Plasmatic Biomarkers Associated With Short-term Luspatercept Treatment of Lower Risk Myelodysplastic Syndromes (MDS) Patients
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 150
- 主要终点
- metabolites and biomarkers associated with luspatercept-related asthenia
研究概览
简要总结
Our project aims to identify metabolites and biomarkers associated with luspatercept-related asthenia in a prospective French study of low-risk MDS patients treated per EMA guidelines.
This is a prospective cohort of patients with MDS treated with luspatercept. The main comparisons will consist in exploring differences in biomarkers plasma levels between patients experiencing fatigue after luspatercept initiation, vs those who do not.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed Written Informed Consent
- •Participants must be ≥ 18 years of age
- •Type of Participant and Target Disease Characteristics:
- •Participant had documented diagnosis of MDS according to World Health Organization (WHO 2022) classification
- •IPSS-R : very low, low, or intermediate-risk disease
- •Less than 5% blasts (< 5%) (in bone marrow and < 1% PB blasts)
- •Performance status: Eastern (ECOG) score of 0, 1, or 2
- •Anemic patients with Hb ≤10g/dL
- •First line therapy by luspatercept for lower risk MDS RS+ and MDS-RS- patients
- •Or Second line therapy by luspatercept for MDS-RS+ refractory or intolerant to prior ESA treatment, as defined by any one of the following:
- •Refractory to prior ESA treatment: documentation of nonresponse or response that was no longer maintained to prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF).
- •Intolerant to prior ESA treatment: Documentation of discontinuation of prior ESA-containing regimen, either as a single agent or in combination (eg, with G-CSF), at any time after introduction due to intolerance or an AE
- •Patient or his entourage having a smartphone to load the application for Fidelio ( the chatbot)
- •Affiliated to SS
- •Exclusion critéria:
- •Higher risk MDS, del 5q syndrome, MDS-RS-T, MD- CMML
- •Participant which has known clinically significant anaemia due to iron, vitamin B12, or folate deficiencies, iron deficiency anaemia or autoimmune haemolytic anaemia
- •Prior allogeneic or autologous stem cell transplant
- •Use of any of the following within 2-weeks prior to treatment:
- •Anticancer cytotoxic chemotherapeutic agent or treatment Any investigational agents within 30 days
- •Psychiatric contraindications
- •Protected people according to articles L1121-5 à L1121-8 of CSP
排除标准
- 未提供
研究组 & 干预措施
LUSPATERCEPT
干预措施: Luspatercept (Drug)
结局指标
主要结局
metabolites and biomarkers associated with luspatercept-related asthenia
时间窗: 6 months of treatement
Plasma biomarkers, including metabolites (energy metabolism, amino acids, lipid mediators), cortisol, iron-metabolism markers, serotonin, inflammatory cytokines, and selected exosomes derived microRNAs, will be explored, and associated with asthenia evaluated using CTCAE v 6.0. and PROs within the first 6 months of treatment with luspatercept. Fatigue will be graded as follows: * Grade 1: fatigue relieved by rest, * Grade 2: fatigue not relieved by rest, limiting instrumental ADL (activities of daily living) * Grade 3: fatigue not relieved by rest, limiting self-care ADL
次要结局
- Evolution of fatigue between luspatercept initiation and up to 6 months(baseline, Week6, Week12, Week18, Week24)
- Metabolites and plasma biomarkers associated with response to luspatercept(V0 to V 4 (Week0, Week6, Week12, Week24 except Month 4.5))
- CTCAE fatigue grading(baseline, Week6, Week12, Week18, Week24 and weekly by chatbot up to 24 weeks)
- Satisfaction questionnaire about the chatbot(Weekly and at the end of the trial up to 24 weeks)
- AE and grading according to CTCAE(baseline, Week6, Week12, Week18, Week24)