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Trial Title:
Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS
NCT ID:
NCT06006949
Condition:
Myelodysplastic Syndromes
Conditions: Official terms:
Preleukemia
Myelodysplastic Syndromes
Syndrome
Luspatercept
Study type:
Interventional
Study phase:
Phase 4
Overall status:
Not yet recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Roxadustat
Description:
Roxadustat (150 mgqod)
Arm group label:
roxadustat and luspatercept
Intervention type:
Drug
Intervention name:
Luspatercept
Description:
Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to
blood pattern, up to 1.75mg/kg)
Arm group label:
luspatercept
Arm group label:
roxadustat and luspatercept
Summary:
In a randomized controlled phase II/III clinical trial, 58% of patients with lower-risk
MDS had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks
after roxadustat treatment. In a randomized controlled phase III clinical trial,
luspatercept significantly improved transfusion dependence in erythropoietin-stimulating
agents (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life,
compared to placebo. This study aimed to evaluate the efficacy and safety of roxadustat
combined with luspatercept versus luspatercept monotherapy in the treatment of refractory
MDS-RS.
Detailed description:
Myelodysplastic neoplasms (MDS) are heterogeneous clonal disorders of stem cells that
result in peripheral blood cytopenia and ineffective hematopoiesis, with the potential
risk of the development of acute myeloid leukemia (AML). Most patients with
myelodysplastic syndromes with ring sideroblasts (MDS-RS) are stratified into lower-risk
groups by the revised International Prognostic Scoring System (IPSS). At present, the
main therapies for MDS-RS are red blood cell and platelet transfusion, erythropoietin
(EPO), androgen, and iron chelation therapy. Roxadustat can up-regulate transferrin
receptors to increase iron absorption, up-regulate transferrin to promote iron transport,
and down-regulate ferritin levels to indirectly improve iron absorption and transport,
promote plasma iron entry into the bone marrow to generate red blood cells and promote
the production of EPO in the physiological range. Luspatercept generally promotes
advanced erythrocyte maturation by inhibiting the TGF-β/smad2/3 signaling pathway. In a
randomized controlled phase II/III clinical trial, 58% of patients with lower-risk MDS
had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks
after roxadustat treatment. In a randomized controlled phase III clinical trial,
luspatercept significantly improved transfusion dependence in erythropoietin-stimulating
agents (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life,
compared to placebo. The aim of this study was to evaluate the efficacy and safety of
roxadustat combined with luspatercept versus luspatercept monotherapy in the treatment of
refractory MDS-RS. If it is proved that the combination of the two drugs is better than
luspatercept monotherapy, it can quickly improve the anemia of refractory MDS-RS and
improve the quality of life.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Age >18 years old.
2. Patients with a definite diagnosis of MDS-RS and stratified as lower-risk according
to IPSS-R.
3. After at least 6 weeks of rhEPO treatment, with hemoglobin<100g/L
4. Adequate hepatic functions with alanine transaminase (ALT)/aspartate. transaminase
(AST) levels within 2 times of the normal upper limit and total bilirubin levels
within 2 times of the normal upper limit.
5. ECOG≤2 with an expected life span of more than 6 months
6. Documented patient consent.
Exclusion Criteria:
1. Age <18 years old.
2. Complicated with active or uncontrolled infections.
3. Complicated with other malignancies.
4. Creatinine/transaminase ≥ 2 normal upper limit.
5. Complicated with myelofibrosis.
6. Pregnant or lactating women, or men with recent fertility needs
7. Allergic to luspatercept or excipients
8. Patients with history of polysorbate 80 allergy
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Peking union medical college hospital
Address:
City:
Beijing
Country:
China
Contact:
Last name:
Bing Han, Doctor
Phone:
+86-010-69155760
Email:
hanbingtg123@163.com
Start date:
August 2023
Completion date:
August 2025
Lead sponsor:
Agency:
Peking Union Medical College Hospital
Agency class:
Other
Source:
Peking Union Medical College Hospital
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06006949
