Patient-related outcome measures (PROM) for use in patients with alpha-1-antitrypsin deficiency (AATD)

Patient-related outcome measures (PROM) for use in patients with alpha-1-antitrypsin deficiency (AATD) - PROMs for AATD

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00037649

Enrollment

400

Registered

2025-08-12

Start date

2022-10-01

Completion date

Unknown

Last updated

2025-10-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

alpha-1-antitrypsin deficiency (E88.00) E88.00

Interventions

Group 1: Psychometric validation (online survey): Patients with AATD were contacted via three registries and invited to complete the PROM questionnaire online to assess validity and reliability indica

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

Inclusion criteria: - Registered in an AATD patient registry, appointment at a clinic, or participation in a patient event - Written informed consent to participate in the study - Mentally and physically able to complete the PROM - Sufficient language skills to understand and respond to the questionnaire

Exclusion criteria

Exclusion criteria: - No confirmed diagnosis of alpha-1 antitrypsin deficiency (AATD)

Design outcomes

Primary

Measure	Time frame
Primary outcome is the evaluation of the psychometric properties (validity, reliability), acceptance, and comprehensibility of the PROM used to assess health-related quality of life in patients with AATD (online study).	—

Secondary

Measure	Time frame
Secondary outcome is the evaluation of the feasibility of the PROM in routine clinical care.	—

Countries

Germany

Contacts

Public ContactMichael Koller

Universitätsklinikum Regensburg, Zentrum für Klinische Studien

michael.koller@ukr.de+49 941 944 5630

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026