Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Authorised, recruitment pending
Participants planned
45
Countries
6
Sites
10
Alpha-1 Antitrypsin Deficiency
Key facts
- Sponsor
- Airna Corp.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Respiratory Tract Diseases [C08], Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Digestive System Diseases [C06]
- Decision date (initial)
- 2026-04-21
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2025-523558-14-00
- WHO UTN
- U1111-1330-1003
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Alpha-1 Antitrypsin Deficiency
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 28.0 | LLT | 10001806 | Alpha-1 anti-trypsin deficiency | 10010331 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Airna Corp.
- Sponsor organisation
- Airna Corp.
- Address
- 1 Kendall Square Building 300
- City
- Cambridge
- Postcode
- 02139-1562
- Country
- United States
Scientific contact point
- Organisation
- Airna Corp.
- Contact name
- Kristina Storey
Public contact point
- Organisation
- Airna Corp.
- Contact name
- AiRNA Corporation
Locations
6 EU/EEA countries · 10 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Authorised, recruitment pending | 6 | 4 |
| Ireland | Authorised, recruitment pending | 1 | 1 |
| Netherlands | Authorised, recruitment pending | 2 | 1 |
| Portugal | Authorised, recruitment pending | 4 | 1 |
| Spain | Authorised, recruitment pending | 4 | 2 |
| Sweden | Authorised, recruitment pending | 2 | 1 |
| Rest of world
Georgia, United Kingdom, Australia, New Zealand
|
— | 26 | — |
Investigational sites
Universitaet Des Saarlandes
Int. Medicine and Pneumology, Kirrberger Strasse 100, 66421, Homburg
Medizinische Hochschule Hannover
Pneumology, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Universitaetsklinikum Tuebingen AöR
Medicine, Otfried-Mueller-Strasse 10, Nordstadt, Tuebingen
Universitaetsklinikum Aachen AöR
Medizinische Klinik Ill, Pauwelsstrasse 30, 52074, Aachen
Leids Universitair Medisch Centrum (LUMC)
Department of Pulmonology, Albinusdreef 2, 2333 ZA, Leiden
Unidade Local De Saude De Matosinhos E.P.E.
Pneumology, Rua Doutor Eduardo Torres, 4464-513, Senhora Da Hora
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-12-17 | Sweden | Acceptable 2026-04-16
|
2026-04-17 |