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

• ClinicalTrials.gov Identifier: NCT04262284
• Recruitment Status: Recruiting
• First Posted: February 10, 2020
• Last Update Posted: August 2, 2022
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

Tracking Information

• First Submitted Date: February 4, 2020
• First Posted Date: February 10, 2020
• Last Update Posted Date: August 2, 2022
• Actual Study Start Date: October 18, 2019
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 6, 2020)

• Percentage of patients who switch to self-administration without the presence of a nurse at the end of the learning program [ Time Frame: Up to 8 weeks ]
  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
• Percentage of patients who switch to self-administration without the presence of a nurse [ Time Frame: Up to 12 months ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: February 6, 2020)

• Percentage of patients who switch to self-administration without the presence of a nurse [ Time Frame: Up to 8 weeks ]
  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
• Patient characteristics [ Time Frame: At inclusion ]
  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)
• Degree of autonomy [ Time Frame: At inclusion and 12 months after self-administration ]
  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
• The time frame for autonomy [ Time Frame: Up to 12 months ]
  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.
• 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 [ Time Frame: Up to 12 months ]
  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
• Satisfaction about learning program [ Time Frame: Up to 8 weeks ]
  Satisfaction about learning program will be assessed at the end of the learning program by VAS
• Tolerance and safety [ Time Frame: Up to 12 months ]
  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).
• Compliance/Observance [ Time Frame: Up to 8 weeks ]
  Compliance/observance measured by the number of scheduled and unrealized sessions.
• Determining factors for switch to self-administration [ Time Frame: Up to 8 weeks ]
  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).

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

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

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.

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Probability Sample
• Study Population: Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®
• Condition: Alpha-1 Antitrypsin Deficiency
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: February 6, 2020): 60
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2023
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Clinical Trials Registration Coordinator; 610-878-4000; clinicaltrials@cslbehring.com

• Listed Location Countries: France

Administrative Information

• NCT Number: NCT04262284
• Other Study ID Numbers: AmAREtTI; 2018-A02601-54 ( Other Identifier: ANSM )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: CSL Behring
• Original Responsible Party: Same as current
• Current Study Sponsor: CSL Behring
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Study Physician; CSL Behring SA

• PRS Account: CSL Behring
• Verification Date: August 2022