1L LUSPA VS ESA IN NTD LOW-RISK MDS PATIENTS
- Conditions
- Myelodysplastic Syndrome (MDS)MedDRA version: 20.0Level: LLTClassification code: 10068361Term: MDS Class: 10029104Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2022-500430-29-00
- Lead Sponsor
- Celgene Corp.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 367
Participant must be = 18 years of age (or local age of consent) at the time of signing the informed consent, Participant has documented diagnosis of MDS according to World Health Organization (WHO) 2016 (Appendix 5) 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: - < 5% blasts in bone marrow and < 1% blasts in peripheral blood., Participant has a baseline endogenous serum erythropoietin level of = 500 U/L., Participant must be transfusion independent, according to IWG 2018 criteria (Appendix 15) as documented by the following criteria: - Received no RBC transfusions within 16 weeks prior to randomization. Note: RBC transfusions of 1 to 2 units within the 16 weeks prior to enrollment are allowed provided those 1-2 RBC transfusion units are administered for an acute event/illness (ie, surgical procedure, bleeding, infection) or presence of comorbidity (including cardiovascular, pulmonary, cerebrovascular), and not for the treatment of low hemoglobin (with or without symptoms) alone, Participant has a mean baseline Hb concentration prior to randomization of = 9.5 g/dL. Mean Hb is defined as the mean of all central/local/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 > 21 days following a transfusion are acceptable. The last measurement must be within 35 days of randomization., Participant has symptom(s) of anemia: - 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 Eastern Cooperative Oncology Group score of 0, 1, or 2., 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.
Participant with MDS associated with del(5q) cytogenetic abnormality or MDS unclassifiable (MDS-U) according to WHO 2016 classification, Participant with myelodysplastic/myeloproliferative neoplasms (MDS/MPN) according to WHO 2016 classification (Appendix 5; ie, chronic myelomonocytic leukemia, Atypical chronic myeloid leukemia, BCR-ABL12, juvenile myelomonocytic leukemia , MDS/MPN unclassifiable)., Participant with secondary MDS (ie, 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 clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia (eg, severe G6PD deficiency, pyruvate kinase deficiency, etc), or hypothyroidism, or any type of known clinically significant bleeding or sequestration. Participant with drug induced anemia (eg, mycophenolate). - Iron deficiency to be determined by serum ferritin < 100 µg/L and additional testing if clinically indicated (eg, calculated transferrin saturation [iron/total iron binding capacity = 20%] or bone marrow aspirate stain for iron)., Bleeding disorders manifested by frequent bleeding episodes (eg, menorrhagia, epistaxis, clotting disorders), Participant with known history of diagnosis of acute myeloid leukemia, Uncontrolled hypertension defined as repeated elevations of systolic blood pressure of = 140 mmHg and/or diastolic blood pressure = 90 mmHg despite adequate treatment or with a history of hypertensive crisis or hypertensive encephalopathy., Participant with prior history of malignancies, other than MDS, unless the participant has been free of the disease for = 5 years. However, participants with the following history/concurrent conditions are allowed: - Basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix or breast - Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis clinical staging system - Other solid tumor with no known active disease, in the opinion of the investigator., Participant with absolute neutrophil count (ANC) = 500/µL (0.5 x 10^9/L) or platelet count = 50,000/µL (50 x 10^9/L).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method