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

Alpha-1 Antitrypsin Deficiency Clinical Trial

— AmAREtTI  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04262284
• Other study ID #: AmAREtTI
• Secondary ID: 2018-A02601-54
• Status: Active, not recruiting
• Start date: October 18, 2019
• Est. completion date: March 31, 2025

Study information

• Verified date: April 2024
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 60
• Est. completion date: March 31, 2025
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency
• Alpha-1 Antitrypsin Deficiency

Locations

Country	Name	City	State
France	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	CHU Nancy Brabois	Vandœuvre-lès-Nancy

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

France, 

Outcome

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