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A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

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: NCT05489640
Recruitment Status : Not yet recruiting
First Posted : August 5, 2022
Last Update Posted : September 21, 2022
Sponsor:
Information provided by (Responsible Party):
Takeda

Brief Summary:

The main aim of this study is to describe the treatment patterns, characteristics and outcomes of people with HAE who are currently receiving icatibant in the homecare setting in the United Kingdom (UK).

Participants will be treated with icatibant according to their routine practice via homecare service for icatibant within the UK. Data will be directly collected from participants via study diaries and questionnaires. Participants will be contacted approximately every 90 days during study duration (this can occur via phone or as a face-to-face visit).


Condition or disease Intervention/treatment
Hereditary Angioedema (HAE) Other: No Intervention

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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Real-World Study to Determine Patient Characteristics, Treatment Patterns, Clinical and Patient-Reported Outcomes of Patients With Hereditary Angioedema That Self-Administered Icatibant Using Homecare Services in the UK
Estimated Study Start Date : September 30, 2022
Estimated Primary Completion Date : November 30, 2023
Estimated Study Completion Date : November 30, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Icatibant

Group/Cohort Intervention/treatment
HAE Participants
Adult participants with a diagnosis of HAE who are receiving treatment according to routine clinical practice and prescribed all treatments in accordance with the approved marketing authorization will be enrolled in this study. Data will be directly collected from participants via patient reported diaries and paper- based and/or electronic homecare records as appropriate for UK participants using homecare services for icatibant. No study specific intervention will be administered in this study.
Other: No Intervention
No Intervention will be administered in this study.




Primary Outcome Measures :
  1. Number of Participants Treated with Icatibant in the Homecare Setting who Were Receiving Prophylaxis Treatment at Index [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
  2. Number of Participants Treated with Icatibant in the Homecare Setting in Each Prophylactic Treatment Type [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]

Secondary Outcome Measures :
  1. Number of Participants Categorized by Their Demographic Characteristics [ Time Frame: Baseline Period [3 months prior to Index (day of the first homecare telephonic consultation/visit)] ]
    Demographic characteristics will include age at index (categories: 18-29, 30-39, 40-49, 50-59, 60+ years), sex (male, female, non-binary), and ethnic group.

  2. Number of Participants Categorized by Their Clinical Characteristics [ Time Frame: At Index (day of the first homecare telephonic consultation/visit) ]
    Clinical characteristics will include patient reported HAE type (Type I or Type II), categories of duration (participant's self-administration of icatibant at home and time since HAE diagnosis) at index, comorbidities, prior HAE related concomitant medication details, categories of details of deaths during the study observation period.

  3. Rate of New HAE Attacks [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]

    Rate of new HAE attacks and rate of new HAE attacks that required treatment in homecare setting will be recorded as number of HAE attacks per participant per month. Data would be reported for pre-defined time-period categories as follows:

    between index to month 3, between month 3 to 6, month 6 to 9 and month 9 to 12.


  4. Frequency of Visits to Accident and Emergency Services Related to an HAE Attack [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    Frequency of visits to Accident and Emergency Services related to an HAE attack will be defined as number of visits to Accidents and Emergency Services during pre-defined time period. Data would be reported for pre-defined time-period categories as follows: between index to month 3, between month 3 to 6, month 6 to 9 and month 9 to 12.

  5. Time from the Start of Each HAE Attack to Administration of Icatibant in the Homecare Setting [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    The summary data for time (in hours) from the start of each HAE attack to administration of icatibant in the homecare setting will be reported.

  6. Frequency of Icatibant Administration Following Start of Each HAE Attack [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    The summary data for number of icatibant administration following start of each HAE attack during the predefined time period will be reported.

  7. Number of Participants by Treatment Patterns [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    Treatment patterns will include assessment of number of participants by treatment dose received for HAE attack and average dose per participant received post-index.

  8. Number of Participants by Treatment Management Patterns [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    Treatment management patterns will include assessment of number of participants by discontinuations of icatibant treatment and reasons for discontinuation (this would be assessed for participants who received prophylaxis treatment during the study), number of face-to-face and telephone-based homecare consultations.

  9. EQ-5D-5L Score [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    The EQ-5D-5L questionnaire will be used to record impact of HAE on participant's quality-of-life (QoL). The EQ-5D-5L is a descriptive system of Health-Related Quality-of-Life (HRQoL) states consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each of which can take 1 of 5 responses. The responses record 5 levels of severity [no problems, slight problems, moderate problems, severe problems, and extreme problems] within a particular dimension. The scores will be the sum of scores from each dimension and can range from 5 (no problems) to 25 (extreme problems).

  10. AE-QoL Total Score [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    The Angioedema Quality of Life (AE-QoL) includes 17 items and 4 domains: functioning, fatigue/mood, fears/shame, and food. Raw scores from each domain will be linearly transformed to a total score of 0 to 100, with higher scores indicating a stronger impairment.

  11. Angioedema Control Test (AECT) Total Score [ Time Frame: Up to 12 months from Index (day of the first homecare telephonic consultation/visit) ]
    The AECT is used to assess participants with recurrent angioedema. The test uses a questionnaire with 4 items, each of which has 5 options and is used to measure the participant's current disease control. HAE control score will be recorded with 5 levels of answers [1=very often, 2=often, 3=sometimes, 4=seldom, 5=not at all] to the following questions: 1) In the last 3 months, how often have you had angioedema? 2) In the last 3 months, how much has your QoL 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? The total scores are the sum of individual scores from 4 items and will range from 4 (Very often) to 20 (not at all). Higher scores will indicate no recurrence.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adult participants with a diagnosis of HAE I or II who are receiving treatment according to routine clinical practice and prescribed all treatments in accordance with the approved marketing authorization will be enrolled in this study.
Criteria

Inclusion Criteria

  • Participants self-administering or receiving care assisted administration of icatibant treatment for patient confirmed diagnosis of HAE Type I or II in the homecare setting
  • Participants who are able and willing to complete the study questionnaires
  • Participants who are willing to be visited or contacted by a member of the homecare or research team at pre-arranged intervals in order to complete questionnaires

Exclusion Criteria

- Participants who have received icatibant as an investigational medicine as part of a clinical trial


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): NCT05489640


Contacts
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Contact: Takeda Contact +1-877-825-3327 medinfoUS@takeda.com

Locations
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United Kingdom
Sciensus
London, United Kingdom, W2 6LA
Contact: Site Contact    07827 831117    Christopher.Appleby@sciensus.com   
Principal Investigator: Christopher Appleby         
Sponsors and Collaborators
Takeda
Investigators
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Study Director: Study Director Takeda
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Responsible Party: Takeda
ClinicalTrials.gov Identifier: NCT05489640    
Other Study ID Numbers: TAK-667-4002
MACS-2021-061502 ( Other Identifier: Takeda )
First Posted: August 5, 2022    Key Record Dates
Last Update Posted: September 21, 2022
Last Verified: September 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment 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 on https://vivli.org/ourmember/takeda/. 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://vivli.org/ourmember/takeda/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Takeda:
Drug Therapy
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
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes