Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Ended
Participants planned
30
Countries
1
Sites
2
Alzheimer’s Disease in Individuals with Down Syndrome
Key facts
- Sponsor
- Ionis Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 12 Feb 2025 → 21 Aug 2025
- Decision date (initial)
- 2024-10-10
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-509257-31-00
- WHO UTN
- U1111-1306-9498
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Alzheimer’s Disease in Individuals with Down Syndrome
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10013616 | Down's syndrome | 10010331 |
| 20.0 | LLT | 10001896 | Alzheimer's disease | 10029205 |
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 at Ionis Pharmaceuticals, Inc.
Public contact point
- Organisation
- Ionis Pharmaceuticals Inc.
- Contact name
- Global Regulatory Affairs at Ionis Pharmaceuticals, Inc.
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ended | 7 | 2 |
| Rest of world
United States, United Kingdom
|
— | 23 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| Spain | 2025-02-12 | 2025-02-12 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-16 | Spain | Acceptable with conditions 2024-10-10
|
2024-10-10 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-10-30 | Spain | Acceptable with conditions | 2024-12-05 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-12-06 | Spain | Acceptable with conditions | 2024-12-06 |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-12-11 | Spain | Acceptable with conditions | 2025-02-04 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-03-06 | Spain | Acceptable with conditions | 2025-03-06 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-03-18 | Acceptable with conditions |