Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Authorised, recruitment pending
Participants planned
55
Countries
1
Sites
4
Clonal Cytopenia of Undetermined Significance with SF3B1 mutation
Key facts
- Sponsor
- Regeneron Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Decision date (initial)
- 2026-06-22
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Clonal Cytopenia of Undetermined Significance with SF3B1 mutation
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 27.0 | PT | 10090299 | Clonal cytopenia of undetermined significance | 100000004851 |
| 27.0 | PT | 10028533 | Myelodysplastic syndrome | 100000004864 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Regeneron Pharmaceuticals Inc.
- Sponsor organisation
- Regeneron Pharmaceuticals Inc.
- Address
- 777 Old Saw Mill River Road
- City
- Tarrytown
- Postcode
- 10591-6717
- Country
- United States
Scientific contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Public contact point
- Organisation
- Regeneron Pharmaceuticals Inc.
- Contact name
- Medical Affairs
Locations
1 EU/EEA country · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Authorised, recruitment pending | 15 | 4 |
| Rest of world
Australia, United Kingdom
|
— | 40 | — |
Investigational sites
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2026-04-16 | Belgium | Acceptable 2026-06-22
|
2026-06-22 |