Human alpha1-proteinase inhibitor for Severe Alpha-1 antitrypsin deficiency (AATD)

Inclusion criteria

• {"criterion_text":"- Signed written informed consent obtained prior to any study-related procedure\n- At least 18 years of age\n- Able to understand and read German at a level sufficient to comprehend patient documents, including the informed consent, instruction materials and questionnaires. Note: Assistance for any required writing tasks can be provided by the caregiver.\n- A documented diagnosis of severe Alpha-1 Antitrypsin Deficiency\n- Treatment with Prolastin® for at least 3 months with documented good treatment tolerability\n- Ability and willingness to learn self-infusion and perform infusions at home or being accompanied by a third person (i.e. caregiver), who has the ability and willingness to learn and perform infusions without HCP supervision (after appropriate training). Note: For patients requiring a caregiver, the availability of a suitable caregiver must be ensured in order to be eligible for inclusion. For patients who perform the self-infusion themselves, the availability of an emergency contact, i.e. a person who is willing and able to be present and monitor the patient during every self-infusion throughout the main study period, must be ensured in order to be eligible for inclusion.\n- Having access to and being able to use (incl. reading and typing) a functional electronic device that enables phone calls and video calls (for remote calls from the study site or home-care study nurses) and access to an internet browser (for completion of the questionnaires)\n- Suitable veins for venepuncture, according to the investigator’s discretion\n- Life expectancy of over 3 years"}

Exclusion criteria

• {"criterion_text":"- Contraindications as listed in the current SmPC: a) Individuals with selective immunoglobulin A (IgA) deficiency b) Known hypersensitivity against human Alpha-1 Proteinase Inhibitor or to any of the other ingredients in the formulation or component of the container\n- Occurrence of serious adverse drug reactions due to Prolastin® infusion in the last 3 months prior to V0\n- Patients who have undergone lung transplantation\n- Current smokers\n- Patients with venous access via port\n- Participation in another clinical study with investigational medicinal product within the last 3 months prior to baseline visit or simultaneous participation in another clinical study\n- Patients with psychiatric illnesses, imprisoned persons, persons admitted to nursing homes, persons under legal guardianship and persons not able to express their consent (e.g. due to mental impairment)\n- Breastfeeding or pregnant women"}

Primary endpoints

• {"endpoint_text":"- Occurrence of AEs during the training and/or main study period","definition_or_measurement_approach":"Recording and reporting of adverse events (AEs) occurring during the training period and main study period (i.e. collection of AEs throughout training and main study visits)."}

Secondary endpoints

• {"endpoint_text":"- 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\n- CAT total score at V0, at VT5 (if applicable), VM1, VM5, VM9, and EOS as well as changes to V0\n- 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\n- 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\n- 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\n- Patient satisfaction at each visit\n- Ease of use at each visit","definition_or_measurement_approach":"SGRQ-C and CAT: patient-reported questionnaires administered at specified visits (baseline V0 and end-of-study EOS and intermediate visits as listed) with change from baseline analysed. Lung function parameters: spirometry and DLCO assessments at V0 and EOS with change from baseline. GOLD grade: classification at V0 and EOS. Patient satisfaction and ease of use: assessed at each visit (questionnaires/scales as per protocol)."}

Germany

Earliest CTIS Part Ii Submission Date

19-02-2025

Latest Decision Or Authorization Date

26-02-2026

Processing Time Days

372

Number Of Sites

8

Number Of Participants

33

Primary sponsor

Full Name

Grifols S.A.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Spain

Third parties

• {"country":"Germany","full_name":"Emovis GmbH","duties_or_roles":"Provider of home-care study nurses","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Germany","full_name":"GKM Gesellschaft fuer Therapieforschung mbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}