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

Study Overview
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Brief Summary
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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.

Official Title
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Respreeza® Self-administration and Learning Program (AmAREtTI Study- Auto-Administration de Respreeza® et Programme d'apprenTIssage)

Conditions
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Alpha-1 Antitrypsin Deficiency

Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Up to 8 weeks | 
| Percentage of patients who switch to self-administration without the presence of a nurse |  | Up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients who switch to self-administration without the presence of a nurse | 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 | 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) | 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 | 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. | Up to 12 months | 
| Evolution of patients Quality of Life with Saint George's Respiratory Questionnaire (SGRQ) |  | 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 | Up to 12 months | 
| Satisfaction about learning program | Satisfaction about learning program will be assessed at the end of the learning program by VAS | 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). | Up to 12 months | 
| Compliance/Observance | Compliance/observance measured by the number of scheduled and unrealized sessions. | 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). | Up to 8 weeks |