Ignite Creation Date:
2025-12-24 @ 9:56 PM
Ignite Modification Date:
2025-12-30 @ 8:33 AM
Study NCT ID:
NCT06927232
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-04-15
First Post:
2025-04-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
AZA+Lus VS AZA Monotherapy in HR-MDS
Sponsor:
Peking Union Medical College Hospital
Organization:
Study Overview
Official Title:
Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: