A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II

Hereditary Angioedema (HAE) Clinical Trial

— ENABLE  

Official title:

A Three-year, Non-interventional, Prospective, Multicenter Study to Evaluate the Long- Term Effectiveness of Lanadelumab in Real-world Clinical Practice (ENABLE)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04130191
• Other study ID #: SHP643-402
• Secondary ID: TAK-743-402
• Status: Active, not recruiting
• Start date: December 11, 2019
• Est. completion date: September 30, 2024

Study information

• Verified date: April 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 140
• Est. completion date: September 30, 2024
• Est. primary completion date: September 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Locations

Country	Name	City	State
Austria	Medical University of Vienna	Wien
Germany	Charité - Universitätsmedizin Berlin	Berlin
Germany	Universitatsklinikum Dusseldorf	Düsseldorf
Germany	Klinikum der Johann-Wolfgang Goethe-Universitat	Frankfurt	Hessen
Germany	Universitatsklinikum Schleswig-Holstein	Lübeck
Germany	Hämophilie Zentrum Rhein Main GmbH	Mörfelden-Walldorf
Germany	Klinikum rechts der Isar der Technischen Universität München	München
Germany	Universitätsklinikum Münster	Münster
Germany	Universitätsklinikum Ulm	Ulm
Israel	Bnai Zion Medical Center	Haifa
Israel	Sheba Medical Center - PPDS	Ramat Gan
Italy	ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco	Milano
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli	Campania
Italy	Azienda Ospedale Università Padova	Padova
Kuwait	Al-Rashed Allergy Center	Kuwait City
Portugal	Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria	Lisboa
Spain	Hospital Universitario Vall d'Hebron - PPDS	Barcelona
Spain	Hospital Universitario de Bellvitge	L'Hospitalet de Llobregat	Barcelona
Switzerland	Universitätsspital Basel	Basel
Switzerland	Luzerner Kantonsspital LUKS	Luzern 16

Sponsors (2)

Lead Sponsor	Collaborator
Shire	Takeda Development Center Americas, Inc.

