NCT05181592

Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.

A Phase IIIb, Open-label, Single Arm Study to Evaluate the Efficacy and Safety of Luspatercept in Patients With Lower-risk MDS and Ring-sideroblastic Phenotype (MDS-RS)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: GWT-TUD GmbH · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of luspatercept in patients with lower-risk MDS and ring-sideroblastic phenotype (MDS-RS)

Detailed description

This is a phase IIIb, single arm, multicenter study to further explore the efficacy and safety of luspatercept in subjects with anemia due to IPSS-R very low-, low-, or intermediate-risk MDS with RS who require RBC transfusions. The study will consist of a screening period, a treatment period (primary phase and extension phase), and a posttreatment follow-up period. The study will involve study sites in Germany, France, Austria and Switzerland. It is planned to include 70 patients to receive treatment with luspatercept to end up with 64 evaluable subjects.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	Luspatercept	Once 1.75 mg/kg on Day 1 of each 21-day cycle for 24 weeks (9 cycles)

Timeline

• Start date: 2021-10-27
• Primary completion: 2026-10-31
• Completion: 2027-02-28
• First posted: 2022-01-06
• Last updated: 2025-09-30

Locations

13 sites across 4 countries: Austria, Germany, Spain, Switzerland