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Hereditary Angioedema (HAE)CHOPINNCT05147181

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

An observational study of Hereditary Angioedema (HAE), run by Takeda at 14 sites in Poland.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Hereditary Angioedema (HAE)
How many people
48 participants
Where
14 sitesPoland
Study dates
11 Mar 2022to 13 Nov 2025
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Participants with HAE are included in this study.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

14 sites in 1 country.

14 sites in 1 country

Poland14 sites

  • Uniwersytecki Szpital Kliniczny w Białymstoku

    Bialystok, Podlaskie Voivodeship, 15-276

  • Uniwersytet Medyczny w Białymstoku

    Bialystok, Podlaskie Voivodeship, 15-540

  • Szpital Uniwersytecki nr 2

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-168

  • Szpital Powiatowy w Chmielniku

    Chmielknik, Świętokrzyskie Voivodeship, 26-020

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, Pomeranian Voivodeship, 80-211

  • Szpital Uniwersytecki w Krakowie

    Krakow, Lesser Poland Voivodeship, 31-501

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

    Lodz, Łódź Voivodeship, 92-213

  • Wojewódzki Szpital Specjalistyczny im. Stefana Kardynała Wyszyńskiego

    Lublin, Lublin Voivodeship, 20-718

  • Samodzielny Publiczny Zespół Gruźlicy i Chorób Płuc w Olsztynie

    Olsztyn, Warmian-Masurian Voivodeship, 10-357

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

    Poznan, Greater Poland Voivodeship, 60-569

  • Kliniczny Szpital Wojewódzki nr 1 im. Fryderyka Chopina w Rzeszowie

    Rzeszów, Podkarpackie Voivodeship, 35-001

  • SPSK nr 2 PUM w Szczecinie

    Szczecin, Zachodniopomorske, 70-111

  • Wojskowy Instytut Medyczny

    Warsaw, Masovian Voivodeship, 04-141

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu

    Wroclaw, Lower Silesian Voivodeship, 50-566

Other trials in Poland

About this study

Official title: Lanadelumab Treatment Outcomes in HAE Polish Patients Treated in the Scope of National Drug Program (NDP). Prospective, Multicenter Observational Study

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.

Medical subject headings
Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT05147181
Study groupRoleWhat they receive
Participants With HAEParticipants 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

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Baseline up to 3 months

  2. Percentage of Participants Without HAE Attacks at Month 6

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

    Measured: At Month 6

  3. Percentage of Participants Without HAE Attacks at Month 12

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

    Measured: At Month 12

  4. Percentage of Participants Without HAE Attacks at Month 18

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

    Measured: At Month 18

  5. Percentage of Participants Without HAE Attacks at Month 24

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

    Measured: At Month 24

  6. 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.

    Measured: Up to 24 months

  7. 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.

    Measured: Up to 24 months

  8. 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.

    Measured: Up to 24 months

  9. 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.

    Measured: Up to 24 months

  10. 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.

    Measured: Up to 24 months

  11. 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.

    Measured: Up to 24 months

  12. Duration of HAE Attack

    Duration of HAE Attack will be analyzed and reported.

    Measured: Up to 24 months

Secondary outcomes (5)

  1. Number of Participants Categorized Based on Lanadelumab Treatments Patterns

    Number of participants categorized based on lanadelumab treatments patterns will be analyzed and reported.

    Measured: Up to 24 months

  2. Number of Participants Who Received Lanadelumab Treatment Prior to Discontinuation

    Number of participants who received lanadelumab treatment prior to discontinuation will be analyzed and reported.

    Measured: Up to 24 months

  3. Number of Participants Based on Reason for Lanadelumab Treatment Discontinuation

    Number of participants based on reason for lanadelumab treatment discontinuation will be analyzed and reported.

    Measured: Up to 24 months

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

    The WPAI:GH is a generic questionnaire to measure the effect of general health and symptom severity on work productivity and regular activities during the past seven days. It can be self or interviewer-administered to adults aged 18 years or older. This six-item PRO instrument covers work (five items) and daily activities (one item) using yes/no or numerical answers (number of hours). It takes approximately two to three minutes to complete. WPAI:GH outcomes are expressed as impairment percentages. An overall work productivity score (health or symptom) [%WP], is calculated by multiplying the percentage of work time spent working (health or symptom) [% WTW] by the percentage productivity at work (health or symptom) [%PW]: %WP = %WTW*%PW. High scores indicate prolonged sick leave or impairment and decreased productivity. Change from baseline to the end of lanadelumab therapy in WPAI:GH will be modelled with a linear model using baseline total score and time of therapy as predictors.

    Measured: Up to 24 months

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

    Angioedema quality of life (AE-QoL) questionnaire was a self-administered validated angioedema disease-specific quality of life instrument. It consisted of 17 specific questions that were associated with work, physical activity, free time, social relations, and diet. Each of the 17 items had a 5-point response scale ranging from 1 (Never) to 5 (Very Often). The questionnaire was scored according to the developers' guidelines to produce a total score and 4 domain scores (functioning, fatigue/mood, fear/shame, nutrition). Raw domain scores (mean of the item scores within each scale) and the raw total score (mean of all item scores) were rescaled using linear transformations into final percentage scores ranging 0 to 100, based on the maximum possible score, where the higher the score the greater the QoL impairment. Change from baseline in AE-QOL at end of landelumab therapy will be analyzed.

    Measured: Up to 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Dec 2021
  2. Study start11 Mar 2022
  3. Primary completion13 Nov 2025
  4. Study completion13 Nov 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
48 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05147181
Sponsor's protocol ID
TAK-743-4003
Other IDs
MACS-2020-081003
Acronym
CHOPIN
Status last verified
Mar 2026
First submitted
24 Nov 2021
Last updated
31 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

An observational study of Hereditary Angioedema (HAE), run by Takeda at 14 sites in Poland.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Total Number of HAE Attacks to 3 Months on Lanadelumab Therapy (time frame: Baseline up to 3 months). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05147181.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2026.

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