Countries where clinical trial is conducted

Austria,  Germany,  Israel,  Italy,  Kuwait,  Portugal,  Spain,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks	HAE attack is defined as a discrete episode during which the participant progress from no angioedema to symptoms of angioedema. Incidence rate ratio of on-treatment patient-reported HAE attacks up to three years after initiation of lanadelumab compared to the history of HAE attacks of last three months prior to lanadelumab use will be reported.	Up to 36 months
Secondary	Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70	Rate of participant-reported HAE attacks during treatment with lanadelumab from day 70 will be assessed.	From Day 70 up to 36 months
Secondary	Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks	The overall severity of attack is determined using following definitions: mild (Temporary or mild discomfort), moderate (Activity limited mildly or moderately. Some assistance may be needed), severe (Activity considerably limited, assistance needed).	Up to 36 months
Secondary	Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location	Number of on-treatment participant-reported HAE attacks based on anatomical (peripheral, abdominal, laryngeal) location will be assessed.	Up to 36 months
Secondary	Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used	Proportion of HAE attacks for which participants use on-demand therapy will be assessed.	Up to 36 months
Secondary	Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used	Time to first HAE attack for which on-demand therapy is used will be assessed.	Up to 36 months
Secondary	Proportion of Hereditary Angioedema (HAE) Attacks Requiring Visit to an Healthcare Provider (HCP), Access to an Emergency Room (ER), or Hospitalization	Proportion of HAE Attacks requiring visit to HCP, access to an ER, or hospitalization will be assessed.	Up to 36 months
Secondary	Angioedema Quality of Life (AE-QoL)	The AE-QoL is developed to measure participant-reported health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. It is a self-administered participant related outcome (PRO) with a recall period of 4 weeks. There are 17 items across 4 domains: functioning (4 items), fatigue/mood (5 items), fears/shame (6 items), and food (2 items). Responses use a 5-point Likert scale ranging from 'never' to 'very often.' Global scores range from 0 to 100 and scores by domains range from 0 to 100, where 0 indicates highest quality of life and 100 lowest quality of life.	Up to 36 months
Secondary	Fatigue Severity Scale (FSS)	Participant reported fatigue will be measured by the FSS. The FSS is a 9-item questionnaire measuring participants fatigue severity and its impact on motivation, exercise, physical functioning, and work and social life. It uses a 7-point Likert scale response (1 = strongly disagree, 7 = strongly agree) and the final score is obtained as mean of the response scores to the individual questions.	Up to 36 months
Secondary	Hospital Anxiety and Depression Scale (HADS)	The HADS is a self-rating scale developed to detect the levels of depression and anxiety experienced by participants. It is a self-administered PRO composed of 14 items, of which seven relate to anxiety and seven relate to depression. Each item on the questionnaire is scored from 0 to 3, which means that a person can score between 0 and 21 for either anxiety or depression. Recommended cut-off scores are 8 to 10 for doubtful cases and greater than or equal to (> or =) 11 for definite cases.	Up to 36 months
Secondary	Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)	The TSQM is a generic questionnaire to measure participants satisfaction with medication using yes/no and 5- or 7-point Likert scale response options. It is a self-administered PRO instrument designed for adults aged 18 years or older with a recall period of two to three weeks, or since the last medication use. Version TSQM-9 includes three domains: effectiveness (three items), convenience (three items), and global satisfaction scale (three items).	Up to 36 months
Secondary	Work Productivity and Activity Impairment: General Health (WPAI:GH)	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).	Up to 36 months
Secondary	Adult Carer Quality of Life (AC-QoL) Questionnaire	The AC-QoL is a 40-item tool that measures the overall QoL for adult carers, and subscale scores for eight domains of QoL: support for caring, caring choice, caring stress, money matters; personal growth; sense of value, ability to care, and carer satisfaction. It is self-administered to adult carers and should take no longer than 10 minutes to complete. Scores range from 0 to 120, with higher scores indicating greater QoL.	Up to 36 months
Secondary	Dose of Lanadelumab	Dose of lanadelumab used during the study will be assessed.	From start of the study up to 36 months
Secondary	Frequency of Administration of Lanadelumab	Frequency of lanadelumab injections during the study will be assessed.	From start of the study up to 36 months
Secondary	Participants' Adherence Rate to Treatment with Lanadelumab	Adherence is defined as the proportion of time on treatment over the study observation time period, times the proportion of actual lanadelumab doses taken per label as reported by participants through an administration diary over the total expected lanadelumab doses per label during the treatment period.	Up to 36 months
Secondary	Frequency of Use of Approved Lanadelumab Dosing Regimens	Frequency of use of approved lanadelumab dosing regimens will be assessed.	Up to 36 months
Secondary	Frequency of Administration Modalities of Lanadelumab	Frequency of administration modalities of lanadelumab (self-administration versus (vs). administration by a caregiver, HCP, or other) will be assessed.	Up to 36 months
Secondary	Number of Administrations of Lanadelumab Before Participant Discontinuation	Number of administrations of lanadelumab before participant discontinuation from the study will be assessed.	Up to 36 months
Secondary	Frequency of Reasons for Discontinuation of Treatment With Lanadelumab Reported by Participants	Frequency of reasons for discontinuation of treatment with lanadelumab reported by participants will be assessed.	Up to 36 months
Secondary	Adverse Event (AE) Incidence, Type, Seriousness and Relatedness to Lanadelumab Treatment	An AE can be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product whether or not related to the medicinal product. AE incidence, type, seriousness and relatedness to lanadelumab treatment will be assessed.	From start of the study up to 36 months
Secondary	Severity of Non-Serious Adverse Events (AEs)	Severity of AEs is determined by using the following definitions: Mild: A type of AE that is usually transient and may require only minimal treatment or therapeutic intervention, the event does not generally interfere with activities of daily living. Moderate: A type of AE that is usually alleviated with specific therapeutic intervention, the event interferes with usual activities of daily living, causing discomfort, but poses no significant or permanent risk of harm to the research participant. Severe: A type of AE that interrupts usual activities of daily living (ADL), or significantly affects clinical status, or may require intensive therapeutic intervention. Severity of non-serious AEs will be assessed.	From start of the study up to 36 months

External Links

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