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

CSL Behring15 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Alpha-1 Antitrypsin Deficiency

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

CHU Angers

Angers, France

CHU Bordeaux - Hôpital Haut-Lévèque

Bordeaux, France

CHU Grenoble-Alpes

Grenoble, France

CHU Lille

Lille, France

Hospices Civils de Lyon

Lyon, France

Hôpital Saint Joseph

Marseille, France

Hôpital Bichat-Claude-Bernard

Paris, France

CHU Rennes - Hôpital Pontchaillou

Rennes, France

CHU Strasbourg

Strasbourg, France

CHRU Tours

Tours, France

...and 1 more locations

Outcomes

Primary Outcomes

Percentage of patients who switch to self-administration without the presence of a nurse at the end of the learning program

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

Time frame: Up to 8 weeks

Percentage of patients who switch to self-administration without the presence of a nurse

Time frame: Up to 12 months

Secondary Outcomes

Percentage of patients who switch to self-administration without the presence of a nurse

Time frame: Up to 8 weeks

Patient characteristics

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)

Time frame: At inclusion

Degree of autonomy

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

Time frame: At inclusion and 12 months after self-administration

The time frame for autonomy

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.

Time frame: Up to 12 months

Evolution of patients Quality of Life with Saint George's Respiratory Questionnaire (SGRQ)

Time frame: Up to 12 months

Patients satisfaction for treatment administration by a nurse and for self-administration

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

Time frame: Up to 12 months

Satisfaction about learning program

Satisfaction about learning program will be assessed at the end of the learning program by VAS

Time frame: Up to 8 weeks

Tolerance and safety

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).

Time frame: Up to 12 months

Compliance/Observance

Compliance/observance measured by the number of scheduled and unrealized sessions.

Time frame: Up to 8 weeks

Determining factors for switch to self-administration

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).

Time frame: Up to 8 weeks

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

Overview

Conditions

Eligibility

Locations (11)

Outcomes

Primary Outcomes

Secondary Outcomes