Clinical trial NCT04130191: A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II
Official title: A Three-year, Non-interventional, Prospective, Multicenter Study to Evaluate the Long- Term Effectiveness of Lanadelumab in Real-world Clinical Practice (ENABLE)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2019-10-17
Enrollment: 140
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling.

Brief summary: The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started.

Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started the study before 1 March 2021) will be collected for 36 months. Participants will report information in a smartphone application at study start and for the next 3 months and then every 6 months until the study ends; data will also be collected by the study doctor during routine clinic visits


Eligibility criteria: Inclusion Criteria:

* Voluntarily provision of written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
* Initiation of treatment with lanadelumab in accordance with current product labelling. Decision to start treatment with lanadelumab must be made before and be independent from enrollment in the study.
* Availability of information for HAE attack-related variables assessed at study enrollment for the previous three months.
* Ability to use a smartphone for data collection in this study.

Exclusion Criteria:

* Inability to provide written, signed, and dated informed consent.
* Participation in the study not considered appropriate by the treating physician/investigator.
* Contraindication to treatment with lanadelumab according to current product labelling.
* Pregnant or breastfeeding.
* Concomitant exposure to any investigational therapy (including medications not used for HAE).
* Current or planned participation in other interventional studies.

Primary outcomes:
measure: Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks
timeFrame: Up to 36 months
Secondary outcomes:
measure: Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70
timeFrame: From Day 70 up to 36 months
measure: Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks
timeFrame: Up to 36 months
measure: Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location
timeFrame: Up to 36 months
measure: Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used
timeFrame: Up to 36 months
measure: Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used
timeFrame: Up to 36 months
measure: Proportion of Hereditary Angioedema (HAE) Attacks Requiring Visit to an Healthcare Provider (HCP), Access to an Emergency Room (ER), or Hospitalization
timeFrame: Up to 36 months
measure: Angioedema Quality of Life (AE-QoL)
timeFrame: Up to 36 months
measure: Fatigue Severity Scale (FSS)
timeFrame: Up to 36 months
measure: Hospital Anxiety and Depression Scale (HADS)
timeFrame: Up to 36 months
measure: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)
timeFrame: Up to 36 months
measure: Work Productivity and Activity Impairment: General Health (WPAI:GH)
timeFrame: Up to 36 months
measure: Adult Carer Quality of Life (AC-QoL) Questionnaire
timeFrame: Up to 36 months
measure: Dose of Lanadelumab
timeFrame: From start of the study up to 36 months
measure: Frequency of Administration of Lanadelumab
timeFrame: From start of the study up to 36 months
measure: Participants' Adherence Rate to Treatment with Lanadelumab
timeFrame: Up to 36 months
measure: Frequency of Use of Approved Lanadelumab Dosing Regimens
timeFrame: Up to 36 months
measure: Frequency of Administration Modalities of Lanadelumab
timeFrame: Up to 36 months
measure: Number of Administrations of Lanadelumab Before Participant Discontinuation
timeFrame: Up to 36 months
measure: Frequency of Reasons for Discontinuation of Treatment With Lanadelumab Reported by Participants
timeFrame: Up to 36 months
measure: Adverse Event (AE) Incidence, Type, Seriousness and Relatedness to Lanadelumab Treatment
timeFrame: From start of the study up to 36 months
measure: Severity of Non-Serious Adverse Events (AEs)
timeFrame: From start of the study up to 36 months
Other outcomes:
Locations:
city: Wien
country: Austria
facility: Medical University of Vienna
city: Frankfurt
country: Germany
facility: Klinikum der Johann-Wolfgang Goethe-Universitat
state: Hessen
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Düsseldorf
country: Germany
facility: Universitatsklinikum Dusseldorf
city: Lübeck
country: Germany
facility: Universitatsklinikum Schleswig-Holstein
city: Mörfelden-Walldorf
country: Germany
facility: Hämophilie Zentrum Rhein Main GmbH
city: München
country: Germany
facility: Klinikum rechts der Isar der Technischen Universität München
city: Münster
country: Germany
facility: Universitätsklinikum Münster
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm
city: Haifa
country: Israel
facility: Bnai Zion Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center - PPDS
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
state: Campania
city: Milano
country: Italy
facility: ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco
city: Padova
country: Italy
facility: Azienda Ospedale Università Padova
city: Kuwait City
country: Kuwait
facility: Al-Rashed Allergy Center
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria
city: L'Hospitalet de Llobregat
country: Spain
facility: Hospital Universitario de Bellvitge
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron - PPDS
city: Basel
country: Switzerland
facility: Universitätsspital Basel
city: Luzern 16
country: Switzerland
facility: Luzerner Kantonsspital LUKS
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: