Last reviewed · How we verify
NCT05520749: FISiM-Luspa
Efficacy and Safety of Luspatercept: a Study by Fondazione Italiana Sindromi Mielodisplastiche
trial testing Luspatercept in Myeloid Dysplasia in 215 participants. Completed in 31 January 2023.
31 December 2022
Quick facts
| Lead sponsor | Fondazione Italiana Sindromi Mielodisplastiche-ETS |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 215 |
| Start date | 1 January 2022 |
| Primary completion | 31 December 2022 |
| Estimated completion | 31 January 2023 |
| Sites | 1 location across Italy |
Drugs / interventions tested
- Luspatercept (LUSPATERCEPT) — full drug profile →
Conditions studied
- Myeloid Dysplasia — all drugs for Myeloid Dysplasia →
Sponsor
Fondazione Italiana Sindromi Mielodisplastiche-ETS — full company profile →
Who can join
18 and older, any sex, with Myeloid Dysplasia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Myelodysplastic syndromes (MDS) are a group of malignancies characterized by reduced differentiation and increased apoptosis of hematopoietic progenitor cells, leading to ineffective hematopoiesis. Treatment of MDS varies according to prognosis. Patients with low IPSS-R risk have a low probability of progression to acute myeloid leukemia (AML) and the treatment is aimed at controlling cytopenia and improving quality of life (QOL). Anemia is the most common disease feature, occurring in 80%-85% of low-risk patients, 40% of whom eventually become RBC transfusion-dependent (TD). Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta (TGF-beta) superfamily. Luspatercept binds to GDF11, GDF8, activin B, and other ligands. This binding leads to inhibition of Smad2/3 signaling, which is abnormally high in disease models of ineffective erythropoiesis such as MDS, resulting in erythroid maturation and differentiation. Luspatercept is now approved for the treatment of adult patients with TD anemia due to very low-, low-, and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. FISiM (Fondazione Italiana Sindromi Mielodidplastiche) promotes a multicenter, retrospective observational study to collect information on the efficacy and safety of luspatercept in a real world Italian population of adult patients with transfusion-dependent anemia due to very low- and intermediate-risk MDS with ring sideroblasts
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Targeting transforming growth factor beta signaling in metastatic osteosarcoma.
Ge R, Huang GM. · · 2023 · cited 25× · PMID 38021074 · DOI 10.1016/j.jbo.2023.100513 -
Response to luspatercept can be predicted and improves overall survival in the real-life treatment of LR-MDS.
Consagra A, Lanino L, Al Ali NH, Aguirre L, et al · · 2025 · cited 6× · PMID 39944234 · DOI 10.1002/hem3.70086
Verify or expand the search:
- PubMed search for NCT05520749
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Luspatercept
Trials testing the same drug.
- NCT07463820 — A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Tre · Phase 2 · not yet recruiting
- NCT07450313 — The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT · Phase 2 · not yet recruiting
- NCT07215975 — A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East · recruiting
- NCT07465029 — A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in · active not recruiting
- NCT07362095 — Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT) · Phase 1, PHASE2 · recruiting
Other recruiting trials for Myeloid Dysplasia
Currently open trials in the same condition.
- NCT06785077 — Genetic Landscape in Women with Metastatic Ovarian Cancer Before and During Treatment with PARP Inhibitors · NA · recruiting
Other Fondazione Italiana Sindromi Mielodisplastiche-ETS trials
Trials by the same sponsor.
- NCT04212390 — Personalized Medicine Program on Myelodysplastic Syndromes: Characterization of the Patient's Genome for Clinical Decisi · completed
- NCT03920657 — Early and Low Dose Deferasirox (3.5 mg/kg FCT) to Suppress NTBI and LPI as Early Intervention to Prevent Tissue Iron Ove · Phase 2 · terminated
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05520749 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Fondazione Italiana Sindromi Mielodisplastiche-ETS
- Last refreshed: 4 March 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05520749.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing