Real-life Ecological Momentary Assessment of Lived Burden in Hereditary AngioEdema

NCT07448181 | Recruiting

• 1 other identifier: 2025-3.11/388
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to conduct an in-depth analysis of the Burden of Disease (BoD) perceived by patients with Hereditary Angioedema (HAE), through daily prospective observations based on Ecological Momentary Assessment (EMA) via digital surveys and standardised questionnaires. Participants will answer online survey questions about their perceived burden of disease for 8 consecutive weeks. The main hypothesis is that daily prospective observation (EMA) will reveal a higher and more fluctuating burden of disease compared to traditional retrospective scales, providing a more accurate representation of the impact of HAE on patients' daily lives.

Conditions

Hereditary Angioedema With C1 Esterase Inhibitor Deficiency; Hereditary Angioedema - Type 1; Hereditary Angioedema - Type 2

Keywords

Hereditary Angioedema; Burden of disease; Ecological Momentary Assessment; Quality of Life

Outcome Measures

Primary Outcomes (5)

• Emotional Burden via Ecological Momentary Assessment (EMA)

  Evaluation of emotional states (anxiety, mood, stress, and fear of attacks) through a Visual Analogue Scale (VAS). Each item is scored on a range from 0 to 100, where 0 indicates "no emotional impact" and 100 indicates "maximum emotional distress.

  Every other day for 8 weeks

• Socio-occupational Impairment via Ecological Momentary Assessment (EMA)

  Assessment of the impact of Hereditary Angioedema (HAE) on daily, social, and professional activities. Measured via a scale ranging from 1 to 10, where 1 indicates "no impairment" and 10 indicates "maximum perceived impairment"

  Every other day for 8 weeks

• Disease Activity

  Assessment of the physical burden using the Angioedema Activity Score (AAS): A daily symptom diary, total score from 0 to 15.

  AAS is collected every other day for 8 weeks

• Angioedema Quality of Life

  Assessment of quality of life impairment using the AE-QoL questionnaire. The total score ranges from 17 to 85, where higher scores indicate greater impairment.

  After 4 weeks and at the end of the 8 weeks

• Disease Control

  Assessment of disease control using the AECT. The total score ranges from 0 to 16, where lower scores indicate poorer disease control.

  After 4 weeks and at the end of the 8 weeks.

Secondary Outcomes (5)

• Anxiety and Depressive Symptoms

  Every other week for 8 weeks

• Work Productivity and Activity Impairment (WPAI:GH)

  Once a week for 8 weeks

• Within-person Variability of Emotional States (EMA)

  Every other day for 8 weeks

• Difference in Burden of Disease between LTP and ODT groups

  8 weeks

• Correlation between EMA scores and Traditional Questionnaires

  8 weeks

Interventions

ecological momentary assessment OTHER

A real-time data collection method involving brief, repeated digital surveys completed by participants in their natural environments. Participants will receive two notifications every other day for 8 weeks via a mobile application. Surveys are designed to assess physical symptoms, socio-occupational status, and emotional states related to Hereditary Angioedema (HAE) to capture daily fluctuations in disease burden.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The study involves the participation of adult patients with a confirmed diagnosis of hereditary angioedema, belonging to the ITACA (Italian Network for Hereditary and Acquired Angioedema) national cohort and receiving follow-up care at the IRCCS Maugeri Milano (Via Camaldoli) center. Participation in the study is voluntary and subject to the signing of informed consent. Patients will complete the questionnaires independently via their own smartphones, with no requirement for in-person visits.

You may qualify if:

• Confirmed diagnosis of Type 1 or Type 2 hereditary angioedema;
• Age ≥ 18 years;
• Ability to understand instructions and provide informed consent;
• Ownership and proficiency in using a personal smartphone compatible with the m-Path application (Android or iOS);
• Willingness to participate in the study for the entire duration of the observation period (8 weeks).

You may not qualify if:

• Diagnosis of acquired angioedema or other forms of angioedema unrelated to C1-inhibitor deficiency;
• Severe cognitive or psychiatric disorders that compromise the ability to complete the questionnaires independently;
• Age \< 18 years;

Sponsors & Collaborators

• Istituti Clinici Scientifici Maugeri SpA: lead

Study Sites (1)

Istituti Clinici Scientifici Maugeri, Milan, Milan 20138

Milan, 20138, Italy

RECRUITING

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

Ecological Momentary Assessment

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Intervention Hierarchy (Ancestors)

Psychological Tests; Behavioral Disciplines and Activities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: March 4, 2026
• Study Start: February 23, 2026
• Primary Completion (Estimated): February 28, 2027
• Study Completion (Estimated): February 28, 2027
• Last Updated: March 4, 2026

Record last verified: 2026-03

Locations

IT (1)