Safety and clinical parameter assessment of self-infusion therapy with Prolastin® in patients with severe AATD

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Grifols S.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

1 Jul 2025 → ongoing

Decision date (initial)

2025-03-05

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Grifols S.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy, Others

To assess the safety of self-infusion therapy with Prolastin® in patients with severe AATD

Secondary objectives 2

1. To evaluate QOL, COPD symptoms and clinical health of patients with severe AATD treated with Prolastin® self-infusion therapy
2. To evaluate ease of use and patient satisfaction regarding Prolastin® self-infusion therapy

Conditions and MedDRA coding

Severe Alpha-1 Antitrypsin Deficiency (AATD)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Signed written informed consent obtained prior to any study-related procedure
2. At least 18 years of age
3. 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.
4. A documented diagnosis of severe Alpha-1 Antitrypsin Deficiency
5. Treatment with Prolastin® for at least 3 months with documented good treatment tolerability
6. 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.
7. 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)
8. Suitable veins for venepuncture, according to the investigator’s discretion
9. Life expectancy of over 3 years

Exclusion criteria 8

1. 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
2. Occurrence of serious adverse drug reactions due to Prolastin® infusion in the last 3 months prior to V0
3. Patients who have undergone lung transplantation
4. Current smokers
5. Patients with venous access via port
6. 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
7. 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)
8. Breastfeeding or pregnant women

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

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

Secondary endpoints 7

1. 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
2. CAT total score at V0, at VT5 (if applicable), VM1, VM5, VM9, and EOS as well as changes to V0
3. 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
4. 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
5. 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
6. Patient satisfaction at each visit
7. Ease of use at each visit

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Grifols S.A.

Sponsor organisation

Grifols S.A.

Address

Avinguda De La Generalitat 152-158

City

Sant Cugat Del Valles

Postcode

08174

Country

Spain

Scientific contact point

Organisation

Grifols S.A.

Contact name

Grifols Deutschland GmbH

Public contact point

Organisation

Grifols S.A.

Contact name

Grifols Deutschland GmbH

Third parties 2

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 17 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications