ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
- Conditions
- Myelodysplastic Syndromes
- Interventions
- Registration Number
- NCT05949684
- Lead Sponsor
- Bristol-Myers Squibb
- Brief Summary
The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naïve participants who are non-transfusion dependent (NTD).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 360
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Luspatercept Luspatercept - Epoetin Alfa Epoetin Alfa -
- Primary Outcome Measures
Name Time Method Number of participants with lower-risk non-transfusion dependent myelodysplastic syndromes (NTD-MDS) who converted to Transfusion Dependence (TD) during any continuous 16-week interval within the 96-week treatment period Up to Week 96 TD is defined as ≥ 3 red blood cells (RBC) units/16 weeks assessed by International Working Group (IWG) 2018.
- Secondary Outcome Measures
Name Time Method Number of transfusions Up to 5 years Number of transfusions visits/units Up to 5 years Number of participants with adverse events (AEs) Up to Week 102 Pharmacokinetics (PK): Serum concentration Up to Week 96 Time to AML progression Up to 5 years Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 grams/deciliter (g/dL) in any continuous 16-week interval within the 48 week Treatment Period in the absence of transfusion Up to Week 48 Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 g/dL in any continuous 24-week interval within the 48-week and 96-week treatment period in the absence of transfusion Up to Week 96 Number of participants with an increase from baseline in mean Hb values of ≥ 1.0 g/dL in any continuous 24-week interval within the 48-week and 96-week treatment period in the absence of transfusion Up to Week 96 Mean Hb change over fixed 24-week periods compared to the baseline Hb Baseline, Week 24, Week 48, Week 72, Week 96 Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 g/dL in any continuous 16-week interval within the 96-week treatment period in the absence of transfusion Up to Week 96 Number of participants with TD by week 48 Up to Week 48 Time from first Luspatercept dose to first RBC transfusion Up to 5 years Duration of median hematologic improvement in erythroid response(mHI-E) in participants with an increase from baseline in mean Hb values of ≥1.5g/dL in any continuous 16-week interval within 48-week treatment period in absence of transfusion Up to Week 48 Number of participants with RBC transfusion independence over at least a consecutive 24-week period Up to 5 years Time to TD (IWG 2018 defined as ≥ 3 RBC units/16 weeks) during any continuous 16-week interval until the end of study Up to 5 years Duration of median hematologic improvement in erythroid response(mHI-E) in participants with an increase from baseline in mean Hb values of ≥1.5g/dL in any continuous 16-week interval within 96-week treatment period in absence of transfusion Up to Week 96 Number of participants with antidrug antibody (ADA) (positive or negative) Up to Week 102 Number of participants with a neutrophil response at Week 24, Week 48 and Week 96 Up to Week 96 Neutrophil response is defined as an absolute increase from baseline of \> 0.5 × 10\^9/L neutrophils at Week 24, Week 48 and Week 96.
Number of participants with acute myeloid leukemia (AML) progression Up to 5 years Number of participants with high risk myelodysplastic syndromes (MDS) progression Up to 5 years Time to high-risk MDS progression Up to 5 years Time from first dose to first day of response (increase in mean Hb values of ≥ 1.5 g/dL in any continuous 16-week interval within the 48-week Treatment Period in the absence of transfusion) Up to Week 48 Time from first dose to first day of response (increase in mean Hb values of ≥ 1.5 g/dL in any continuous 16-week interval within the 96-week Treatment Period in the absence of transfusion) Up to Week 96 Change from baseline in subscales of self-reported health-related quality-of-life (HRQoL) assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An) Baseline, Up to 5 years Change from baseline in self-reported HRQoL assessed by the European quality of life questionnaire 5-dimension (EQ-5D-5L) Baseline, Up to 5 years PK: Area under the plasma concentration time curve (AUC) Up to Week 96 Number of participants with a platelet response at Week 24, Week 48 and Week 96 Up to Week 96 Platelet response is defined as an increase from baseline in number of platelets to ≥ 30 × 10\^9/L at Week 24, Week 48 and Week 96.
Time from date of randomization up to death due to any cause Up to 5 years
Trial Locations
- Locations (169)
Community Cancer Institute
🇺🇸Clovis, California, United States
Compassionate Cancer Care Medical Group
🇺🇸Fountain Valley, California, United States
Local Institution - 0095
🇺🇸Fresno, California, United States
Cancer and Blood Specialty Clinic
🇺🇸Los Alamitos, California, United States
UCLA Hematology/Oncology - Westwood (Building 200 Suite 120)
🇺🇸Los Angeles, California, United States
St. Joseph Hospital
🇺🇸Orange, California, United States
Ventura County Hematology Oncology Specialists
🇺🇸Oxnard, California, United States
John Muir Health - Behring Pavilion
🇺🇸Walnut Creek, California, United States
Local Institution - 0098
🇺🇸Fort Collins, Colorado, United States
Hartford Hospital (HH)
🇺🇸Hartford, Connecticut, United States
Scroll for more (159 remaining)Community Cancer Institute🇺🇸Clovis, California, United StatesHaifaa Abdulhaq, Site 0070Contact559-618-6208