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Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to Myelodysplastic Syndromes with del5q refractory/resistant/intolerant to Prior Treatments, Who Require Red Blood Cell Transfusion.

Status
Not yet recruiting
Phases
Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-519310-31-00
Acronym
QOL-ONE Phoenix
Enrollment
22
Registered
2025-01-27
Start date
Unknown
Completion date
Unknown
Last updated
2025-11-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Anemia due to MDS with del5q with IPSS-R very low, low, or intermediate risk MDS and abone marrow blast count of < 5%, Refractory or intolerant to, or ineligible for, prior ESA treatment and for prior lenalidomide treatment and who require RBC transfusions.

Brief summary

RBC Transfusion Independence (for 8 weeks in the first 24 weeks)

Detailed description

Safety and tolerability of Luspatercept, RBC-TI at 48 weeks and end of the study, Duration of RBC-TI, Reduction in RBC transfusions, Increase in hemoglobin, Change in quality of life scores (ie. QOL-E and HM-PRO), Change in Serum Ferritin, Change in iron chelation therapy use, Time to RBC TI

Interventions

Sponsors

Associazione Qol-One
Lead SponsorOTHER

Eligibility

Sex/Gender
All
Age
18 Years to No maximum

Design outcomes

Primary

MeasureTime frame
RBC Transfusion Independence (for 8 weeks in the first 24 weeks)

Secondary

MeasureTime frame
Safety and tolerability of Luspatercept, RBC-TI at 48 weeks and end of the study, Duration of RBC-TI, Reduction in RBC transfusions, Increase in hemoglobin, Change in quality of life scores (ie. QOL-E and HM-PRO), Change in Serum Ferritin, Change in iron chelation therapy use, Time to RBC TI

Countries

Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026