Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Ongoing, recruiting
Participants planned
80
Countries
4
Sites
11
Multiple System Atrophy
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
- 27 May 2020 → ongoing
- Decision date (initial)
- 2024-08-20
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-512528-13-00
- EudraCT number
- 2019-001105-24
- WHO UTN
- U1111-1308-8964
- ClinicalTrials.gov
- NCT04165486
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Multiple System Atrophy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10064060 | Multiple system atrophy | 100000004852 |
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 · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ongoing, recruiting | 15 | 1 |
| France | Ongoing, recruiting | 18 | 3 |
| Germany | Ongoing, recruiting | 20 | 6 |
| Portugal | Ongoing, recruiting | 12 | 1 |
| Rest of world
United Kingdom
|
— | 15 | — |
Investigational sites
Hospices Civils De Lyon
Neurology, 59 Boulevard Pinel, 69500, Bron
Assistance Publique Hopitaux De Paris
Neurology, 43 Boulevard De L Hopital, 75013, Paris
Centre Hospitalier Universitaire De Toulouse
Neurology, 1 Place Du Docteur Joseph Baylac, 31300, Toulouse
Universitaetsmedizin Goettingen
Neurology, Robert-Koch-Strasse 40, Weende, Goettingen
Medizinische Hochschule Hannover
Neurology, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Universitaetsklinikum Ulm AöR
Neurology, Oberer Eselsberg 45, Eselsberg, Ulm
Universitaetsklinikum Duesseldorf AöR
Neurology, Moorenstrasse 5, Bilk, Duesseldorf
Philipps-Universitaet Marburg
Neurology, Baldingerstrasse, 35043, Marburg
Klinikum der Universitaet Muenchen AöR
Neurology, Marchioninistrasse 15, Hadern, Munich
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 |
|---|---|---|---|---|---|
| Austria | 2020-05-27 | 2020-06-12 | |||
| France | 2020-06-17 | 2020-06-25 | |||
| Germany | 2020-10-22 | 2020-11-10 | |||
| Portugal | 2023-06-26 | 2023-07-13 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-04 | Austria | Acceptable with conditions 2024-08-13
|
2024-08-14 |
| 2 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-11-06 | Acceptable with conditions | 2025-01-14 | |
| 3 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-04-09 | Austria | Acceptable 2025-06-05
|
2025-06-11 |
| 4 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-08-13 | Austria | Acceptable 2025-11-24
|
2025-11-25 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-01-28 | Acceptable 2025-11-24
|
2026-01-28 |