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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05949684




Registration number
NCT05949684
Ethics application status
Date submitted
10/07/2023
Date registered
18/07/2023

Titles & IDs
Public title
ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
Scientific title
A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial
Secondary ID [1] 0 0
2022-500430-29-00
Secondary ID [2] 0 0
CA056-025
Universal Trial Number (UTN)
Trial acronym
ELEMENT-MDS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Luspatercept
Treatment: Other - Epoetin Alfa

Experimental: Luspatercept -

Active comparator: Epoetin Alfa -


Treatment: Other: Luspatercept
Specified dose on specified days

Treatment: Other: Epoetin Alfa
Specified dose on specified days
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to Week 96
Secondary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to Week 48
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Up to Week 48
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
From Week 49 to Week 96
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
Up to Week 96
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
Up to Week 48
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
From Week 49 to Week 96
Secondary outcome [7] 0 0
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
Timepoint [7] 0 0
Up to Week 96
Secondary outcome [8] 0 0
Mean Hb change over fixed 24-week periods compared to the baseline Hb
Timepoint [8] 0 0
Baseline, Week 24, Week 48, Week 72, Week 96
Secondary outcome [9] 0 0
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
Timepoint [9] 0 0
Up to Week 96
Secondary outcome [10] 0 0
Number of participants with TD by week 48
Timepoint [10] 0 0
Up to Week 48
Secondary outcome [11] 0 0
Time to TD (IWG 2018 defined as = 3 RBC units/16 weeks) during any continuous 16-week interval until the end of study
Timepoint [11] 0 0
Up to 5 years
Secondary outcome [12] 0 0
Time from first Luspatercept dose to first RBC transfusion
Timepoint [12] 0 0
Up to 5 years
Secondary outcome [13] 0 0
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
Timepoint [13] 0 0
Up to Week 48
Secondary outcome [14] 0 0
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
Timepoint [14] 0 0
Up to Week 96
Secondary outcome [15] 0 0
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)
Timepoint [15] 0 0
Up to Week 48
Secondary outcome [16] 0 0
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)
Timepoint [16] 0 0
Up to Week 96
Secondary outcome [17] 0 0
Number of participants with RBC transfusion independence over at least a consecutive 24-week period
Timepoint [17] 0 0
Up to 5 years
Secondary outcome [18] 0 0
Number of transfusions
Timepoint [18] 0 0
Up to 5 years
Secondary outcome [19] 0 0
Number of transfusions visits/units
Timepoint [19] 0 0
Up to 5 years
Secondary outcome [20] 0 0
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)
Timepoint [20] 0 0
Baseline, Up to 5 years
Secondary outcome [21] 0 0
Change from baseline in self-reported HRQoL assessed by the European quality of life questionnaire 5-dimension (EQ-5D-5L)
Timepoint [21] 0 0
Baseline, Up to 5 years
Secondary outcome [22] 0 0
Number of participants with adverse events (AEs)
Timepoint [22] 0 0
Up to Week 102
Secondary outcome [23] 0 0
Number of participants with antidrug antibody (ADA) (positive or negative)
Timepoint [23] 0 0
Up to Week 102
Secondary outcome [24] 0 0
Pharmacokinetics (PK): Serum concentration
Timepoint [24] 0 0
Up to Week 96
Secondary outcome [25] 0 0
PK: Area under the plasma concentration time curve (AUC)
Timepoint [25] 0 0
Up to Week 96
Secondary outcome [26] 0 0
Number of participants with a platelet response at Week 24, Week 48 and Week 96
Timepoint [26] 0 0
Up to Week 96
Secondary outcome [27] 0 0
Number of participants with a neutrophil response at Week 24, Week 48 and Week 96
Timepoint [27] 0 0
Up to Week 96
Secondary outcome [28] 0 0
Number of participants with acute myeloid leukemia (AML) progression
Timepoint [28] 0 0
Up to 5 years
Secondary outcome [29] 0 0
Time to AML progression
Timepoint [29] 0 0
Up to 5 years
Secondary outcome [30] 0 0
Number of participants with high risk myelodysplastic syndromes (MDS) progression
Timepoint [30] 0 0
Up to 5 years
Secondary outcome [31] 0 0
Time to high-risk MDS progression
Timepoint [31] 0 0
Up to 5 years
Secondary outcome [32] 0 0
Time from date of randomization up to death due to any cause
Timepoint [32] 0 0
Up to 5 years

