A two-center, randomized, double-blind, placebo-controlled study of intravenous plasma-purified alpha-1 antitrypsin for hospitalized patients with COPD exacerbations (AECOPD study)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fondazione IRCCS Policlinico San Matteo

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

16 Oct 2025 → ongoing

Decision date (initial)

2025-03-04

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

to evaluate the safety and efficacy (from a biological perspective) of a single administration of IV Prolastin as an anti-inflammatory treatment for patients admitted to hospital because of a COPD exacerbation leading to an acute or an acute on chronic respiratory failure, by reducing circulating levels of inflammatory markers

Secondary objectives 3

1. to determine the anti-inflammatory and immunomodulatory effects of IV Prolastin administered once at 120 mg per kilogram of body weight on plasma concentration of other biomarkers which have been implicated in pulmonary and systemic inflammation, and also to be suppressed by AAT in vivo
2. to identify treatment failure as assessed by: a) need for either NIV or CPAP b) need of ETI c) need of transfer to ICU d) in-hospital death after randomization
3. to evaluate the impact of AECOPD on overall health, daily life, and perceived well-being in patients with obstructive airways disease at discharge

Conditions and MedDRA coding

Chronic Obstructive Pulmonary Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Informed Consent as documented by signature
2. Male and female ≥18 years old
3. Previous COPD diagnosis with a documented post-bronchodilator FEV1 to FVC ratio (FEV1/FVC) equal to or less than 0.70 or LLN
4. Hospitalized for a moderate to severe exacerbation, according to the Rome proposal
5. Admission to the respiratory ward by ≤24 hours
6. Acute or acute on chronic respiratory failure with SpO2 <92% at room air, or PaO2/FiO2 (or SpO2/FiO2) <300)
7. A positive sputum NEATstik®, that corresponds to an approximate neutrophil elastase concentration of 8 μg·mL−1 (rapid point-of-care test)

Exclusion criteria 12

1. Clinically important pulmonary disease other than COPD (e.g., clinically significant bronchiectasis, pulmonary fibrosis, cystic fibrosis, hypoventilation syndrome associated with obesity, lung cancer, and primary ciliary dyskinesia)
2. Presence of pneumonia or other pleuroparenchymal abnormalities on either chest X-ray or Chest CT scan, performed per routine clinical practice at the hospital admission
3. Current diagnosis of asthma according to the GINA, prior history of asthma, or asthma-COPD overlap
4. Known AATD as homozygous or composite heterozygous mutation of the AAT gene (patients will be screened for AATD but genetic testing will not be available at enrolment)
5. Presence of any active malignancy (other than non-melanoma skin cancer)
6. Any unstable disorder, including, but not limited to, cardiovascular, gastrointestinal, hepatic, renal, neurological, musculoskeletal, infectious, endocrine, metabolic, haematological, psychiatric disorder, major physical and/or cognitive impairment that, in the opinion of the Investigator, could: (a) Affect the safety of the participant throughout the study (b) Influence the findings of the study or their interpretation
7. Known diagnosis of selective IgA deficiency defined as a serum IgA of less than 7 mg/dl (0.07 g/L)
8. Patient with the immediate need for ETI of NIV (patients already on CPAP or NIV can be included)
9. Contraindications to the class of drugs under study, e.g. known hypersensitivity or allergy to class of drugs or the investigational product
10. Women who are pregnant or breastfeeding
11. Participants that have previously received Prolastin® 1000 mg/40 ml
12. Participation in another interventional clinical trial with investigational drugs within the 30 days preceding and during the present study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in plasma concentration of IL-6 at 7 days after randomization

Secondary endpoints 3

1. Change in plasma concentration of IL-1b, IL-5, IL-8, IL-10, and soluble TNF receptor 1(sTNFR1), CRP at 7 days after randomization
2. Treatment failure (need for either NIV or CPAP or ETI or transfer to ICU or in-hospital death after randomization)
3. Differences in SGRQ score at discharge

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondazione IRCCS Policlinico San Matteo

Sponsor organisation

Fondazione IRCCS Policlinico San Matteo

Address

Viale Camillo Golgi 19

City

Pavia

Postcode

27100

Country

Italy

Scientific contact point

Organisation

Fondazione IRCCS Policlinico San Matteo

Contact name

Angelo Guido Corsico

Public contact point

Organisation

Fondazione IRCCS Policlinico San Matteo

Contact name

Angelo Guido Corsico

Locations

1 EU/EEA country · 2 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 9 files

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

Application history

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