Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2026-03-26 @ 3:20 PM
Ignite Modification Date:
2026-03-26 @ 3:20 PM
NCT ID:
NCT07331818
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.
Study:
NCT07331818
Study Brief:
Protocol Section:
NCT07331818