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

NCT04262284 | Completed

• 2 other identifiers: AmAREtTI2018-A02601-54
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 11

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.

Conditions

Alpha-1 Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (10)

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

  Up to 8 weeks

• Patient characteristics

  At inclusion

• Degree of autonomy

  At inclusion and 12 months after self-administration

• The time frame for autonomy

  Up to 12 months

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

  Up to 12 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• Adult patients (age ≥18 years old).
• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (11)

CHU Angers

Angers, France

Location

CHU Bordeaux - Hôpital Haut-Lévèque

Bordeaux, France

Location

CHU Grenoble-Alpes

Grenoble, France

Location

CHU Lille

Lille, France

Location

Hospices Civils de Lyon

Lyon, France

Location

Hôpital Saint Joseph

Marseille, France

Location

Hôpital Bichat-Claude-Bernard

Paris, France

Location

CHU Rennes - Hôpital Pontchaillou

Rennes, France

Location

CHU Strasbourg

Strasbourg, France

Location

CHRU Tours

Tours, France

Location

CHU Nancy Brabois

Vandœuvre-lès-Nancy, France

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Study Physician

  CSL Behring SA

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2020
• First Posted: February 10, 2020
• Study Start: October 18, 2019
• Primary Completion: May 6, 2024
• Study Completion: May 6, 2024
• Last Updated: May 28, 2025

Record last verified: 2025-05

Locations

FR (11)