Eligibility
Key inclusion criteria
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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
11/03/2030
Actual
Sample size
Target
360
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Blacktown Hospital - Blacktown
Recruitment hospital [2] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 0 0
Coffs Harbour Health Campus - Coffs Harbour
Recruitment hospital [4] 0 0
Pindara Private Hospital - Gold Coast
Recruitment hospital [5] 0 0
Monash Health - Clayton
Recruitment hospital [6] 0 0
Austin Health - Heidelberg
Recruitment postcode(s) [1] 0 0
2148 - Blacktown
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
2450 - Coffs Harbour
Recruitment postcode(s) [4] 0 0
4217 - Gold Coast
Recruitment postcode(s) [5] 0 0
3168 - Clayton
Recruitment postcode(s) [6] 0 0
3084 - Heidelberg
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Hawaii
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Kentucky
Country [9] 0 0
United States of America
State/province [9] 0 0
Louisiana
Country [10] 0 0
United States of America
State/province [10] 0 0
Maryland
Country [11] 0 0
United States of America
State/province [11] 0 0
Minnesota
Country [12] 0 0
United States of America
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Mississippi
Country [13] 0 0
United States of America
State/province [13] 0 0
New Jersey
Country [14] 0 0
United States of America
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Ohio
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United States of America
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Tennessee
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Texas
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United States of America
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Virginia
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United States of America
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Washington
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Argentina
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Buenos Aires
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Argentina
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Ciudad Autónoma de Buenos Aires
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Brazil
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Ceará
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Brazil
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Rio Grande Do Sul
Country [23] 0 0
Brazil
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Rio de Janeiro
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Brazil
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São Paulo
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Canada
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Alberta
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Canada
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British Columbia
Country [27] 0 0
Canada
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Ontario
Country [28] 0 0
Chile
State/province [28] 0 0
Región Metropolitana De Santiago
Country [29] 0 0
China
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Anhui
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China
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Beijing
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China
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Chongqing
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China
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Fujian
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China
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Guangdong
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China
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Tianjin
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Zhejiang
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Colombia
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Toulouse
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Nordrhein-Westfalen
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Rheinland-Pfalz
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Sachsen
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Thüringen
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Berlin
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Düsseldorf
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Lübeck
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Mutlangen
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Würzburg
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Greece
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Acha?a
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Attikí
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Alexandroupolis
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Hksar
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Heves
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Szabolcs-Szatmár-Bereg
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Delhi
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Gujarat
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Karnataka
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Odisha
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Hyderabad
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New Delhi
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Lazio
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Lombardia
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Milano
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Italy
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Piemonte
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Italy
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Toscana
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Italy
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Bologna
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Italy
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Reggio Calabria
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Italy
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Roma
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Italy
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Verona
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Japan
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Fukuoka
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Japan
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Hokkaido
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Hyogo
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Kanagawa
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Miyagi
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Osaka
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Tochigi
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Japan
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Nagasaki
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Japan
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Tokyo
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Mexico
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Huixquilucan
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Mexico
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Mexico City
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Mexico
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Oaxaca
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Mexico
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Puebla
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Poland
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Dolnoslaskie
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Poland
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Gdansk
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Poland
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Katowice
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Poland
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Warsaw
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Puerto Rico
State/province [114] 0 0
San Juan
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Spain
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Barcelona [Barcelona]
Country [116] 0 0
Spain
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Catalunya [Cataluña]
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Spain
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Valenciana, Comunitat
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Spain
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Granada
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Spain
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Madrid
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Spain
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Oviedo
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Spain
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Salamanca
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Spain
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Sevilla

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bristol-Myers Squibb
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bristol-Myers Squibb
Address 0 0
Bristol-Myers Squibb
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
Address 0 0
Country 0 0
Phone 0 0
855-907-3286
Fax 0 0
Email 0 0
Clinical.Trials@bms.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html

Supporting document/s available: Study protocol, Clinical study report (CSR)
When will data be available (start and end dates)?
See Plan Description
Available to whom?
See Plan Description
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05949684