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 (168)
Community Cancer Institute
🇺🇸Clovis, California, United States
John Muir Medical Center - Concord Campus
🇺🇸Concord, 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
Local Institution - 0098
🇺🇸Fort Collins, Colorado, United States
Local Institution - 0126
🇯🇵Amagasaki, Hyogo, Japan
Hartford Hospital (HH)
🇺🇸Hartford, Connecticut, United States
Yale-New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Halifax Health Medical Center
🇺🇸Daytona Beach, Florida, United States
Local Institution - 0250
🇩🇪Kempten, Bayern, Germany
Local Institution - 0230
🇺🇸Daytona Beach, Florida, United States
Florida Cancer Specialists - South
🇺🇸Fort Myers, Florida, United States
Baptist MD Anderson Cancer Center
🇺🇸Jacksonville, Florida, United States
D&H Cancer Research Center LLC
🇺🇸Margate, Florida, United States
BRCR Global
🇺🇸Plantation, Florida, United States
Florida Cancer Specialists - North
🇺🇸Saint Petersburg, Florida, United States
Advanced Research
🇺🇸Tamarac, Florida, United States
Moffitt Cancer Center
🇺🇸Tampa, Florida, United States
Local Institution - 0240
🇺🇸Honolulu, Hawaii, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Orchard Healthcare Research Inc.
🇺🇸Skokie, Illinois, United States
Northwest Cancer Centers, P.C.
🇺🇸Dyer, Indiana, United States
Local Institution - 0232
🇺🇸Pikeville, Kentucky, United States
Our Lady of the Lake RMC
🇺🇸Baton Rouge, Louisiana, United States
Pontchartrain Cancer Center
🇺🇸Covington, Louisiana, United States
Local Institution - 0097
🇺🇸New Orleans, Louisiana, United States
American Oncology Partners of Maryland, PA
🇺🇸Bethesda, Maryland, United States
Metro-Minnesota Community Clinical Oncology
🇺🇸Saint Louis Park, Minnesota, United States
Hattiesburg Clinic Hematology/Oncology
🇺🇸Hattiesburg, Mississippi, United States
John Theurer Cancer Center at Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
🇺🇸Columbus, Ohio, United States
University of Tennessee Medical Center
🇺🇸Knoxville, Tennessee, United States
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
VCU Health Adult Outpatient Pavillion
🇺🇸Richmond, Virginia, United States
Blue Ridge Cancer Care
🇺🇸Roanoke, Virginia, United States
Kadlec Clinic Hematology and Oncology
🇺🇸Kennewick, Washington, United States
Local Institution - 0213
🇺🇸Seattle, Washington, United States
Centro Mdico "Barrio Parque"
🇦🇷Buenos Aires, Argentina
Hospital Británico de Buenos Aires
🇦🇷Buenos Aires, Argentina
Instituto Alexander Fleming
🇦🇷Ciudad Autónoma de Buenos Aires, Argentina
Blacktown Hospital
🇦🇺Blacktown, New South Wales, Australia
Royal Prince Alfred Hospital
🇦🇺Camperdown, New South Wales, Australia
Coffs Harbour Health Campus
🇦🇺Coffs Harbour, New South Wales, Australia
Pindara Private Hospital
🇦🇺Gold Coast, Queensland, Australia
Monash Health
🇦🇺Clayton, Victoria, Australia
Austin Health
🇦🇺Heidelberg, Victoria, Australia
Hospital Universitario Walter Cantidio
🇧🇷Fortaleza, Ceará, Brazil
Hospital São Lucas da PUCRS
🇧🇷Porto Alegre, Rio Grande Do Sul, Brazil
HEMORIO
🇧🇷Rio de Janeiro, Brazil
Hospital das Clinicas FMUSP
🇧🇷São Paulo, Brazil
Arthur J.E. Child Comprehensive Cancer Centre
🇨🇦Calgary, Alberta, Canada
St. Paul's Hospital
🇨🇦Vancouver, British Columbia, Canada
Victoria Hospital & Children's Hospital - London Health Sciences Centre
🇨🇦London, Ontario, Canada
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
Enroll SpA
🇨🇱Santiago, Región Metropolitana De Santiago, Chile
FALP
🇨🇱Santiago, Región Metropolitana De Santiago, Chile
