Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Authorised, recruiting
Participants planned
17
Countries
4
Sites
4
congenital neuromuscular disorders
Key facts
- Sponsor
- Argenx
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 24 Sep 2024 → ongoing
- Decision date (initial)
- 2024-09-02
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-509872-41-00
- ClinicalTrials.gov
- NCT06436742
- ISRCTN
- ISRCTN83274361
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
congenital neuromuscular disorders
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.0 | PT | 10083942 | Congenital myasthenic syndrome | 100000004850 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Argenx
- Sponsor organisation
- Argenx
- Address
- Industriepark-Zwijnaarde 7
- City
- Gent
- Postcode
- 9052
- Country
- Belgium
Scientific contact point
- Organisation
- Argenx
- Contact name
- Chief Scientific Officer
Public contact point
- Organisation
- Argenx
- Contact name
- Vice President Clinical Development
Locations
4 EU/EEA countries · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 2 | 1 |
| France | Ongoing, recruitment ended | 2 | 1 |
| Italy | Ongoing, recruitment ended | 2 | 1 |
| Spain | Ongoing, recruitment ended | 4 | 1 |
| Rest of world
United States, United Kingdom, Canada
|
— | 7 | — |
Investigational sites
Assistance Publique Hopitaux De Paris
Service de Neuromyologie, 47 Boulevard De L Hopital, 75651, Paris Cedex 13
IRCCS Foundation Istituto Neurologico Carlo Besta
Unità di Fase I, 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 | 2024-09-27 | 2024-10-03 | 2024-10-11 | ||
| Italy | 2024-11-05 | 2024-11-28 | 2025-01-09 | ||
| Spain | 2024-09-24 | 2024-09-27 | 2025-01-10 |
Application history
9 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-05-20 | Spain | Acceptable 2024-09-02
|
2024-09-02 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-03-17 | Acceptable | 2025-04-22 | |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-03-17 | Acceptable | 2025-05-13 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-03-17 | Spain | Acceptable | 2025-04-17 |
| 5 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-06-18 | Spain | Acceptable 2025-08-12
|
2025-08-12 |
| 6 | SUBSTANTIAL MODIFICATION | SM-6 | 2025-09-19 | Spain | Acceptable 2025-12-04
|
2025-12-05 |
| 7 | SUBSTANTIAL MODIFICATION | SM-8 | 2025-12-23 | Acceptable | 2026-02-18 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-9 | 2026-01-07 | Spain | Acceptable | 2026-01-29 |
| 9 | SUBSTANTIAL MODIFICATION | SM-7 | 2026-01-08 | Acceptable | 2026-03-02 |