderyka Chopina w Rzeszowie

Rzeszów, Podkarpackie Voivodeship, 35-001, Poland

Location

Uniwersytecki Szpital Kliniczny w Białymstoku

Bialystok, Podlaskie Voivodeship, 15-276, Poland

Location

Uniwersytet Medyczny w Białymstoku

Bialystok, Podlaskie Voivodeship, 15-540, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, Pomeranian Voivodeship, 80-211, Poland

Location

Samodzielny Publiczny Zespół Gruźlicy i Chorób Płuc w Olsztynie

Olsztyn, Warmian-Masurian Voivodeship, 10-357, Poland

Location

SPSK nr 2 PUM w Szczecinie

Szczecin, Zachodniopomorske, 70-111, Poland

Location

SAMODZIELNY PUBLICZNY ZAKŁAD OPIEKI ZDROWOTNEJ - Centralny Szpital Kliniczny Uniwersytety Medycznego w Łodzi

Lodz, Łódź Voivodeship, 92-213, Poland

Location

Szpital Powiatowy w Chmielniku

Chmielknik, Świętokrzyskie Voivodeship, 26-020, Poland

Location

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: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: December 7, 2021
• Study Start: March 11, 2022
• Primary Completion: November 13, 2025
• Study Completion: November 13, 2025
• Last Updated: March 31, 2026

Record last verified: 2026-03

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

PL (14)