A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions
- Registration Number
- NCT04477850
- Lead Sponsor
- Celgene
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 30
- 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
- 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/exclusion criteria apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Luspatercept Administration Luspatercept -
- Primary Outcome Measures
Name Time Method Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks Week 1 through Week 24
- Secondary Outcome Measures
Name Time Method RBC-TI ≥ 12 weeks Week 1 through Week 24 Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG) Week 1 through Week 24 Duration of 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 Incidence of frequency of AEs Screening through 42 days post last dose Incidence of seriousness of AEs Screening through 42 days post last dose Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline Week 9 through Week 24 Frequency of Anti-drug antibodies (ADA) Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose Mean hemoglobin increase ≥ 1.0 g/dL Week 1 through Week 24 Mean decrease in serum ferritin compared to baseline Week 9 through Week 24 Overall survival (OS) Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose Incidence of severity of AEs Screening through 42 days post last dose 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 Incidence of relationship of AEs to study treatment Screening through 42 days post last dose Incidence of type of adverse events (AEs) 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
Trial Locations
- Locations (24)
Local Institution - 203
🇯🇵Nagasaki-shi, Nagasaki, Japan
Local Institution - 105
🇨🇳Guangzhou, China
Local Institution - 103
🇨🇳Guangzhou, China
Local Institution - 109
🇨🇳Guangzhou, China
Local Institution - 100
🇨🇳Beijing, China
Local Institution - 107
🇨🇳Chengdu, Sichuan, China
Local Institution - 102
🇨🇳Hangzhou City, China
Local Institution - 112
🇨🇳Nanchang, China
Local Institution - 114
🇨🇳Shanghai, China
Local Institution - 108
🇨🇳Nanjing, China
Local Institution - 101
🇨🇳Shanghai, China
Local Institution - 104
🇨🇳Suzhu, China
Local Institution - 106
🇨🇳Tianjin, China
Local Institution - 209
🇯🇵Matsuyama, Ehime, Japan
Local Institution - 111
🇨🇳Wenzhou, China
Local Institution - 210
🇯🇵Osakasayama, Osaka, Japan
Local Institution - 110
🇨🇳Wuhan, China
Local Institution - 206
🇯🇵Kamogawa, Japan
Local Institution - 201
🇯🇵Mibu-Machi, Japan
Local Institution - 208
🇯🇵Ogaki, Japan
Local Institution - 204
🇯🇵Sagamihara, Japan
Local Institution - 205
🇯🇵Osaka, Japan
Local Institution - 207
🇯🇵Sendai, Japan
Local Institution - 202
🇯🇵Shinagawa-ku, Tokyo, Japan