Clinical trial NCT05147181: A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland
Official title: Lanadelumab Treatment Outcomes in HAE Polish Patients Treated in the Scope of National Drug Program (NDP). Prospective, Multicenter Observational Study
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-12-07
Enrollment: 48
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with HAE are included in this study.

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.


Eligibility criteria: Inclusion Criteria

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.

Exclusion Criteria

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.

Primary outcomes:
measure: Change From Baseline in Total Number of HAE Attacks to 3 Months on Lanadelumab Therapy
timeFrame: Baseline up to 3 months
measure: Percentage of Participants Without HAE Attacks at Month 6
timeFrame: At Month 6
measure: Percentage of Participants Without HAE Attacks at Month 12
timeFrame: At Month 12
measure: Percentage of Participants Without HAE Attacks at Month 18
timeFrame: At Month 18
measure: Percentage of Participants Without HAE Attacks at Month 24
timeFrame: At Month 24
measure: Number of Participants Based on Type of Rescue Treatment Received
timeFrame: Up to 24 months
measure: Percentage of Participants With HAE Attacks Who Received Rescue Treatment
timeFrame: Up to 24 months
measure: Number of Participants Based on Severity of HAE Attacks
timeFrame: Up to 24 months
measure: Number of Participants Based on Anatomical Location of HAE Attacks
timeFrame: Up to 24 months
measure: Time to First HAE Attack Requiring Rescue Treatment
timeFrame: Up to 24 months
measure: Percentage of Participants With HAE Attacks Based on Visit to Healthcare Professional (HCP), Electronic Record (ER) or Hospitalizations
timeFrame: Up to 24 months
measure: Duration of HAE Attack
timeFrame: Up to 24 months
Secondary outcomes:
measure: Number of Participants Categorized Based on Lanadelumab Treatments Patterns
timeFrame: Up to 24 months
measure: Number of Participants Who Received Lanadelumab Treatment Prior to Discontinuation
timeFrame: Up to 24 months
measure: Number of Participants Based on Reason for Lanadelumab Treatment Discontinuation
timeFrame: Up to 24 months
measure: Change From Baseline in Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH) at End of Landelumab Therapy
timeFrame: Up to 24 months
measure: Change From Baseline in Angioedema Quality of Life (AE-QoL) at End of Landelumab Therapy
timeFrame: Up to 24 months
Other outcomes:
Locations:
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu
state: Dolnośląskie
city: Bydgoszcz
country: Poland
facility: Szpital Uniwersytecki nr 2
state: Kujawsko-Pomorskie
city: Lublin
country: Poland
facility: Wojewódzki Szpital Specjalistyczny im. Stefana Kardynała Wyszyńskiego
state: Lubelskie
city: Warszawa
country: Poland
facility: Wojskowy Instytut Medyczny
state: Mazowieckie
city: Kraków
country: Poland
facility: Szpital Uniwersytecki w Krakowie
state: Małopolskie
city: Rzeszów
country: Poland
facility: Kliniczny Szpital Wojewódzki nr 1 im. Fryderyka Chopina w Rzeszowie
state: Podkarpackie
city: Białystok
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Białymstoku
state: Podlaskie
city: Białystok
country: Poland
facility: Uniwersytet Medyczny w Białymstoku
state: Podlaskie
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
state: Pomorskie
city: Olsztyn
country: Poland
facility: Samodzielny Publiczny Zespół Gruźlicy i Chorób Płuc w Olsztynie
state: Warmińsko-Mazurskie
city: Poznań
country: Poland
facility: Wielkopolskie Centrum Pulmonologii i Torakochirurgii im. Eugenii i Janusza Zeylandów/Szpital w Poznaniu
state: Wielkopolskie
city: Szczecin
country: Poland
facility: SPSK nr 2 PUM w Szczecinie
state: Zachodniopomorske
city: Łódź
country: Poland
facility: SAMODZIELNY PUBLICZNY ZAKŁAD OPIEKI ZDROWOTNEJ - Centralny Szpital Kliniczny Uniwersytety Medycznego w Łodzi
state: Łódzkie
city: Chmielknik
country: Poland
facility: Szpital Powiatowy w Chmielniku
state: Świętokrzyskie
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: