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Study to Evaluate the Real-world Effectiveness of Lanadelumab in Participants With Hereditary Angioedema (HAE) (EMPOWER)

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ClinicalTrials.gov Identifier: NCT03845400
Recruitment Status : Not yet recruiting
First Posted : February 19, 2019
Last Update Posted : February 19, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
This observational, prospective study aims to evaluate the real-world effectiveness of lanadelumab in participants with hereditary angioedema (HAE). Participant-reported attack diaries and patient-reported outcomes (PROs) as well as physician assessments at standard of care (SoC) visits will be used to describe HAE attack rates, treatment patterns, healthcare utilization and participant quality of life.

Condition or disease
Hereditary Angioedema (HAE)

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Study Type : Observational
Estimated Enrollment : 270 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Observational, Non-interventional, Study of Patients With Hereditary Angioedema in the United States and Canada (EMPOWER Study)
Estimated Study Start Date : March 31, 2019
Estimated Primary Completion Date : September 30, 2023
Estimated Study Completion Date : September 30, 2023

Resource links provided by the National Library of Medicine


Group/Cohort
Type I or Type II HAE Participants
Participants will be followed for 36 months after the enrollment date up to the time of withdrawal, lost to follow-up, death or end of follow-up (36 months) whichever comes first.



Primary Outcome Measures :
  1. Number of Hereditary Angioedema (HAE) Attacks [ Time Frame: From enrollment up to 36 months ]
    Number of HAE attacks before and after lanadelumab initiation will be reported.


Secondary Outcome Measures :
  1. Dose of Lanadelumab Injections [ Time Frame: From enrollment up to 36 months ]
    Dose of lanadelumab injections used during the study will be reported.

  2. Frequency of Lanadelumab Injections [ Time Frame: From enrollment up to 36 months ]
    Frequency of lanadelumab injections during the study will be reported.

  3. Proportion of Injections Based on the Type of Administration of Lanadelumab [ Time Frame: From enrollment up to 36 months ]
    Proportion of injections based on the type of administration of lanadelumab (self, caregiver, health care provider [HCP], other) will be reported.

  4. Time From Diagnosis to Lanadelumab Initiation [ Time Frame: From enrollment up to 36 months ]
    Time from diagnosis of HAE attack(s) to lanadelumab treatment initiation will be reported.

  5. Proportion of Participants who Discontinue Lanadelumab [ Time Frame: From enrollment up to 36 months ]
    Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Proportion of participants who discontinue lanadelumab treatment will be reported.

  6. Number of Lanadelumab Injections Before Discontinuation per Reason for Injection [ Time Frame: From enrollment up to 36 months ]
    Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Number of injections administered by participant before discontinuation per reason for injection will be reported.

  7. Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s) [ Time Frame: From enrollment up to 36 months ]
    Number of hospitalizations due to HAE attack(s) will be reported.

  8. Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s) [ Time Frame: From enrollment up to 36 months ]
    Number of emergency room (ER) visits due to HAE attack(s) will be reported.

  9. Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s) [ Time Frame: From enrollment up to 36 months ]
    Number of physician visits due to HAE attack(s) will be reported.

  10. Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s) [ Time Frame: From enrollment up to 36 months ]
    Number, type of rescue medications taken by the participants at the time of HAE attack(s) per the type of administration (self, caregiver, HCP, other) will be reported.

  11. Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT) [ Time Frame: From enrollment up to 36 months (every 3 months) ]
    AECT is a questionnaire and not a validated patient-reported outcome (PRO). HAE control score is evaluated as frequency of answers [very often, often, sometimes, seldom, not at all] to the following questions at record closest to enrollment date): 1. In the last 3 months, how often have you had angioedema? 2. In the last 3 months, how much has your quality of life been affected by angioedema? 3. In the last 3 months, how much has the unpredictability of your angioedema bothered you? 4. In the last 3 months, how well has your angioedema been controlled by your therapy?

  12. Angioedema Quality of Life (AE-QoL) Score [ Time Frame: From enrollment up to 36 months (every 3 months) ]
    The AE-QoL is developed to measure health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. It is a self-administered PRO designed for adults aged 18 years and older 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.

  13. Work Productivity and Activity Impairment: General Health (WPAI:GH) Score [ Time Frame: From enrollment up to 36 months (every 3 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 7 days. It can be self- or interviewer-administered to adults aged 16 years or older. This 6-item PRO covers work (5 items) and daily activities (1 item) using yes/no or numerical answers (number of hours). WPAI:GH outcomes are expressed as impairment percentages. An overall work productivity score (health or symptom) [%0WP], 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]: %0WP = %WTW * %PW.

  14. Treatment Satisfaction (TSQM-9) Score [ Time Frame: From enrollment up to 36 months (every 3 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 designed for adults aged 18 years or older with a recall period of 2 to 3 weeks, or since the last medication use. Version TSQM-9 includes 3 domains: effectiveness (3 items), convenience (3 items), and global satisfaction scale (3 items). Scores range from 0 to 100.



Information from the National Library of Medicine

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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 a diagnosis of Type I or Type II HAE. The study will target 70% enrollment of new lanadelumab users.
Criteria

Inclusion Criteria:

  • Voluntarily provide written, signed, and dated (personally or via a legally-authorized representative) informed consent/and assent as applicable to participate in the study. Expression of understanding and agreement by fully informed parent(s) or legal guardian is required to permit the investigator to enroll a child in this study. The choice of the terms parental consent or parental permission in different regions may reflect local legal/regulatory and ethical considerations.
  • Diagnosis of HAE Type I or Type II.
  • Ability to use a mobile device for data collection in the study.

Exclusion Criteria:

  • Participation in any interventional clinical trial at the time of enrollment.
  • Unable to provide written, signed, and dated informed consent/assent.
  • Investigator believes that the participant is not a suitable candidate for the study.

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): NCT03845400


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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United States, Ohio
Bernstein Clinical Research Center, LLC Not yet recruiting
Cincinnati, Ohio, United States, 45231
Contact: Site Contact    513-354-1746    jonathan.bernstein@uc.edu   
Principal Investigator: Jonathan Bernstein         
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03845400     History of Changes
Other Study ID Numbers: SHP643-403
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: February 19, 2019
Last Verified: February 2019
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

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn