Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - First administration to humans
Status
Ongoing, recruitment ended
Participants planned
36
Countries
4
Sites
4
Prion diseases
Key facts
- Sponsor
- Ionis Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 24 Jan 2024 → ongoing
- Decision date (initial)
- 2024-04-15
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-503355-98-00
- WHO UTN
- U1111-1289-3291
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Prion diseases
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10070343 | Prion agent test positive | 100000004848 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ionis Pharmaceuticals Inc.
- Sponsor organisation
- Ionis Pharmaceuticals Inc.
- Address
- 2855 Gazelle Court
- City
- Carlsbad
- Postcode
- 92010-6670
- Country
- United States
Scientific contact point
- Organisation
- Ionis Pharmaceuticals Inc.
- Contact name
- Global Regulatory Affairs
Public contact point
- Organisation
- Ionis Pharmaceuticals Inc.
- Contact name
- Global Regulatory Affairs
Locations
4 EU/EEA countries · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruitment ended | 2 | 1 |
| Germany | Ongoing, recruitment ended | 2 | 1 |
| Italy | Ongoing, recruitment ended | 2 | 1 |
| Spain | Ongoing, recruitment ended | 2 | 1 |
| Rest of world
Canada, United States, Australia, Israel, United Kingdom, Japan
|
— | 28 | — |
Investigational sites
Assistance Publique Hopitaux De Paris
Neurology, Num Voie 47 A 83, 47 Boulevard De L Hopital, Paris
Universitaetsmedizin Goettingen
Neurology, Robert-Koch-Strasse 40, Weende, Goettingen
IRCCS Foundation Istituto Neurologico Carlo Besta
Department of Neuroimmunology and Neuromuscular Diseases, Neurologia IV, Via Giovanni Celoria 11, 20133, Milan
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 |
|---|---|---|---|---|---|
| France | 2024-07-11 | 2024-10-14 | 2024-10-21 | ||
| Germany | 2024-01-30 | 2024-02-07 | 2024-12-05 | ||
| Italy | 2024-07-25 | 2024-09-24 | 2024-10-28 | ||
| Spain | 2024-01-24 | 2024-02-13 | 2024-12-02 |
Application history
7 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-06-21 | Italy | Acceptable 2023-10-16
|
2023-10-19 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2023-11-24 | Italy | Acceptable 2023-10-16
|
2023-11-24 |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2024-01-18 | Acceptable 2023-10-16
|
2024-04-15 | |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-04-26 | Italy | Acceptable 2023-10-16
|
2024-04-26 |
| 5 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-07-12 | Italy | Acceptable 2024-09-30
|
2024-09-30 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-06-09 | Italy | Acceptable 2025-09-15
|
2025-09-15 |
| 7 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-12-19 | Italy | Acceptable 2026-04-14
|
2026-04-14 |