A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland
- Conditions
- Hereditary Angioedema (HAE)
- Registration Number
- NCT05147181
- Lead Sponsor
- Takeda
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 48
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Percentage of Participants With HAE Attacks Who Received Rescue Treatment Up to 24 months Percentage of participants with HAE attacks who received rescue treatment will be analyzed and reported.
Number of Participants Based on Severity of HAE Attacks Up to 24 months 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.
Change From Baseline in Total Number of HAE Attacks to 3 Months on Lanadelumab Therapy Baseline up to 3 months 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.
Percentage of Participants Without HAE Attacks at Month 6 At Month 6 Percentage of participants without HAE attacks will be analyzed and reported at Month 6.
Percentage of Participants Without HAE Attacks at Month 18 At Month 18 Percentage of participants without HAE attacks will be analyzed and reported at Month 18.
Percentage of Participants Without HAE Attacks at Month 24 At Month 24 Percentage of participants without HAE attacks will be analyzed and reported at Month 24.
Number of Participants Based on Anatomical Location of HAE Attacks Up to 24 months 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.
Time to First HAE Attack Requiring Rescue Treatment Up to 24 months 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.
Percentage of Participants Without HAE Attacks at Month 12 At Month 12 Percentage of participants without HAE attacks will be analyzed and reported at Month 12.
Number of Participants Based on Type of Rescue Treatment Received Up to 24 months Number of participants based on type of rescue treatment received will be analyzed and reported.
Duration of HAE Attack Up to 24 months Duration of HAE Attack will be analyzed and reported.
Percentage of Participants With HAE Attacks Based on Visit to Healthcare Professional (HCP), Electronic Record (ER) or Hospitalizations Up to 24 months Percentage of participants with HAE Attacks will be analyzed and reported based on Visit to HCP, access to an ER or hospitalizations.
- Secondary Outcome Measures
Name Time Method Number of Participants Categorized Based on Lanadelumab Treatments Patterns Up to 24 months Number of participants categorized based on lanadelumab treatments patterns will be analyzed and reported.
Number of Participants Who Received Lanadelumab Treatment Prior to Discontinuation Up to 24 months Number of participants who received lanadelumab treatment prior to discontinuation will be analyzed and reported.
Number of Participants Based on Reason for Lanadelumab Treatment Discontinuation Up to 24 months Number of participants based on reason for lanadelumab treatment discontinuation will be analyzed and reported.
Change From Baseline in Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH) at End of Landelumab Therapy Up to 24 months 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.
Change From Baseline in Angioedema Quality of Life (AE-QoL) at End of Landelumab Therapy Up to 24 months 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.
Trial Locations
- Locations (14)
Wojew贸dzki Szpital Specjalistyczny im. Stefana Kardyna艂a Wyszy艅skiego
馃嚨馃嚤Lublin, Lubelskie, Poland
Szpital Uniwersytecki w Krakowie
馃嚨馃嚤Krak贸w, Ma艂opolskie, Poland
Kliniczny Szpital Wojew贸dzki nr 1 im. Fryderyka Chopina w Rzeszowie
馃嚨馃嚤Rzesz贸w, Podkarpackie, Poland
Wojskowy Instytut Medyczny
馃嚨馃嚤Warszawa, Mazowieckie, Poland
Uniwersytet Medyczny w Bia艂ymstoku
馃嚨馃嚤Bia艂ystok, Podlaskie, Poland
SAMODZIELNY PUBLICZNY ZAK艁AD OPIEKI ZDROWOTNEJ - Centralny Szpital Kliniczny Uniwersytety Medycznego w 艁odzi
馃嚨馃嚤艁贸d藕, 艁贸dzkie, Poland
Uniwersytecki Szpital Kliniczny w Bia艂ymstoku
馃嚨馃嚤Bia艂ystok, Podlaskie, Poland
Uniwersyteckie Centrum Kliniczne
馃嚨馃嚤Gda艅sk, Pomorskie, Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroc艂awiu
馃嚨馃嚤Wroc艂aw, Dolno艣l膮skie, Poland
Szpital Powiatowy w Chmielniku
馃嚨馃嚤Chmielknik, 艢wi臋tokrzyskie, Poland
Samodzielny Publiczny Zesp贸艂 Gru藕licy i Chor贸b P艂uc w Olsztynie
馃嚨馃嚤Olsztyn, Warmi艅sko-Mazurskie, Poland
SPSK nr 2 PUM w Szczecinie
馃嚨馃嚤Szczecin, Zachodniopomorske, Poland
Wielkopolskie Centrum Pulmonologii i Torakochirurgii im. Eugenii i Janusza Zeyland贸w/Szpital w Poznaniu
馃嚨馃嚤Pozna艅, Wielkopolskie, Poland
Szpital Uniwersytecki nr 2
馃嚨馃嚤Bydgoszcz, Kujawsko-Pomorskie, Poland