Not provided
Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2018-A02601-54 | Other Identifier | ANSM |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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:
| Up to 8 weeks |
| Percentage of patients who switch to self-administration without the presence of a nurse | Up to 12 months |
| 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:
|
Not provided
Inclusion Criteria:
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.
Not provided
Not provided
Not provided
Patients suffering from alpha-1 antitrypsin deficiency (AATD) treated by Respreeza®
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Physician | CSL Behring SA | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU Angers | Angers | France | ||||
| CHU Bordeaux - Hôpital Haut-Lévèque |
Not provided
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 |
| 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 | France |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |