ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
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).
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria
* Participant has documented diagnosis of MDS according to World Health Organization (WHO) 2016 that meet IPSS-R classification of very low, low, or intermediate-risk disease, (intermediate-risk of ≤ 3.5 IPSS-R score) confirmed via bone marrow aspirate and:.
i) \< 5% blasts in bone marrow and \< 1% blasts in peripheral blood.
* Participant is not transfusion dependent (NTD) based on IWG2018 criteria.
* Participant is erythropoiesis-stimulating agent naive. Participants may be randomized at the investigator's discretion if the participant received no more than 2 prior doses of epoetin alfa, epoetin alfa biosimilar, or darbepoetin alfa, with the last dose at least 8 weeks prior to randomization.
* Participant has a baseline endogenous serum erythropoietin (sEPO) level of ≤ 500 U/L.
* Participant has symptoms of anemia:.
i) Participant records a severity score of "moderate" or greater on at least 1 PGI-S item of fatigue, weakness, shortness of breath, or dizziness performed during the screening period.
* Participant has a baseline Hb concentration prior to randomization of ≤ 9.5 g/dL. The baseline Hb will be calculated using the mean of the two lowest available Hb measurements within 16 weeks prior to randomization and must include at least one central lab Hb reading done within the screening period (no more than 35 days before randomization). The two Hb measurements must have been performed at least seven days apart. Hb levels less than 21 days following RBC transfusion should not be used. Split samples for local assessments are not required.
Exclusion Criteria
* Participant with secondary MDS (that is, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
* Participant with known history of diagnosis of AML.
* Participant with history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venous thrombosis within 6 months prior to randomization.
* Participant with a history of pure red cell aplasia and/or antibody against erythropoietin.
* Other protocol-defined Inclusion/Exclusion criteria apply.
Primary outcome measure(s)
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.
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
Trial sites (144)
Facility
City
Region
Status
Local Institution - 0070
Clovis
California
Local Institution - 0179
Fountain Valley
California
Local Institution - 0211
Los Alamitos
California
Local Institution - 0258
Los Angeles
California
Local Institution - 0247
Orange
California
Local Institution - 0209
Oxnard
California
Local Institution - 0183
Walnut Creek
California
Local Institution - 0227
Hartford
Connecticut
Local Institution - 0173
New Haven
Connecticut
Halifax Health Medical Center
Daytona Beach
Florida
Local Institution - 0202
Fort Myers
Florida
Local Institution - 0180
Jacksonville
Florida
Local Institution - 0224
Margate
Florida
Local Institution - 0163
Plantation
Florida
Local Institution - 0201
St. Petersburg
Florida
Local Institution - 0238
Tamarac
Florida
Local Institution - 0106
Tampa
Florida
Local Institution - 0240
Honolulu
Hawaii
Local Institution - 0272
Chicago
Illinois
Local Institution - 0184
Skokie
Illinois
Local Institution - 0270
Dyer
Indiana
Local Institution - 0191
Baton Rouge
Louisiana
Local Institution - 0044
Covington
Louisiana
Local Institution - 0007
Bethesda
Maryland
Local Institution - 0222
Saint Louis Park
Minnesota
Local Institution - 0128
Hattiesburg
Mississippi
Local Institution - 0212
Hackensack
New Jersey
Local Institution - 0206
Columbus
Ohio
Local Institution - 0174
Knoxville
Tennessee
Local Institution - 0228
Houston
Texas
Local Institution - 0236
Richmond
Virginia
Local Institution - 0229
Roanoke
Virginia
Local Institution - 0203
Kennewick
Washington
Local Institution - 0010
Buenos Aires
Argentina
Local Institution - 0009
Buenos Aires
Argentina
Local Institution - 0012
Buenos Aires
Argentina
Local Institution - 0071
Blacktown
New South Wales
Local Institution - 0113
Camperdown
New South Wales
Local Institution - 0254
Coffs Harbour
New South Wales
Local Institution - 0246
Gold Coast
Queensland
+ 104 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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