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

Hereditary Angioedema (HAE) Clinical Trial

— CHOPIN  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05147181
• Other study ID #: TAK-743-4003
• Secondary ID: MACS-2020-081003
• Status: Recruiting
• Start date: March 11, 2022
• Est. completion date: March 15, 2026

Study information

• Verified date: August 2023
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: March 15, 2026
• Est. primary completion date: March 15, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Locations

Country	Name	City	State
Poland	Uniwersytecki Szpital Kliniczny w Bialymstoku	Bialystok	Podlaskie
Poland	Uniwersytet Medyczny w Bialymstoku	Bialystok	Podlaskie
Poland	Szpital Uniwersytecki nr 2	Bydgoszcz	Kujawsko-Pomorskie
Poland	Szpital Powiatowy w Chmielniku	Chmielknik	Swietokrzyskie
Poland	Uniwersyteckie Centrum Kliniczne	Gdansk	Pomorskie
Poland	Szpital Uniwersytecki w Krakowie	Kraków	Malopolskie
Poland	SAMODZIELNY PUBLICZNY ZAKLAD OPIEKI ZDROWOTNEJ - Centralny Szpital Kliniczny Uniwersytety Medycznego w Lodzi	Lódz	Lódzkie
Poland	Wojewódzki Szpital Specjalistyczny im. Stefana Kardynala Wyszynskiego	Lublin	Lubelskie
Poland	Samodzielny Publiczny Zespól Gruzlicy i Chorób Pluc w Olsztynie	Olsztyn	Warminsko-Mazurskie
Poland	Wielkopolskie Centrum Pulmonologii i Torakochirurgii im. Eugenii i Janusza Zeylandów/Szpital w Poznaniu	Poznan	Wielkopolskie
Poland	Kliniczny Szpital Wojewódzki nr 1 im. Fryderyka Chopina w Rzeszowie	Rzeszów	Podkarpackie
Poland	SPSK nr 2 PUM w Szczecinie	Szczecin	Zachodniopomorske
Poland	Wojskowy Instytut Medyczny	Warszawa	Mazowieckie
Poland	Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu	Wroclaw	Dolnoslaskie

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	Duration of HAE Attack	Duration of HAE Attack will be analyzed and reported.	Up to 24 months
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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

External Links

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