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

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

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 Overview

• Status: Active, not recruiting
• Conditions: Alpha-1 Antitrypsin Deficiency

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

Study Locations

• France
  • Angers, France
    • CHU Angers
  • Bordeaux, France
    • CHU Bordeaux - Hopital Haut-Leveque
  • Grenoble, France
    • CHU Grenoble-Alpes
  • Lille, France
    • CHU Lille
  • Lyon, France
    • Hospices Civils de Lyon
  • Marseille, France
    • Hôpital Saint Joseph
  • Paris, France
    • Hôpital Bichat-Claude-Bernard
  • Rennes, France
    • CHU Rennes - Hôpital Pontchaillou
  • Strasbourg, France
    • CHU Strasbourg
  • Tours, France
    • Chru Tours
  • Vandœuvre-lès-Nancy, France
    • CHU Nancy Brabois

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Physician, CSL Behring SA

Study record dates

Study Major Dates

• Study Start (Actual): October 18, 2019
• Primary Completion (Estimated): March 31, 2025
• Study Completion (Estimated): March 31, 2025

Study Registration Dates

• First Submitted: February 4, 2020
• First Submitted That Met QC Criteria: February 6, 2020
• First Posted (Actual): February 10, 2020

Study Record Updates

• Last Update Posted (Actual): April 19, 2024
• Last Update Submitted That Met QC Criteria: April 18, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Liver Diseases; Genetic Diseases, Inborn; Subcutaneous Emphysema; Emphysema; Alpha 1-Antitrypsin Deficiency
• Other Study ID Numbers: AmAREtTI; 2018-A02601-54 (Other Identifier: ANSM)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No