Clínica Inmunocel
🇨🇱Santiago, Región Metropolitana De Santiago, Chile
Bradfordhill
🇨🇱Santiago, Región Metropolitana De Santiago, Chile
Anhui Provincial Hospital
🇨🇳Hefei, Anhui, China
Peking University People's Hospital
🇨🇳Beijing, Beijing, China
Beijing Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
The First Affiliated Hospital of Chongqing Medical University
🇨🇳Chongqing, Chongqing, China
Local Institution - 0096
🇨🇳Fuzhou Fujian, Fujian, China
The First Affiliated hospital of Xiamen University
🇨🇳Xiamen, Fujian, China
Local Institution - 0031
🇨🇳Guangzhou, Guangdong, China
The Second Afilliated Hospital of Hebei Medical University
🇨🇳Shijiazhuang, Hebei, China
The First Hospital of Harbin
🇨🇳Harbin, Heilongjiang, China
People's Hospital of Henan Province
🇨🇳Zhengzhou, Henan, China
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
🇨🇳Wuhan, Hubei, China
Xiangya Hospital Central South University
🇨🇳Changsha, Hunan, China
Jiangsu Province Hospital
🇨🇳Nanjing, Jiangsu, China
The First Affiliated Hospital of Soochow University
🇨🇳Suzhou, Jiangsu, China
The First Affiliated Hospital of Nanchang University
🇨🇳Nanchang, Jiangxi, China
The First Hospital of Jilin University
🇨🇳Changchun, Jilin, China
The First Hospital of China Medical University
🇨🇳Shenyang, Liaoning, China
The Affiliated Hospital of Qingdao University
🇨🇳Qingdao, Shandong, China
Shanghai Sixth People's Hospital
🇨🇳Shanghai, Shanghai, China
Shaanxi provincial people's hospital
🇨🇳Xi'an, Shanxi, China
West China Hospital of Sichuan University
🇨🇳Cheng Du, Sichuan, China
Institute of hematology&blood disease hospital
🇨🇳Tianjin, Tianjin, China
The first Affiliated Hospital, Zhejiang University School of Medicine
🇨🇳Hangzhou, Zhejiang, China
The First Affiliated Hospital of Wenzhou Medical University
🇨🇳Wenzhou, Zhejiang, China
Hospital Pablo Tobon Uribe
🇨🇴Medellin, Antioquia, Colombia
Sociedad De Oncologia Y Hematologia Del Cesar
🇨🇴Valledupar, Cesar, Colombia
Local Institution - 0267
🇨🇴Piedecuesta, Santander, Colombia
Oncomédica S.A.S
🇨🇴Montería, Colombia
Fakultní nemocnice Brno Bohunice
🇨🇿Brno, Brno-město, Czechia
Fakultni nemocnice Ostrava
🇨🇿Ostrava, Moravskoslezský Kraj, Czechia
Vseobecna fakultni nemocnice v Praze
🇨🇿Praha 2, Czechia
Centre Hospitalier Universitaire de Nice - Hôpital l'Archet
🇫🇷Nice, Alpes-Maritimes, France
CHU Bordeaux Haut-Leveque
🇫🇷Pessac, Aquitaine, France
Centre Hospitalier Régional Universitaire de Tours - Hôpital Bretonneau
🇫🇷Tours, Indre-et-Loire, France
Chu Grenoble Alpes
🇫🇷La Tronche, Isère, France
Centre Hospitalier Régional Universitaire de Nancy - Hôpitaux de Brabois
🇫🇷Vandoeuvre lès Nancy, Lorraine, France
Centre Hospitalier Universitaire d'Angers
🇫🇷Angers, Maine-et-Loire, France
Gustave Roussy
🇫🇷Villejuif, Val-de-Marne, France
Hôpital Saint-Louis
🇫🇷Paris, France
Institut Universitaire du Cancer Toulouse - Oncopole - CHU de TOULOUSE
🇫🇷Toulouse, France
Onkologie Erding
🇩🇪Erding, Bayern, Germany
Gemeinschaftspraxis für Hämatologie und Onkologie Münster
🇩🇪Münster, Nordrhein-Westfalen, Germany
InVO Institut für Versorgungsforschung in der Onkologie
🇩🇪Koblenz, Rheinland-Pfalz, Germany
Universitätsklinikum Leipzig
🇩🇪Leipzig, Sachsen, Germany
Universitätsklinikum Jena
🇩🇪Jena, Thüringen, Germany
Praxis fur Hamatologie und Onkologie
🇩🇪Berlin, Germany
Local Institution - 0028
🇩🇪Berlin, Germany
Universitaetsklinikum Duesseldorf
🇩🇪Düsseldorf, Germany
Local Institution - 0255
🇩🇪Lübeck, Germany
Kliniken Ostalb, Stauferklinikum
🇩🇪Mutlangen, Germany
