CSL964_5003: Benefits of patient self-administration and Respreeza® 4g and 5g vials - an observational study

• Conditions: E88.0; Disorders of plasma-protein metabolism, not elsewhere classified

• Registration Number: DRKS00030885
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-12-08
• Study Completion: 2023-08-01
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

Proven diagnosis of AATD, defined as AAT serum level <11 µM (50 mg·dL-1) and/or proteinase inhibitor genotypes ZZ, SZ and compound heterozygotes or homozygotes of other rare deficient variants.
- Treated with / or self-administered Respreeza® IV according to current SmPC
- Understanding of the study and agreement to give written informed consent.
- Able and willing to participate in the study

Exclusion Criteria

- Lack of patient consent

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary endpoint will be the overall assessment of the treatment regime (see question 18, Appendix 17.2).

Secondary Outcome Measures

Name	Time	Method
Secondary endpoints are<br>? Time for reconstitution and time for administration of a dose (nurse)<br>? Evaluation of a patient’s perceived feasibility and satisfaction of the Respreeza® self-administration home treatment