Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - Other
Status
Ended
Participants planned
24
Countries
1
Sites
1
Glycosphingolipid lysosomal storage disorders
Key facts
- Sponsor
- Azafaros B.V.
- Participant type
- Healthy volunteers
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Trial duration
- 12 Mar 2024 → 6 Jan 2025
- Decision date (initial)
- 2024-02-27
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-510058-17-00
- WHO UTN
- U1111-1301-2485
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Glycosphingolipid lysosomal storage disorders
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 26.1 | HLT | 10024579 | Lysosomal storage disorders | 10027433 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Azafaros B.V.
- Sponsor organisation
- Azafaros B.V.
- Address
- J.H. Oortweg 21
- City
- Leiden
- Postcode
- 2333 CH
- Country
- Netherlands
Scientific contact point
- Organisation
- Azafaros B.V.
- Contact name
- Christian Freitag
Public contact point
- Organisation
- Azafaros B.V.
- Contact name
- Christian Freitag
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ended | 24 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Pharmaceutical Research Associates Group B.V.
Early Development Service, Van Swietenlaan 6, 9728 NZ, Groningen
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Netherlands | 2024-03-12 | 2025-01-06 | 2024-04-17 | 2024-12-10 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-01-08 | Netherlands | Acceptable 2024-02-27
|
2024-02-27 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-08-12 | Netherlands | Acceptable 2024-02-27
|
2024-08-12 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-09-10 | Netherlands | Acceptable 2024-09-18
|
2024-09-18 |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-12-17 | Netherlands | Acceptable 2025-01-13
|
2025-01-13 |