Gemeinschaftspraxis Dres. med. Björn Schöttker & Dominik Pretscher
🇩🇪Würzburg, Germany
Local Institution - 0242
🇬🇷Patras, Achaḯa, Greece
University Hospital of Patras
🇬🇷Patras, Achaḯa, Greece
General Hospital of Athens "G. Gennimatas"
🇬🇷Athens, Attikí, Greece
Local Institution - 0245
🇬🇷Athens, Attikí, Greece
Attikon General University Hospital
🇬🇷Chaidari, Attikí, Greece
University Hospital of Alexandroupolis
🇬🇷Alexandroupolis, Greece
Local Institution - 0034
🇭🇰Hksar, Hong Kong
Heves Vármegyei Markhot Ferenc Oktatókórház és Rendelőintézet
🇭🇺Eger, Heves, Hungary
Szabolcs Szatmár Bereg Vármegyei Oktatókórház
🇭🇺Nyiregyhaza, Szabolcs-Szatmár-Bereg, Hungary
Semmelweis Egyetem
🇭🇺Budapest, Hungary
All India Institute of Medical Sciences
🇮🇳New Delhi, Delhi, India
Rajiv Gandhi Cancer Institute And Research Centre
🇮🇳New Delhi, Delhi, India
Hemato Oncology Clinic
🇮🇳Ahmedabad, Gujarat, India
Local Institution - 0273
🇮🇳Ahmedabad, Gujarat, India
Local Institution - 0177
🇮🇳Bengaluru, Karnataka, India
Institute of Medical Sciences and Sum Hospital
🇮🇳Bhubaneswar, Odisha, India
Local Institution - 0185
🇮🇳Hyderabad, India
Local Institution - 0186
🇮🇳New Delhi, India
Fondazione Policlinico Tor Vergata
🇮🇹Rome, Lazio, Italy
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Lombardia, Italy
ASST Grande Ospedale Metropolitano Niguarda
🇮🇹Milan, Milano, Italy
Humanitas
🇮🇹Rozzano, Milano, Italy
Azienda Ospedaliera Ordine Mauriziano di Torino
🇮🇹Torino, Piemonte, Italy
Azienda Ospedaliera Universitaria Careggi
🇮🇹Firenze, Toscana, Italy
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'Orsola
🇮🇹Bologna, Italy
Grande Ospedale Metropolitano - Presidio Morelli
🇮🇹Reggio Calabria, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Università Cattolica del Sacro Cuore
🇮🇹Roma, Italy
Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Borgo Roma
🇮🇹Verona, Italy
Local Institution - 0123
🇯🇵Kitakyushu-shi, Fukuoka, Japan
Local Institution - 0117
🇯🇵Sapporo, Hokkaido, Japan
Local Institution - 0120
🇯🇵Sagamihara, Kanagawa, Japan
Local Institution - 0118
🇯🇵Sendai-shi, Miyagi, Japan
Local Institution - 0125
🇯🇵Osaka-sayama, Osaka, Japan
Local Institution - 0121
🇯🇵Shimotsuga, Tochigi, Japan
Local Institution - 0124
🇯🇵Nagasaki, Japan
Local Institution - 0122
🇯🇵Osaka, Japan
Local Institution - 0119
🇯🇵Tokyo, Japan
Hematológica Alta Especialidad SC, consultorio 830
🇲🇽Huixquilucan, Mexico
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
🇲🇽Mexico City, Mexico
Local Institution - 0156
🇲🇽Oaxaca, Mexico
Unidad Médica Onco-hematológica
🇲🇽Puebla, Mexico
Specjalistyczny Szpital im. dra Alfreda Sokołowskiego w Wałbrzychu
🇵🇱Wałbrzych, Dolnośląskie, Poland
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdańsk, Poland
Pratia Onkologia Katowice
🇵🇱Katowice, Poland
MTZ Clinical Research powered by Pratia
🇵🇱Warsaw, Poland
Auxilio Mutuo Cancer Center
🇵🇷San Juan, Puerto Rico
Local Institution - 0237
🇵🇷San Juan, Puerto Rico
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Barcelona [Barcelona], Spain
Institut Català d'Oncologia - L'Hospitalet
🇪🇸L'Hospitalet de Llobregat, Catalunya [Cataluña], Spain
Hospital Clinico de Valencia
🇪🇸Valencia, Valenciana, Comunitat, Spain
Hospital Universitario Virgen Nieves
🇪🇸Granada, Spain
Hospital La Princesa
🇪🇸Madrid, Spain
Hospital Universitario Central de Asturias
🇪🇸Oviedo, Spain
Hospital Universitario de Salamanca - Complejo Asistencial Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Universitario Virgen Del Rocio
🇪🇸Sevilla, Spain