Respreeza® Self-administration and Learning Program (AmAREtTI Study)

Active, not recruiting

• Conditions: Alpha-1 Antitrypsin Deficiency

• Registration Number: NCT04262284
• Lead Sponsor: CSL Behring

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-10-18
• Study First Submitted: 2020-02-04
• Study First Submitted QC: 2020-02-06
• Study First Posted: 2020-02-10
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-18
• Last Update Posted: 2024-04-19
• Primary Completion: 2025-03-31
• Study Completion: 2025-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 60

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.

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percentage of patients who switch to self-administration without the presence of a nurse	Up to 12 months
Percentage of patients who switch to self-administration without the presence of a nurse at the end of the learning program	Up to 8 weeks	A patient will be permitted to switch to self-administration without the presence of a nurse if the following conditions are met: * Three consecutive successful self-administrations by the patient in the presence of the nurse but without assistance during the learning phase.; * Validation of the learning grid The percentage is based on number of patient switching to self-administration and remains in self-administration 12 months after

Secondary Outcome Measures

Name	Time	Method
Tolerance and safety	Up to 12 months	General and local tolerance at the administration site assessed by the nature and number of adverse events (AEs) and immunoglobulin E impaired gas exchange (IGEs).
The time frame for autonomy	Up to 12 months	The time to achieve autonomy will be defined by the time between the first learning session for self-administration and the first self-administration without nurse.
Percentage of patients who switch to self-administration without the presence of a nurse	Up to 8 weeks	A patient will be permitted to switch to self-administration without the presence of a nurse if the following conditions are met: * Three consecutive successful self-administrations by the patient in the presence of the nurse but without assistance during the learning phase.; * Validation of the learning grid The percentage is based on number of patient switching to self-administration
Evolution of patients Quality of Life with Saint George's Respiratory Questionnaire (SGRQ)	Up to 12 months
Patient characteristics	At inclusion	Socio-demographic data, disease history, concomitant treatments, substitution protocol, percentage of theoretical forced expiratory volume at one second (FEV1), diffusing capacity of lung for carbon monoxide (DLCO), distance from home to hospital, type of follow-up (in hospital, in surgery)
Degree of autonomy	At inclusion and 12 months after self-administration	The change in the degree of autonomy will be defined as the difference between the autonomy assessed at 12 months and inclusion according to an EVA
Patients satisfaction for treatment administration by a nurse and for self-administration	Up to 12 months	Satisfaction for treatment administration by a nurse will be assessed at inclusion by visual analog scale (VAS) Satisfaction for self-administration will be assessed after 12 months after the 1st self-administration or in case of study withdrawal by VAS
Satisfaction about learning program	Up to 8 weeks	Satisfaction about learning program will be assessed at the end of the learning program by VAS
Compliance/Observance	Up to 8 weeks	Compliance/observance measured by the number of scheduled and unrealized sessions.
Determining factors for switch to self-administration	Up to 8 weeks	Determining factors that may be associated with the transition to self-administration (demographics, disease history, concomitant treatments, substitution protocol, FEV1, QoL at inclusion, duration of learning program).

Trial Locations

Locations (11)

CHU Lille; 🇫🇷 Lille, France

Hospices Civils de Lyon; 🇫🇷 Lyon, France

CHU Angers; 🇫🇷 Angers, France

CHU Bordeaux - Hôpital Haut-Lévèque; 🇫🇷 Bordeaux, France

CHU Rennes - Hôpital Pontchaillou; 🇫🇷 Rennes, France

CHU Grenoble-Alpes; 🇫🇷 Grenoble, France

Hôpital Saint Joseph; 🇫🇷 Marseille, France

Hôpital Bichat-Claude-Bernard; 🇫🇷 Paris, France

CHRU Tours; 🇫🇷 Tours, France

CHU Strasbourg; 🇫🇷 Strasbourg, France

CHU Nancy Brabois; 🇫🇷 Vandœuvre-lès-Nancy, France