Study to Compare the Efficacy and Safety ofLuspatercept vs Epoetin Alfa for the Treatment of Anemia Myelodysplastic Syndrome
- Conditions
- Health Condition 1: D758- Other specified diseases of bloodand blood-forming organs
- Registration Number
- CTRI/2024/06/068588
- Lead Sponsor
- Bristol Myers Squibb India Private Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Yet Recruiting
- Sex
- Not specified
- Target Recruitment
- 0
1) Males and females = 18 years of age (or local age of consent) at the time of signing the informed consent.
2) 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 less than 3.5 IPSS-R score) confirmed via bone marrow aspirate and:
a) less than 5 percentage blasts in bone marrow and less than 1 percentage blasts in peripheral blood
3) Participant has a baseline endogenous serum erythropoietin level of less than 500 UL.
4) Participant must be transfusion independent, according to IWG 2018 criteria as documented by the following criteria
a) Received no RBC transfusions within 16 weeks prior to randomization
5) Participant has a mean baseline Hb concentration prior to randomization of less than or equal to 9.5 g per dL. Mean Hb is defined as the mean of all central or local or pre-transfusion available Hb measurements during the 16 weeks prior to randomization (with a minimum of 2 measurements at least 1 week apart). Only Hb levels greater than 21 days following a transfusion are acceptable. The last measurement must be within 35 days of randomization
6) Participant has symptom(s) of anemia
a) 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
7) Participant has Eastern Cooperative Oncology Group score of 0, 1, or 2
8) Participant must be Erythropoiesis-stimulating agent (ESA) Naive. Prior treatment with epoetin alfa or epoetin biosimilars, or darbepoetin alfa, is not acceptable for entry into the study
1)Participant with MDS associated with del (5q) cytogenetic abnormality or MDS unclassifiable (MDS-U) according to WHO 2016 classification
2)Participant with myelodysplastic or myeloproliferative neoplasms (MDS or MPN) according to WHO 2016 classification that is chronic myelomonocytic leukemia, Atypical chronic myeloid leukemia, BCR-ABL12, juvenile myelomonocytic leukemia, MDS or MPN unclassifiable)
3) Participant with secondary MDS (MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and or radiation for other diseases)
4) Participant with known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia (Example- severe G6PD deficiency, pyruvate kinase deficiency, etc), or hypothyroidism, or any type of known clinically significant bleeding or sequestration. Participant with drug induced anemia (Example - mycophenolate).
a) Iron deficiency to be determined by serum ferritin less than 100 ug per L and additional testing if clinically indicated (Example - calculated transferrin saturation [iron or total iron binding capacity less than or equal to 20 percentage] or bone marrow aspirate stain for iron).
5)Bleeding disorders manifested by frequent bleeding episodes (Example - menorrhagia, epistaxis, clotting disorders)
6)Participant with known history of diagnosis of acute myeloid leukemia
7)Uncontrolled hypertension defined as repeated elevations of systolic blood pressure of greater than or equal to 140 mmHg and or diastolic blood pressure greater than or equal to 90 mmHg despite adequate treatment or with a history of hypertensive crisis or hypertensive encephalopathy
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To compare the proportion of participants with lower-risk NTD MDS who convert to TD (=3 RBC units/16weeks based on IWG 2018)70between luspatercept vs epoetin alfaTimepoint: Week 1 through Week 96
- Secondary Outcome Measures
Name Time Method