A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema

Browse trials for

• Angioedema 164
• Angioedemas, Hereditary 171

Angioedema

164 studies on the registry are indexed under Angioedema; 19 are open to participants now.

This study's enrollment of 221 is above the median of 163 across 44 observational studies indexed under Angioedema.

Lead sponsor

Takeda is the lead sponsor of 1,002 studies on the registry; 92 are open to participants now.

Of its 173 completed or terminated interventional studies of FDA-regulated products, 149 (86%) have results posted.

Study population

Groups and cohorts

• Participants with HAE

  Participants' data will be collected retrospectively from electronic medical records using chart review of de-identified data on participant demographics, HAE medical history and information on diagnostics, treatments and disease course assessments that are routinely performed in accordance with current guidelines and/or local standard of care which are entered into an Electronic data capture (EDC) system over multiple data collection waves up to 6 months.

Primary outcomes

1. Number of Participants With HAE Attacks Occurrence During Study Period

   HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) during study period will be reported.

   Time frame: Throughout the study period (up to 6 months)

2. Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period

   HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening will be reported.

   Time frame: Throughout the study period (up to 6 months)

3. Time Interval Between HAE Attacks During Study Period

   Time interval between HAE attacks during the study period will be reported.

   Time frame: Throughout the study period (up to 6 months)

4. Duration of Each HAE Attacks During Study Period

   Duration of each HAE attacks during study period will be reported.

   Time frame: Throughout the study period (up to 6 months)

5. Number of Participants Based on Type of HAE Prophylactic Treatments

   Number of participants based on type of HAE prophylactic treatment will be reported.

   Time frame: Throughout the study period (up to 6 months)

6. Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment

   Number of participants based on long-term or short-term HAE prophylactic treatment will be reported.

   Time frame: Throughout the study period (up to 6 months)

7. Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation

   Number of participants categorized based on reasons for HAE prophylactic treatment initiation will be reported.

   Time frame: Throughout the study period (up to 6 months)

8. Duration of HAE Prophylactic Treatment

   Duration of HAE prophylactic treatment includes treatment start and end dates will be reported.

   Time frame: Throughout the study period (up to 6 months)

9. Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment

   Number of participants categorized based on dose and route of administration for HAE prophylactic treatment will be reported.

   Time frame: Throughout the study period (up to 6 months)

10. Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation

    Number of participants categorized based on reasons for HAE prophylactic treatment discontinuation will be reported.

    Time frame: Throughout the study period (up to 6 months)

11. Number of Participants Based on Type of HAE On-demand Treatment

    Number of participants based on type of HAE on-demand treatment will be reported.

    Time frame: Throughout the study period (up to 6 months)

12. Duration of HAE On-demand Treatment

    Duration of HAE on-demand treatment includes treatment start and end dates will be reported.

    Time frame: Throughout the study period (up to 6 months)

13. Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment

    Number of participants categorized based on dose, route of administration and setting of administration for HAE on-demand treatment will be reported.

    Time frame: Throughout the study period (up to 6 months)

14. Number of Participants Associated Between Prescriptions Patterns and HAE Attacks

    Number of participants with association between prescriptions patterns and HAE attacks related to frequency or severity. Generalized linear models (GLM) will be used to evaluate the association of outcomes of interest with relevant covariates between prescription pattern, HAE attacks, frequency and severity of HAE attacks will be reported.

    Time frame: Throughout the study period (up to 6 months)

Secondary outcomes

1. Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)

   HRU is measured by the number of visits to HCP will be reported.

   Time frame: Throughout the study period (up to 6 months)

2. HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks

   Number of participants with ER/ED visits due to HAE attacks will be reported.

   Time frame: Throughout the study period (up to 6 months)

3. HRU: Number of Participants With Hospitalizations due to HAE Attacks

   Number of participants with hospitalizations due to HAE attacks will be reported.

   Time frame: Throughout the study period (up to 6 months)

4. Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire

   The AE-QoL was developed to measure HRQoL in participants with recurrent angioedema. It is a self-administered patient-reported outcome instrument with a recall period of four weeks. There are 17 items across four domains: functioning (four items), fatigue/mood (five items), fears/shame (six items), and food (two items). Responses use a 5-point Likert scale ranging from 1 (Never) to 5 (Very Often). Global scores range from 0 to 100 and scores by domains range from 0 to 100. An overall score is calculated and a higher score indicates lower quality of life.

   Time frame: Throughout the study period (up to 6 months)

5. HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire

   The EQ-5D-5L descriptive system assesses health in five dimensions (Mobility, Self-Care, Usual Activities, Pain / Discomfort, Anxiety / Depression), each of which has five levels of response (no problems, slight problems, moderate problems, severe problems, extreme problems/unable to). This part of the EQ-5D questionnaire provides a descriptive profile that can be used to generate a health state profile. Health state index scores generally range from less than 0 (where 0 is the value of a health state equivalent to dead; negative values representing values as worse than dead) to 1 (the value of full health), with higher scores indicating higher health utility. The second part of the questionnaire consists of a visual analogue scale (VAS) on which the participant rates his/her perceived health from 0 (the worst imaginable health) to 100 (the best imaginable health).

   Time frame: Throughout the study period (up to 6 months)

6. HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)

   The EQ-5D-Y is composed of two sections. The first section contains one question for each of the five dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. Participants select from three response levels (no problems, some problems, a lot of problems) for each dimension. A health state profile score can be calculated from the responses on these five questions. The second section includes the same visual analogue scale as the EQ-5D-5L. The scale is scored from 0-100. The reference to a high score indicates a better outcome of quality of life.

   Time frame: Throughout the study period (up to 6 months)

7. Number of Participants With HAE Attacks Occurrence in Relevant Subgroups

   HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) in relevant subgroups will be reported.

   Time frame: Throughout the study period (up to 6 months)

8. Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups

   HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).

   Time frame: Throughout the study period (up to 6 months)

9. Time Interval Between HAE Attacks in Relevant Subgroups

   Time interval between HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).

   Time frame: Throughout the study period (up to 6 months)

10. Duration of Each HAE Attacks in Relevant Subgroups

    Duration of each HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).

    Time frame: Throughout the study period (up to 6 months)

11. HAE Attacks Prescription Pattern in Relevant Subgroups

    Prescription pattern of HAE attacks medication by drug class in relevant participant subgroups during study period. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).

    Time frame: Throughout the study period (up to 6 months)

Related links

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

Individual participant data

Plan to share: Yes — 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 information: Study protocol, Sap, Icf, Csr