AZA+Lus VS AZA Monotherapy in HR-MDS

NCT06927232 | Not Yet Recruiting Phase 3

• 1 other identifier: AZA-Lus-HRMDS
• Study Type: interventional
• Target: 86
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Higher-risk Myelodysplastic Syndrome

Keywords

luspatercept; azacitidine; higher-risk myelodysplastic syndrome

Outcome Measures

Primary Outcomes (1)

• overall response rate

  3 months, 6 months

Secondary Outcomes (6)

• complete response rate

  3 months, 6 months

• rate of transfusion independence

  3 months, 6 months

• adverse event rate

  through study completion, an average of 1 year

• relapse rate

  through study completion, an average of 1 year

• progress-free survival

  through study completion, an average of 1 year

Study Arms (2)

Azacitidine+Luspatercept

EXPERIMENTAL

Drug: Azacitidine (AZA); Drug: Luspatercept

Azacitidine

ACTIVE COMPARATOR

Drug: Azacitidine (AZA)

Interventions

Azacitidine (AZA) DRUG

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

Azacitidine; Azacitidine+Luspatercept

Luspatercept 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+Luspatercept

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Interventions

Azacitidine; luspatercept

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 1, 2025
• First Posted: April 15, 2025
• Study Start: April 1, 2025
• Primary Completion: January 31, 2026
• Study Completion (Estimated): January 31, 2027
• Last Updated: April 15, 2025

Record last verified: 2025-04