Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - Other
Status
Ended
Participants planned
48
Countries
2
Sites
2
Activated Phosphoinositide 3-Kinase Delta Syndrome
Key facts
- Sponsor
- Pharming Technologies B.V.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 26 Jan 2024 → 9 Nov 2025
- Decision date (initial)
- 2024-01-10
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Activated Phosphoinositide 3-Kinase Delta Syndrome
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10078281 | Activated PI3 kinase delta syndrome | 100000004850 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Pharming Technologies B.V.
- Sponsor organisation
- Pharming Technologies B.V.
- Address
- Darwinweg 24
- City
- Leiden
- Postcode
- 2333 CR
- Country
- Netherlands
Scientific contact point
- Organisation
- Pharming Technologies B.V.
- Contact name
- Clinical Department
Public contact point
- Organisation
- Pharming Technologies B.V.
- Contact name
- Clinical Department
Locations
2 EU/EEA countries · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ended | 32 | 1 |
| Hungary | Ended | 16 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
CRS Clinical Research Services Management GmbH
Clinical Pharmacology Unit, Lornsenstrasse 7, Brunswik, Kiel
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 |
|---|---|---|---|---|---|
| Germany | 2024-01-26 | 2025-11-09 | 2024-01-31 | 2024-05-29 | |
| Hungary | 2025-03-31 | 2025-11-09 | 2025-04-10 | 2025-11-04 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-02 | Germany | Acceptable 2024-01-10
|
2024-01-10 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-12-19 | Germany | Acceptable | 2024-12-30 |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2024-12-20 | 2025-03-10 | ||
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-04-10 | Germany | 2025-04-10 | |
| 5 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-04-25 | Germany | Acceptable 2025-07-02
|
2025-07-04 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-11-06 | Germany | Acceptable 2025-07-02
|
2025-11-06 |