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

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.

Study Overview

• Status: Recruiting
• Conditions: Hereditary Angioedema (HAE)

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

• Study Contact: Name: Takeda Contact; Phone Number: +1-877-825-3327; Email: medinfoUS@takeda.com

Study Locations

• Poland
  • Dolnośląskie
    • Wrocław, Dolnośląskie, Poland, 50-566
      • Recruiting
      • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu
      • Contact: Site Contact
      • Principal Investigator: Robert Pawłowicz, MD
  • Kujawsko-Pomorskie
    • Bydgoszcz, Kujawsko-Pomorskie, Poland, 85-168
      • Recruiting
      • Szpital Uniwersytecki nr 2
      • Contact: Site Contact
      • Principal Investigator: Zbigniew Bartuzi, Prof
  • Lubelskie
    • Lublin, Lubelskie, Poland, 20-718
      • Recruiting
      • Wojewódzki Szpital Specjalistyczny im. Stefana Kardynała Wyszyńskiego
      • Contact: Site Contact
      • Principal Investigator: Ewa Trębas-Pietraś, MD
  • Mazowieckie
    • Warszawa, Mazowieckie, Poland, 04-141
      • Recruiting
      • Wojskowy Instytut Medyczny
      • Contact: Site Contact
      • Principal Investigator: Aleksandra Kucharczyk, MD, PhD
  • Małopolskie
    • Kraków, Małopolskie, Poland, 31-501
      • Recruiting
      • Szpital Uniwersytecki w Krakowie
      • Contact: Site Contact
      • Principal Investigator: Grzegorz Porębski, Prof
  • Podkarpackie
    • Rzeszów, Podkarpackie, Poland, 35-001
      • Recruiting
      • Kliniczny Szpital Wojewodzki Nr 1 im. Fryderyka Chopina w Rzeszowie
      • Contact: Site Contact
      • Principal Investigator: Małgorzata Pawlukiewicz, MD
  • Podlaskie
    • Białystok, Podlaskie, Poland, 15-276
      • Recruiting
      • Uniwersytecki Szpital Kliniczny w Białymstoku
      • Contact: Site Contact
      • Principal Investigator: Marcin Moniuszko, Prof
    • Białystok, Podlaskie, Poland, 15-540
      • Recruiting
      • Uniwersytet Medyczny w Białymstoku
      • Contact: Site Contact
      • Principal Investigator: Wojciech Naumnik, MD
  • Pomorskie
    • Gdańsk, Pomorskie, Poland, 80-211
      • Recruiting
      • Uniwersyteckie Centrum Kliniczne
      • Contact: Site Contact
      • Principal Investigator: Krzysztof Kuziemski, Prof
  • Warmińsko-Mazurskie
    • Olsztyn, Warmińsko-Mazurskie, Poland, 10-357
      • Recruiting
      • Samodzielny Publiczny Zespół Gruźlicy i Chorób Płuc w Olsztynie
      • Contact: Site Contact
      • Principal Investigator: Magdalena Zakrzewska, MD
  • Wielkopolskie
    • Poznań, Wielkopolskie, Poland, 60-569
      • Recruiting
      • Wielkopolskie Centrum Pulmonologii i Torakochirurgii im. Eugenii i Janusza Zeylandów/Szpital w Poznaniu
      • Contact: Site Contact
      • Principal Investigator: Aldona Juchacz, MD
  • Zachodniopomorske
    • Szczecin, Zachodniopomorske, Poland, 70-111
      • Recruiting
      • SPSK nr 2 PUM w Szczecinie
      • Contact: Site Contact
      • Principal Investigator: Małgorzata Sokołowska, MD, PhD
  • Łódzkie
    • Łódź, Łódzkie, Poland, 92-213
      • Recruiting
      • SAMODZIELNY PUBLICZNY ZAKŁAD OPIEKI ZDROWOTNEJ - Centralny Szpital Kliniczny Uniwersytety Medycznego w Łodzi
      • Contact: Site Contact
      • Principal Investigator: Joanna Jamróz-Brzeska, MD
  • Świętokrzyskie
    • Chmielknik, Świętokrzyskie, Poland, 26-020
      • Not yet recruiting
      • Szpital Powiatowy w Chmielniku
      • Contact: Site Contact
      • Principal Investigator: Oskar Solarski, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Participants with HAE are included in this study.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Categorized Based on Lanadelumab Treatments Patterns	Number of participants categorized based on lanadelumab treatments patterns will be analyzed and reported.	Up to 24 months
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.	Up to 24 months
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.	Up to 24 months
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.	Up to 24 months
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.	Up to 24 months

Collaborators and Investigators

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

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): March 11, 2022
• Primary Completion (Estimated): March 15, 2026
• Study Completion (Estimated): March 15, 2026

Study Registration Dates

• First Submitted: November 24, 2021
• First Submitted That Met QC Criteria: November 24, 2021
• First Posted (Actual): December 7, 2021

Study Record Updates

• Last Update Posted (Actual): August 3, 2023
• Last Update Submitted That Met QC Criteria: August 2, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-743-4003; MACS-2020-081003 (Other Identifier: Takeda)

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; ICF; 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