Study to Evaluate the Real-World Long-Term Effectiveness of Lanadelumab in Participants With Hereditary Angioedema (HAE) (ENABLE)
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|ClinicalTrials.gov Identifier: NCT04130191|
Recruitment Status : Recruiting
First Posted : October 17, 2019
Last Update Posted : September 16, 2020
|Condition or disease|
|Hereditary Angioedema (HAE)|
|Study Type :||Observational|
|Estimated Enrollment :||200 participants|
|Official Title:||A Three-year, Non-interventional, Prospective, Multicenter Study to Evaluate the Long-term Effectiveness of Lanadelumab in Real-world Clinical Practice (ENABLE)|
|Actual Study Start Date :||December 11, 2019|
|Estimated Primary Completion Date :||April 30, 2024|
|Estimated Study Completion Date :||April 30, 2024|
Participants with hereditary angioedema (HAE)
Participants who initiate treatment with lanadelumab according to current product labelling will be enrolled and followed for up to 36 months after the enrollment in the study.
- Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks [ Time Frame: Up to 36 months ]HAE attack is defined as a discrete episode during which the participant progress from no angioedema to symptoms of angioedema. Rate of participant-reported HAE attacks during treatment with lanadelumab up to 36 months will be assessed.
- Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70 [ Time Frame: From Day 70 up to 36 months ]Rate of participant-reported HAE attacks during treatment with lanadelumab from day 70 will be assessed.
- Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks [ Time Frame: Up to 36 months ]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).
- Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location [ Time Frame: Up to 36 months ]Number of on-treatment participant-reported HAE attacks based on anatomical (peripheral, abdominal, laryngeal) location will be assessed.
- Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used [ Time Frame: Up to 36 months ]Proportion of HAE attacks for which participants use on-demand therapy will be assessed.
- Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used [ Time Frame: Up to 36 months ]Time to first HAE attack for which on-demand therapy is used will be assessed.
- Proportion of Hereditary Angioedema (HAE) Attacks Requiring Visit to an Healthcare Provider (HCP), Access to an Emergency Room (ER), or Hospitalization [ Time Frame: Up to 36 months ]Proportion of HAE Attacks requiring visit to HCP, access to an ER, or hospitalization will be assessed.
- Angioedema Quality of Life (AE-QoL) [ Time Frame: Up to 36 months ]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.
- Fatigue Severity Scale (FSS) [ Time Frame: Up to 36 months ]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.
- Hospital Anxiety and Depression Scale (HADS) [ Time Frame: Up to 36 months ]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.
- Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) [ Time Frame: Up to 36 months ]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).
- Work Productivity and Activity Impairment: General Health (WPAI:GH) [ Time Frame: Up to 36 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).
- Adult Carer Quality of Life (AC-QoL) Questionnaire [ Time Frame: Up to 36 months ]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.
- Dose of Lanadelumab [ Time Frame: From start of the study up to 36 months ]Dose of lanadelumab used during the study will be assessed.
- Frequency of Administration of Lanadelumab [ Time Frame: From start of the study up to 36 months ]Frequency of lanadelumab injections during the study will be assessed.
- Participants' Adherence Rate to Treatment with Lanadelumab [ Time Frame: Up to 36 months ]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.
- Frequency of Use of Approved Lanadelumab Dosing Regimens [ Time Frame: Up to 36 months ]Frequency of use of approved lanadelumab dosing regimens will be assessed.
- Frequency of Administration Modalities of Lanadelumab [ Time Frame: Up to 36 months ]Frequency of administration modalities of lanadelumab (self-administration versus (vs). administration by a caregiver, HCP, or other) will be assessed.
- Number of Administrations of Lanadelumab Before Participant Discontinuation [ Time Frame: Up to 36 months ]Number of administrations of lanadelumab before participant discontinuation from the study will be assessed.
- Frequency of Reasons for Discontinuation of Treatment With Lanadelumab Reported by Participants [ Time Frame: Up to 36 months ]Frequency of reasons for discontinuation of treatment with lanadelumab reported by participants will be assessed.
- Adverse Event (AE) Incidence, Type, Seriousness and Relatedness to Lanadelumab Treatment [ Time Frame: From start of the study up to 36 months ]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.
- Severity of Non-Serious Adverse Events (AEs) [ Time Frame: From start of the study up to 36 months ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04130191
|Contact: Takeda Development Center Americas Contact||+1 866 842 5335||ClinicalTransparency@takeda.com|
|Medical University of Vienna||Recruiting|
|Vienna, Austria, 1090|
|Contact: Site Contact +431404007725 firstname.lastname@example.org|
|Principal Investigator: Tamar Kinaciyan|
|Charité - Universitätsmedizin Berlin||Recruiting|
|Berlin, Germany, 10117|
|Contact: Site Contact +4930450518043 email@example.com|
|Principal Investigator: Marcus Maurer|
|Universitatsklinikum Dusseldorf||Not yet recruiting|
|Düsseldorf, Germany, 40225|
|Contact: Site Contact +492118119480 firstname.lastname@example.org|
|Principal Investigator: Martin Wagenmann|
|Klinikum der Johann-Wolfgang Goethe-Universitat||Not yet recruiting|
|Frankfurt, Germany, 60590|
|Contact: Site Contact +496963016312 email@example.com|
|Principal Investigator: Emel Aygören-Pürsün|
|Universitatsklinikum Schleswig-Holstein||Not yet recruiting|
|Lübeck, Germany, 23538|
|Contact: Site Contact +4945150041646 firstname.lastname@example.org|
|Principal Investigator: Andreas Recke|
|Hämophilie Zentrum Rhein Main GmbH||Recruiting|
|Mörfelden-Walldorf, Germany, 64546|
|Contact: Site Contact +4961059638909 email@example.com|
|Principal Investigator: Inmaculada Martinez|
|Klinikum rechts der Isar der Technischen Universität München||Not yet recruiting|
|München, Germany, 81675|
|Contact: Site Contact +498941409592 firstname.lastname@example.org|
|Principal Investigator: Ulrich Strassen|
|Universitätsklinikum Münster||Not yet recruiting|
|Münster, Germany, 48149|
|Contact: Site Contact +492518356506 email@example.com|
|Principal Investigator: Randolf Brehler|
|Universitätsklinikum Ulm||Not yet recruiting|
|Ulm, Germany, 89075|
|Contact: Site Contact +49073150059501 firstname.lastname@example.org|
|Principal Investigator: Jens Greve|
|Bnai Zion Medical Center||Recruiting|
|Haifa, Israel, 31048|
|Contact: Site Contact +97248359607 email@example.com|
|Principal Investigator: Aharon Kessel|
|Sheba Medical Center - PPDS||Recruiting|
|Ramat Gan, Israel, 52621|
|Contact: Site Contact +97235308087 firstname.lastname@example.org|
|Principal Investigator: Nancy Agmon-Levin|
|Luzerner Kantonsspital LUKS||Recruiting|
|Luzern, Switzerland, 6000|
|Contact: Site Contact +41412055147 email@example.com|
|Principal Investigator: Walter Wuillemin|
|Royal London Hospital / Bart's and the London NHS Trust||Not yet recruiting|
|London, United Kingdom, E1 2ES|
|Contact: Site Contact +4402032460264 firstname.lastname@example.org|
|Principal Investigator: Sorena Kiani|
|Study Director:||Study Director||Takeda Development Center Americas|