Myelodysplastic Syndromes
Conditions
Keywords
Myelodysplastic Syndromes, MDS, ACE-536, Anemia, Luspatercept
Brief summary
The purpose of this study is to evaluate the efficacy and safety of luspatercept (ACE-536) for the treatment of anemia due to Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) in Chinese and Japanese participants with ring sideroblasts who require Red Blood Cells (RBC) transfusions.
Interventions
DRUGLuspatercept
Specified dose on specified days
Sponsors
Celgene
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Refractory or intolerant to, or ineligible for, prior Erythropoiesis stimulating agent (ESA) treatment as defined by any one of the following: Refractory to prior ESA treatment, Intolerant to prior ESA treatment, or ESA ineligible. * previously treated with an ESA or granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, both agents must have been discontinued ≥ 4 weeks prior to date of luspatercept treatment * Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
Exclusion criteria
* Prior therapy with disease modifying agents for underlying MDS disease * Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding * Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN) Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks | Week 1 through Week 24 |
Secondary
| Measure | Time frame |
|---|---|
| RBC-TI ≥ 12 weeks | Week 1 through Week 24 |
| Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline | Week 9 through Week 24 |
| Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG) | Week 1 through Week 24 |
| Mean hemoglobin increase ≥ 1.0 g/dL | Week 1 through Week 24 |
| Duration of RBC-TI | Week 1 through Week 24 |
| Mean decrease in serum ferritin compared to baseline | Week 9 through Week 24 |
| Mean decrease in iron chelation therapy (ICT) use compared to baseline | Week 9 through Week 24 |
| Time to RBC-TI | Week 1 through Week 24 |
| Progression to acute myeloid leukemia (AML) | Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose |
| Overall survival (OS) | Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose |
| Incidence of type of adverse events (AEs) | Screening through 42 days post last dose |
| Incidence of frequency of AEs | Screening through 42 days post last dose |
| Incidence of severity of AEs | Screening through 42 days post last dose |
| Incidence of seriousness of AEs | Screening through 42 days post last dose |
| Incidence of relationship of AEs to study treatment | Screening through 42 days post last dose |
| Pharmacokinetics - Area under the curve (AUC) | Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose |
| Pharmacokinetics - Maximum plasma concentration of the drug (Cmax) | Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose |
| Frequency of Anti-drug antibodies (ADA) | Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose |
Countries
China, Japan
Contacts
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed