Ignite Creation Date:
2026-03-26 @ 3:20 PM
Ignite Modification Date:
2026-03-31 @ 11:38 AM
Study NCT ID:
NCT07331818
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-01-12
First Post:
2025-12-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Luspatercept in Patients Affected With Rare Inherited Anemias
Sponsor:
EuroBloodNet Association
Organization:
Study Overview
Official Title:
Luspatercept in Patients Affected With Rare Inherited Anemias
Status:
NOT_YET_RECRUITING
Status Verified Date:
2026-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
LUSPARA
Brief Summary:
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias
Detailed Description:
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias including : ✔ CSA group: constitutional non syndromic sideroblastic anemia () due to germline mutation including those with ALAS2, SLC25A38, SLC19A2, GLRX5, HSPA9. and other gene mutations ✔ CDA group: constitutional dyserythropïetic anemias ( (type I and II) ✔ NTD-DBA group: Diamond-Blackfan anemia (DBA) not requiring regular transfusion support (NTD-DBA) with or without continuous steroid therapy); (therapeutic independence or with continuous steroid therapy); 2 subgroups will be considered: RPS19 versus other genetic subgroups (RPL5, RPL11 and RPS26 mutations) ; to note these 4 genotypes account for the vast majority of patients
Patients will be recruited from centers of expertise within the European Union (France and Italy). In total, 45 patients are will be recruited.
Patients will be recruited from centers of expertise within the European Union (France and Italy). In total, 45 patients are will be recruited.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: