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

Organizational Data

DRKS-ID:
DRKS00030885
Recruitment Status:
Recruiting complete, study complete
Date of registration in DRKS:
2022-12-08
Last update in DRKS:
2023-08-21
Registration type:
Prospective

Acronym/abbreviation of the study

Benefits of Respreeza self-administration

URL of the study

No Entry

Brief summary in lay language

Respreeza® is available as a powder and solvent to be made up into a solution for infusion (drip) into a vein. The first infusion will be given under the supervision of a healthcare professional experienced in the management of AATD. Subsequent infusions can be administered by a caregiver or by the patient. The aim of the present observational study is to obtain opinions from nursing staff and patients regarding the use of Respreeza®. This study consists of two parts: Part 1: Evaluation by medical technical assistants (MTA) This part of the study includes the reconstitution and administration of Respreeza® by a single MTA to 5 patients with 4 g and 5 g vials and further AATD therapy to the same number (5) of patients with 1 g vials, the time for reconstitution and the time taken to administer each dose is recorded by an independent member of the nursing staff. Nursing staff and admitting patients' ratings of the reconstitution and administration processes are recorded using a standard usability scale. Part 2: Patient Assessment This part of the study includes 7 patients who self-administer Respreeza® at home. Patients will be asked about their experiences of self-administration using the same questionnaire, with a focus on perceived advantages and benefits as well as perceived disadvantages.

Brief summary in scientific language

Respreeza® is approved in Germany for treatment of Alpha 1 Antitrypsin Deficiency (AATD). The first administration (infusion) is given under the supervision of a trained healthcare professional (HPC). Following that, routine administrations can either be done by HCPs or by patients themselves. However, results of a survey (4) demonstrated that patients self-administering are very satisfied because of improved independence but require training to self-administer independently. Primary objective of this pilot study is an assessment of a nurse and the patients regarding the reconstitution and administration processes while administering the drug. Hence, the study will have two parts: Part 1: Nurse assessment ▪ This part will involve a single nurse with previous experience of reconstituting and administering Respreeza® and other AATD therapy to reconstitute and administer Respreeza® to 5 patients who has been previously treated with Respreeza, using 4g and 5g vials, and another AATD therapy to the same number (5) of patients (who has been previously treated and matched for their doses to within 0.5g), using 1g vials of that other therapy. That means, ▪ time for reconstitution and time for administration of each dose will be recorded by one dedicated individual, either an independent researcher or the nurse. ▪ assessments of the nurse and the receiving patients regarding the reconstitution and administration processes will be recorded. Part 2: Patient assessment ▪ This part will involve seven (7) patients who have experience with Respreeza® self-administration (at least 3 successful self-administrations). ▪ In addition, patients having completed part 1 and switched (after having received adequate training) to self-administration can also participate in part 2 (see above). ▪ The patients will be asked about their experiences of self-administration, with focus on any perceived advantages and benefits of doing so, as well as any perceived disadvantages. This is a pilot study to retrieve data for a planned larger / confirmatory study. Further, according to AMG §4, 23 this study is categorized as a non-interventional study.

Health condition or problem studied

ICD10:
E88.0 - Disorders of plasma-protein metabolism, not elsewhere classified
Healthy volunteers:
No

Interventions, Observational Groups

Arm 1:
Medical staff assessment
Arm 2:
Patient assessment

Endpoints

Primary outcome:
Primary endpoint will be the overall assessment of the treatment regime (see question 18, Appendix 17.2).
Secondary outcome:
Secondary endpoints are ▪ Time for reconstitution and time for administration of a dose (nurse) ▪ Evaluation of a patient’s perceived feasibility and satisfaction of the Respreeza® self-administration home treatment

Study Design

Purpose:
Treatment
Retrospective/prospective:
Prospective
Study type:
Non-interventional
Longitudinal/cross-sectional:
Cross-sectional study
Study type non-interventional:
Epidemiological study

Recruitment

Recruitment Status:
Recruiting complete, study complete
Reason if recruiting stopped or withdrawn:
No Entry

Recruitment Locations

Recruitment countries:
  • Germany
Number of study centers:
Monocenter study
Recruitment location(s):
  • University medical center Thoraxklinik Heidelberg

Recruitment period and number of participants

Planned study start date:
2022-12-19
Actual study start date:
2023-01-18
Planned study completion date:
2023-09-30
Actual Study Completion Date:
2023-08-01
Target Sample Size:
17
Final Sample Size:
17

Inclusion Criteria

Sex:
All
Minimum Age:
18 Years
Maximum Age:
no maximum age
Additional 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

Addresses

Primary Sponsor

CSL Behring GmbH
Phillip-Reis-Straße 2
65795 Hattersheim
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry
Investigator Sponsored/Initiated Trial (IST/IIT):
No

Contact for Scientific Queries

Thoraxklinik Heidelberg
Prof. Dr. Felix Herth
Röntgenstraße 1
69126 Heidelberg
Germany
Telephone:
06221 396-1201
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Contact for Public Queries

Thoraxklinik Heidelberg
Prof. Dr. Felix Herth
Röntgenstraße 1
69126 Heidelberg
Germany
Telephone:
06221 396-1201
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Principal Investigator

Thoraxklinik Heidelberg
Prof. Dr. Felix Herth
Röntgenstraße 1
69126 Heidelberg
Germany
Telephone:
06221 396-1201
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Other contact for public queries

CRO Dr. med. Kottmann GmbH & Co KG
Beverstr. 64
59077 Hamm
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Sources of Monetary or Material Support

Commercial (pharmaceutical industry, medical engineering industry, etc.)

CSL Behring GmbH
65795 Hattersheim
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Ethics Committee

Address Ethics Committee

Ethikkommission der Medizinischen Fakultät Heidelberg
Alte Glockengießerei 11/1
69115 Heidelberg
Germany
Telephone:
+49-6221-338220
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Vote of leading Ethics Committee

Date of ethics committee application:
2021-08-10
Ethics committee number:
S-656/2021
Vote of the Ethics Committee:
Approved
Date of the vote:
2021-11-09

Further identification numbers

Other primary registry ID:
No Entry
EudraCT Number:
No Entry
UTN (Universal Trial Number):
No Entry
EUDAMED Number:
No Entry

IPD - Individual Participant Data

Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
No
IPD Sharing Plan:
No Entry

Study protocol and other study documents

Study protocols:
No Entry
Study abstract:
No Entry
Other study documents:
No Entry
Background literature:
No Entry
Related DRKS studies:
No Entry

Publication of study results

Planned publication:
No Entry
Publikationen/Studienergebnisse:
No Entry
Date of first publication of study results:
No Entry
DRKS entry published for the first time with results:
No Entry

Basic reporting

Basic Reporting / Results tables:
No Entry
Brief summary of results:
No Entry