2022-500430-29-00招募中3 期
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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 162
- 试验地点
- 51
- 主要终点
- Conversion to TD (IWG 2018 defined as ≥ 3 units/16 weeks) during any continuous 16-week interval within the 96-week Treatment Period
研究概览
简要总结
To compare the proportion of participants with lower-risk NTD MDS who convert to TD (≥ 3 units/16 weeks based on IWG 2018) between luspatercept vs. epoetin alfa.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •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 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). Note: 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.
- •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
- •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 or, epoetin alfa biosimilar, or darbepoetin alfa, with the last dose at least 8 weeks prior to randomization.
排除标准
- •Participant with MDS associated with del(5q) cytogenetic abnormality or MDS unclassifiable (MDS-U) according to WHO 2016 classification
- •Participant with the following subtypes of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) according to WHO 2016,classification81: chronic myelomonocytic leukemia atypical chronic myeloid leukemia, BCR ABL1 negative; juvenile myelomonocytic leukemia,; or MDS/MPN unclassifiable. Note that MDS/MPN-RS-T is not an exclusion.
- •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 (including severe G6PD deficiency, pyruvate kinase deficiency, hemoglobinopathy such as sickle cell disease, etc), or hypothyroidism, or any type of known clinically significant bleeding or sequestration, or drug-induced anemia (eg, mycophenolate). i) Iron deficiency to be determined by serum ferritin below the normal range 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
- •Persistent hypertension. with systolic blood pressure of ≥ 140 mmHg and/or diastolic blood pressure ≥ 90 mmHg or both, during the screening period 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: i) Basal or squamous cell carcinoma of the skin - Carcinoma in situ of the cervix - Carcinoma in situ of the breast - Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis clinical staging system
- •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).
结局指标
主要结局
Conversion to TD (IWG 2018 defined as ≥ 3 units/16 weeks) during any continuous 16-week interval within the 96-week Treatment Period
Conversion to TD (IWG 2018 defined as ≥ 3 units/16 weeks) during any continuous 16-week interval within the 96-week Treatment Period
次要结局
- Achievement of an increase in mean Hb values from baseline of ≥ 1.5 g/dL in any continuous 16-week interval within the 48-week Treatment Period in the absence of transfusion
研究者
GSM-CT
Scientific
Celgene Corp.
研究点 (51)
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