Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study to Evaluate the Real-World Long-Term Effectiveness of Lanadelumab in Participants With Hereditary Angioedema (HAE) (ENABLE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04130191
Recruitment Status : Recruiting
First Posted : October 17, 2019
Last Update Posted : September 16, 2020
Sponsor:
Collaborator:
Takeda Development Center Americas, Inc.
Information provided by (Responsible Party):
Takeda ( Shire )

Brief Summary:
The purpose of this non-interventional, prospective study is to evaluate the real world long-term effectiveness of lanadelumab in participants with type 1 or type 2 HAE.

Condition or disease
Hereditary Angioedema (HAE)

Layout table for study information
Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine


Group/Cohort
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.



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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).

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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).

  11. 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).

  12. 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.

  13. Dose of Lanadelumab [ Time Frame: From start of the study up to 36 months ]
    Dose of lanadelumab used during the study will be assessed.

  14. 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.

  15. 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.

  16. 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.

  17. 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.

  18. 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.

  19. 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.

  20. 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.

  21. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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.

Information from the National Library of Medicine

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


Contacts
Layout table for location contacts
Contact: Takeda Development Center Americas Contact +1 866 842 5335 ClinicalTransparency@takeda.com

Locations
Layout table for location information
Austria
Medical University of Vienna Recruiting
Vienna, Austria, 1090
Contact: Site Contact    +431404007725    tamar.kinaciyan@meduniwien.ac.at   
Principal Investigator: Tamar Kinaciyan         
Germany
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany, 10117
Contact: Site Contact    +4930450518043    marcus.maurer@charite.de   
Principal Investigator: Marcus Maurer         
Universitatsklinikum Dusseldorf Not yet recruiting
Düsseldorf, Germany, 40225
Contact: Site Contact    +492118119480    martin.wagenmann@uni-duesseldorf.de   
Principal Investigator: Martin Wagenmann         
Klinikum der Johann-Wolfgang Goethe-Universitat Not yet recruiting
Frankfurt, Germany, 60590
Contact: Site Contact    +496963016312    emel.aygoeren@kgu.de   
Principal Investigator: Emel Aygören-Pürsün         
Universitatsklinikum Schleswig-Holstein Not yet recruiting
Lübeck, Germany, 23538
Contact: Site Contact    +4945150041646    andreas.recke@uksh.de   
Principal Investigator: Andreas Recke         
Hämophilie Zentrum Rhein Main GmbH Recruiting
Mörfelden-Walldorf, Germany, 64546
Contact: Site Contact    +4961059638909    inmaculada.martinez@hzrm.de   
Principal Investigator: Inmaculada Martinez         
Klinikum rechts der Isar der Technischen Universität München Not yet recruiting
München, Germany, 81675
Contact: Site Contact    +498941409592    u.strassen@lrz.tum.de   
Principal Investigator: Ulrich Strassen         
Universitätsklinikum Münster Not yet recruiting
Münster, Germany, 48149
Contact: Site Contact    +492518356506    brehler@ukmuenster.de   
Principal Investigator: Randolf Brehler         
Universitätsklinikum Ulm Not yet recruiting
Ulm, Germany, 89075
Contact: Site Contact    +49073150059501    jens.greve@uniklinik-ulm.de   
Principal Investigator: Jens Greve         
Israel
Bnai Zion Medical Center Recruiting
Haifa, Israel, 31048
Contact: Site Contact    +97248359607    aharon.kessel@b-zion.org.il   
Principal Investigator: Aharon Kessel         
Sheba Medical Center - PPDS Recruiting
Ramat Gan, Israel, 52621
Contact: Site Contact    +97235308087    nancy.agmon-levin@sheba.health.gov.il   
Principal Investigator: Nancy Agmon-Levin         
Switzerland
Luzerner Kantonsspital LUKS Recruiting
Luzern, Switzerland, 6000
Contact: Site Contact    +41412055147    walter.wuillemin@luks.ch   
Principal Investigator: Walter Wuillemin         
United Kingdom
Royal London Hospital / Bart's and the London NHS Trust Not yet recruiting
London, United Kingdom, E1 2ES
Contact: Site Contact    +4402032460264    skiani@nhs.net   
Principal Investigator: Sorena Kiani         
Sponsors and Collaborators
Shire
Takeda Development Center Americas, Inc.
Investigators
Layout table for investigator information
Study Director: Study Director Takeda Development Center Americas
Layout table for additonal information
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT04130191    
Other Study ID Numbers: SHP643-402
TAK-743-402 ( Other Identifier: Takeda Development Center Americas )
First Posted: October 17, 2019    Key Record Dates
Last Update Posted: September 16, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes