Clinical trial NCT04262284: Respreeza® Self-administration and Learning Program (AmAREtTI Study)
Official title: Respreeza® Self-administration and Learning Program (AmAREtTI Study- Auto-Administration de Respreeza® et Programme d'apprenTIssage)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Alpha-1 Antitrypsin Deficiency
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2020-02-10
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®

Brief summary: According to the Respreeza® Summary of Product Characteristics, the initial infusions must be administered under the supervision of a health professional experienced in the treatment of alpha-1 antitrypsin deficiency, although subsequent infusions may be administered at home by the person responsible for care or by the patient.

Clinical data on self-administration of Respreeza® are however limited and the grounds for self-administration are left to the assessment of the attending physician, who needs to ensure that appropriate training is delivered.

In this context, CSL Behring would like to run a clinical study in order to assess the effectiveness of a home self-administration learning program in terms of switching to self-administration, and the long term maintenance of this administration.


Eligibility criteria: Inclusion Criteria:

* Adult patients (age ≥18 years old).
* Patients suffering from AATD treated by Respreeza® through a peripheral vein at home for at least 3 month (consecutives or not). The 3-month period of treatment with Respreeza® does not necessarily correspond to the 3 months prior to inclusion.
* Patient accompanied by a third person at home
* Patients deemed to be suitable by the investigator for self-administration.
* Patients with a life expectancy of over 3 years.
* Patients who have been informed verbally and in writing via the information leaflet and who have signed the informed consent form.

Exclusion Criteria:

- In order to reflect the reality of everyday practice, no non-inclusion criteria are intended except for patient refusal to take part in the study.

Primary outcomes:
measure: Percentage of patients who switch to self-administration without the presence of a nurse at the end of the learning program
timeFrame: Up to 8 weeks
measure: Percentage of patients who switch to self-administration without the presence of a nurse
timeFrame: Up to 12 months
Secondary outcomes:
measure: Percentage of patients who switch to self-administration without the presence of a nurse
timeFrame: Up to 8 weeks
measure: Patient characteristics
timeFrame: At inclusion
measure: Degree of autonomy
timeFrame: At inclusion and 12 months after self-administration
measure: The time frame for autonomy
timeFrame: Up to 12 months
measure: Evolution of patients Quality of Life with Saint George's Respiratory Questionnaire (SGRQ)
timeFrame: Up to 12 months
measure: Patients satisfaction for treatment administration by a nurse and for self-administration
timeFrame: Up to 12 months
measure: Satisfaction about learning program
timeFrame: Up to 8 weeks
measure: Tolerance and safety
timeFrame: Up to 12 months
measure: Compliance/Observance
timeFrame: Up to 8 weeks
measure: Determining factors for switch to self-administration
timeFrame: Up to 8 weeks
Other outcomes:
Locations:
city: Angers
country: France
facility: CHU Angers
city: Bordeaux
country: France
facility: CHU Bordeaux - Hôpital Haut-Lévèque
city: Grenoble
country: France
facility: CHU Grenoble-Alpes
city: Lille
country: France
facility: CHU Lille
city: Lyon
country: France
facility: Hospices Civils de Lyon
city: Marseille
country: France
facility: Hôpital Saint Joseph
city: Paris
country: France
facility: Hôpital Bichat-Claude-Bernard
city: Rennes
country: France
facility: CHU Rennes - Hôpital Pontchaillou
city: Strasbourg
country: France
facility: CHU Strasbourg
city: Tours
country: France
facility: CHRU Tours
city: Vandœuvre-lès-Nancy
country: France
facility: CHU Nancy Brabois
Study officials:
affiliation: CSL Behring SA
name: Study Physician
role: STUDY_DIRECTOR
References: