Alpha-1 Antitrypsin DeficiencyAmAREtTINCT04262284
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
An observational study of Alpha-1 Antitrypsin Deficiency, run by CSL Behring at 11 sites in France.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha-1 Antitrypsin Deficiency
- How many people
- 15 participants
- Where
- 11 sitesFrance
- Study dates
- 18 Oct 2019to 6 May 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
France11 sites
CHU Angers
Angers
CHU Bordeaux - Hôpital Haut-Lévèque
Bordeaux
CHU Grenoble-Alpes
Grenoble
CHU Lille
Lille
Hospices Civils de Lyon
Lyon
Hôpital Saint Joseph
Marseille
Hôpital Bichat-Claude-Bernard
Paris
CHU Rennes - Hôpital Pontchaillou
Rennes
CHU Strasbourg
Strasbourg
CHRU Tours
Tours
CHU Nancy Brabois
Vandœuvre-lès-Nancy
About this study
Official title: 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.
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 8 weeks
Percentage of patients who switch to self-administration without the presence of a nurse
Measured: Up to 12 months
Secondary outcomes (10)
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
Measured: 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)
Measured: 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
Measured: 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.
Measured: Up to 12 months
Evolution of patients Quality of Life with Saint George's Respiratory Questionnaire (SGRQ)
Measured: 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
Measured: Up to 12 months
Satisfaction about learning program
Satisfaction about learning program will be assessed at the end of the learning program by VAS
Measured: 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).
Measured: Up to 12 months
Compliance/Observance
Compliance/observance measured by the number of scheduled and unrealized sessions.
Measured: 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).
Measured: Up to 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2020
- Study start18 Oct 2019
- Primary completion6 May 2024
- Study completion6 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Physician, Study director, CSL Behring SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04262284
- Sponsor's protocol ID
- AmAREtTI
- Other IDs
- 2018-A02601-54
- Acronym
- AmAREtTI
- Status last verified
- May 2025
- First submitted
- 4 Feb 2020
- Last updated
- 28 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Respreeza® Self-administration and Learning Program (AmAREtTI Study- Auto-Administration de Respreeza® et Programme d'apprenTIssage)
- ClinicalTrials.gov ID
- NCT04262284
- Acronym / sponsor ID
- AmAREtTI / AmAREtTI
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 18 Oct 2019 (actual)
- Primary completion
- 6 May 2024 (actual)
- Study completion
- 6 May 2024 (actual)
- First posted
- 10 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
10 secondary outcomes
Percentage of patients who switch to self-administration without the presence of a nurse
Time frame: Up to 8 weeks
Patient characteristics
Time frame: At inclusion
Degree of autonomy
Time frame: At inclusion and 12 months after self-administration
The time frame for autonomy
Time frame: Up to 12 months
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 about learning program
Time frame: Up to 8 weeks
Tolerance and safety
Time frame: Up to 12 months
Compliance/Observance
Time frame: Up to 8 weeks
Determining factors for switch to self-administration
Time frame: Up to 8 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Physician study director
CSL Behring SA
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha-1 Antitrypsin Deficiency, run by CSL Behring at 11 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2025.