Safety and clinical parameter assessment of a 12-week home self-infusion therapy with Prolastin® in patients with severe alpha-1-antitrypsin (AATD) deficiency. The “Sunshine Study”

Conditions

Severe Alpha-1 Antitrypsin Deficiency (AATD)

Brief summary

Occurrence of AEs during the training and/or main study period

Detailed description

SGRQ-C scores (Symptoms, Activity and Impacts scores) at baseline visit (V0) and end-of-study visit (EOS) as well as change between V0 and EOS, CAT total score at V0, at VT5 (if applicable), VM1, VM5, VM9, and EOS as well as changes to V0, COPD Assessment Test (CAT) total score at V0 and in main study period (VM1, VM5, VM9), and at EOS as well as changes to V0, Lung function parameters (Forced Expiratory Volume in the First Second [FEV1], Forced Vital Capacity [FVC], Diffusing Capacity of the Lungs for Carbon Monoxide [DLCO], Residual Volume [RV], Total Lung Capacity [TLC]) at V0 and EOS as well as change between V0 and EOS, Global Initiative for Chronic Obstructive Lung Disease (GOLD) grade for severity of airflow obstruction (GOLD A, B, E) at V0 and EOS as well as change between V0 and EOS, Patient satisfaction at each visit, Ease of use at each visit

Related papers

No related papers found yet.

Interventions

DRUGProlastin 1000 mg

DRUGPulver und Lösungsmittel zur Her- stellung einer Infusionslösung

DRUGProlastin 4000 mg

DRUGPulver und Lösungsmittel zur Herstellung einer Infusionslösung

DRUGProlastin 5000 mg

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Occurrence of AEs during the training and/or main study period	—

Secondary

Measure	Time frame
SGRQ-C scores (Symptoms, Activity and Impacts scores) at baseline visit (V0) and end-of-study visit (EOS) as well as change between V0 and EOS, CAT total score at V0, at VT5 (if applicable), VM1, VM5, VM9, and EOS as well as changes to V0, COPD Assessment Test (CAT) total score at V0 and in main study period (VM1, VM5, VM9), and at EOS as well as changes to V0, Lung function parameters (Forced Expiratory Volume in the First Second [FEV1], Forced Vital Capacity [FVC], Diffusing Capacity of the Lungs for Carbon Monoxide [DLCO], Residual Volume [RV], Total Lung Capacity [TLC]) at V0 and EOS as well as change between V0 and EOS, Global Initiative for Chronic Obstructive Lung Disease (GOLD) grade for severity of airflow obstruction (GOLD A, B, E) at V0 and EOS as well as change between V0 and EOS, Patient satisfaction at each visit, Ease of use at each visit	—

Measure

Time frame

SGRQ-C scores (Symptoms, Activity and Impacts scores) at baseline visit (V0) and end-of-study visit (EOS) as well as change between V0 and EOS, CAT total score at V0, at VT5 (if applicable), VM1, VM5, VM9, and EOS as well as changes to V0, COPD Assessment Test (CAT) total score at V0 and in main study period (VM1, VM5, VM9), and at EOS as well as changes to V0, Lung function parameters (Forced Expiratory Volume in the First Second [FEV1], Forced Vital Capacity [FVC], Diffusing Capacity of the Lungs for Carbon Monoxide [DLCO], Residual Volume [RV], Total Lung Capacity [TLC]) at V0 and EOS as well as change between V0 and EOS, Global Initiative for Chronic Obstructive Lung Disease (GOLD) grade for severity of airflow obstruction (GOLD A, B, E) at V0 and EOS as well as change between V0 and EOS, Patient satisfaction at each visit, Ease of use at each visit

—

Countries

Germany

Outcome results

None listed