NCT06927232

AZA+Lus VS AZA Monotherapy in HR-MDS

Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 86 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort.

Detailed description

The hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) have been shown to improve survival and delay disease progression in patients with high-risk MDS. They are recommended by the NCCN as first-line treatments for patients with high-risk MDS. Clinical trials have demonstrated an OR rate of approximately 40-50% with AZA in patients with high-risk MDS. Despite the efficacy of HMA therapy, the rate of transfusion independence remains low. Anemia remains the most prominent symptom in refractory patients, with very limited options for subsequent treatment. Prolonged anemia affects every organ function and seriously affects the prognosis of patients. Luspatercept is currently approved for the treatment of patients with both erythropoiesis receptor agonist ( ESA) treatment failures in transfusion-dependent low-risk MDS-RS patients. In a randomized controlled phase III clinical trial, compared to a placebo group, luspatercept significantly improved transfusion dependence and improved hemoglobin and quality of life in refractory MDS-RS patients. A recent conference report suggested that there was no significant difference in efficacy between low-risk and high-risk patients treated with luspatercept and that the HI rate for high-risk patients treated with luspatercept monotherapy was approximately 50%. Thus this study aimed to compare the efficacy of AZA+luspatercept and AZA monotherapy.

Conditions

• Higher-risk Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Azacitidine (AZA)	Azacitidine 75mg/m/ day \*5 days, 28 days for 1 course
DRUG	Luspatercept	Luspatercept 1.0 mg/kg subcutaneously every 3 weeks, adjusted according to hemoglobin, up to 1.75mg/kg. If hemoglobin ≥120g/L, luspatercept can be discontinued.

Timeline

• Start date: 2025-04-01
• Primary completion: 2026-01-31
• Completion: 2027-01-31
• First posted: 2025-04-15
• Last updated: 2025-04-15