NCT05732961招募中2 期
A Phase 2, Single Arm Study of Luspatercept for the Treatment of Anemia in Lower Risk Myelodysplastic Syndromes (MDS) or Non-Proliferative Myelodysplastic Syndromes/ Myeloproliferative Neoplasms (MDS/MPN)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 70
- 试验地点
- 2
- 主要终点
- RBC Transfusion Independence
研究概览
简要总结
The purpose of the study is to see if participants with anemia due to their type of MDS or MDS/MPN will experience a more decreased need for regular blood transfusions if they take luspatercept plus best supportive care, and what effect, good and/or bad, luspatercept has on them and their anemia due to MDS or MDS/MPN. The safety and tolerability of luspatercept will also be evaluated in this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Participant is ≥18 years at the time of signing the informed consent form
- •Participant is willing and able to adhere to the study visit schedule and other protocol requirements
- •Documented diagnosis of MDS or non-proliferative MDS/MPN (WBC < 13,000 U/L)
- •According to WHO 2016 classification
- •Meets IPSS-R classification of very low, low, or intermediate risk disease
- •Documented acquired splicing gene mutation
- •Cohort 1: detectable splicing mutation other than SF3B1: (SRSF2, U2AF1, ZRSR2)
- •Cohort 2: SF3B1 mutation with prior treatment with hypomethylating agent and or lenalidomide
- •<5% blasts in bone marrow
- •Refractory, intolerant to, or ineligible for, prior ESA treatment, as defined by any one of the following:
- •Refractory to prior ESA treatment - non-response or response that is no longer maintained. ESA regimen must have been either:
- •rHu EPO ≥ 40,000 IU/wk for at least 8 doses or equivalent Or darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent
- •Intolerant to prior ESA treatment - discontinuation of prior ESA-containing regimen, at any time after introduction due to intolerance or AE
- •ESA ineligible - Low chance of response to ESA based on endogenous serum EPO > 200 U/L for subjects not previously treated with ESAs
- •Discontinuation of ESAs, G-CSF, GM-CSF ≥ 4 weeks prior to start of study treatment
- •Require RBC transfusions
- •a. Average of ≥ 2 units/8 weeks of pRBCs confirmed for a minimum of 16 weeks immediately preceding registration
- •Applies to on treatment subjects only - females of childbearing potential (FCBP) defined as a sexually mature woman who:
- •has achieved menarche at some point,
- •has not undergone a hysterectomy or bilateral oophorectomy, or
- •has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must:
- •Have two negative pregnancy tests 48 hours apart as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence* from heterosexual contact.
- •Either commit to true abstinence*from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting
- •investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy
- •Applies to on treatment subjects only - Male subjects must:
- •Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy. * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception).
排除标准
- •Prior allogeneic or autologous stem cell transplant
- •MDS associated with del 5q cytogenetic abnormality if no prior lenalidomide treatment
- •Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment
- •ANC < 500/μL (0.5 x 109/L)
- •Platelet count ˂50,000/μL (50 x 109/L)
- •Active other malignancies
- •Severe renal impairment (eGFR < 30 mL/min/1.73 m2)
- •ALT or AST ≥ 3 × ULN
- •Prior treatment with Luspatercept or Sotatercept
- •Pregnant or breastfeeding females
研究组 & 干预措施
Participants with gene mutations other than SF3B1
Experimental
Participants with lower risk MDS or non-proliferative MDS/MPN with somatic splicing gene mutations other than SF3B1
干预措施: Luspatercept (Drug)
Participants with SF3B1 mutation
Experimental
Participants with lower risk MDS or non-proliferative MDS/MPN with SF3B1 mutation who had received hypomethylating agents and or lenalidomide.
干预措施: Luspatercept (Drug)
结局指标
主要结局
RBC Transfusion Independence
时间窗: From start of treatment to up to 18 months
RBC transfusion independence (RBC-TI) as defined by IWG 2006 MDS response criteria
次要结局
- Incidence of treatment related adverse events(From start of treatment to 30 days after the last day of treatment, up to 19 months)
- ASC specks changes with response(End of treatment, up to 18 months)
- Hematological Improvement(From start of treatment to up to 18 months)
- Duration of Response(From start of treatment to up to 18 months)
研究者
研究点 (2)
Loading locations...
相似试验
进行中（未招募）
2 期
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-ThalassemiaAnemiaNCT05664737Bristol-Myers Squibb189
招募中
2 期
Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require TransfusionsMyelodysplastic SyndromesAnemiaNCT05384691University of Leipzig213
招募中
1 期
Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESAMDSMyelodysplastic SyndromesNCT05181735Groupe Francophone des Myelodysplasies150
Unknown
2 期
Darbepoetin Alpha in Myelodysplastic Syndromes (MDS)Myelodysplastic SyndromesNCT00443339Groupe Francophone des Myelodysplasies99
Unknown
2 期
Treatment of the Anemia of Myelodysplastic Syndromes by the Association of Epoetin Beta and All Trans Retinoic AcidAnemia in Myelodysplastic SyndromesNCT00437450Groupe Francophone des Myelodysplasies99