AZA+Lus VS AZA Monotherapy in HR-MDS

NCT ID: NCT06927232

Last Updated: 2025-04-15

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 86 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-04-30
• Study Completion Date: 2027-01-31

Brief 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Azacitidine+Luspatercept

Group Type: EXPERIMENTAL

Azacitidine (AZA)

Intervention Type: DRUG

Azacitidine 75mg/m/ day \*5 days, 28 days for 1 course

Luspatercept

Intervention Type: DRUG

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.

Azacitidine

Group Type: ACTIVE_COMPARATOR

Azacitidine (AZA)

Intervention Type: DRUG

Azacitidine 75mg/m/ day \*5 days, 28 days for 1 course

Interventions

Azacitidine (AZA)

Azacitidine 75mg/m/ day \*5 days, 28 days for 1 course

Intervention Type: 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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Age ≥18 years old
• Diagnosed as higher-risk MDS (IPSS intermediate-2/high-risk, or IPSS-R >3.5, or IPSS-M moderate high-, high-, very high-risk)
• Untreated patients
• Liver and kidney function less than 2 times of upper limit of normal
• ECOG≤2 and expected survival more than 6 months
• Informed consent signed

Exclusion Criteria

• With active infection
• Other malignant tumors
• Obvious abnormal liver and kidney function, or abnormal function of other organs
• Combined with myelofibrosis
• Have undergone bone marrow transplantation
• Pregnant or lactating women, or men who have recent reproductive needs
• Allergic to azacytidine, Rotercept or excipients
• History of polysorbate 80 allergy
• Refuse to sign informed consent
• Researchers consider it inappropriate to participate in the experiment

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Bing Han

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Other Identifiers

AZA-Lus-HRMDS

Identifier Type: -

Identifier Source: org_study_id