2022-500479-29-00招募中3 期
A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) plus Best Supportive Care (BSC) versus Placebo plus BSC in Participants with IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 166
- 试验地点
- 52
- 主要终点
- For Part I (Phase 2) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments, and vital signs
研究概览
简要总结
For Part I (Phase 2) The primary objective is to evaluate the safety and efficacy (CR within 6 cycles) of 2 Oral-Aza dose regimens, and to recommend the optimal dose for the Phase 3 study (RP3D) based on the totality of safety, efficacy, PK, and PD data. For Part II (Phase 3) The primary objective is to evaluate the CR (as defined in the statistical consideration section) within 6 cycles for both study arms
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female participants must be ≥ 18 years of age at the time of signing the informed consent.
- •Participant has a documented diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of low- or intermediate-risk disease (low IPSS-R score: > 1.5 and ≤ 3.0; intermediate IPSS-R score: > 3.0 and ≤ 4.5). For participants in South Korea, the IPSS-R score for inclusion in Part II (Phase 3) is > 1.5 and ≤ 3.
- •MDS subtypes may include (MDS with single lineage dysplasia [MDS-SLD], MDS with multilineage dysplasia [MDS-MLD], MDS with ring sideroblasts [MDS-RS] [SLD or MLD], MDS unclassifiable [MDS-U], MDS with excess blasts-1 [MDS-EB1], MDS with deletion of 5q [MDS-del(5q)], therapy related myeloid neoplasm [t-MN]). MDS diagnosis, WHO classification, and IPSS-R risk classification will be prospectively determined by independent central pathology and cytogenetics review, and applicable central laboratory results
- •Participant has at least 1 cytopenia (anemia, thrombocytopenia, or neutropenia) meeting one of the protocol defined criteria.
- •Have an Eastern Cooperative Oncology Group ECOG performance status of 0, 1, or 2.
排除标准
- •Participants with prior malignancies must have 1) an expected median life expectancy of at least 12 months at the time of inclusion and 2) no active treatment of any sort for at least 24 weeks prior to randomization (including but not limited to immunotherapy or targeted therapy).
- •Hypoplastic MDS with a marrow cellularity of ≤ 10%
- •Participants diagnosed with MDS-EB2
- •Overlap syndrome (MDS/MPN), CMML, atypical chronic myeloid leukemia (CML), unclassifiable myeloproliferative disorder (MPD), and MDS with moderate to extensive marrow fibrosis (Grade 2 or 3).
- •Significant active cardiac disease within the previous 6 months.
- •Known history of Human Immunodeficiency Virus (HIV) or Hepatitis C Virus (HCV) infection, or evidence of active Hepatitis B Virus (HBV)
- •Participants with an absolute neutrophil count (ANC) of less than 0.5 × 109/L within a week (7 days) of randomization or other laboratory abnormalities as defined in Section 6.
- •Prior treatment with azacitidine (any formulation), decitabine, or other hypomethylating agent.
- •Prior treatment with lenalidomide unless the participant received the last dose ≥ 8 weeks prior to randomization.
- •Use of excluded treatment within 35 days prior to randomization as defined in Section 6.2
- •Use of corticosteroid for any medical condition with an average daily dose above 100mg of hydrocortisone or equivalent 1 week prior to randomization
结局指标
主要结局
For Part I (Phase 2) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments, and vital signs
For Part I (Phase 2) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments, and vital signs
For Part I (Phase 2) - Achievement of CR per IWG 2006 criteria within 6 cycles
For Part I (Phase 2) - Achievement of CR per IWG 2006 criteria within 6 cycles
For Part II (Phase 3) - Achievement of CR within 6 cycles
For Part II (Phase 3) - Achievement of CR within 6 cycles
次要结局
- For Part I (Phase 2) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration
- For Part I (Phase 2) - CR duration
- For Part I (Phase 2) - Achievement of 84-day pRBC-TI within 6 cycles; pRBC-TI duration
- For Part I (Phase 2) - Achievement of 84-day PLT-TI within 6 cycles; PLT-TI duration
- For Part II (Phase 3) - Achievement of 84-day pRBC-TI within 6 cycles
- For Part II (Phase 3) - pRBC-TI duration
- For Part II (Phase 3) - Achievement of 84-day platelet-TI within 6 cycles
- For Part II (Phase 3) - Platelet-TI duration
- For Part II (Phase 3) - Achievement of pRBC transfusion reduction; pRBC transfusion reduction duration
- For Part II (Phase 3) - CR duration
- For Part II (Phase 3) - Achievement of OR within 6 cycles (CR, PR, mCR, HI-E, HI-P, H-IN per IWG 2006 criteria), best OR; OR duration
- For Part II (Phase 3) - OS, EFS (event: death, AML, MDS-EB 2), time to AML, time to subsequent therapy
- For Part II (Phase 3) - Iron parameters
- For Part II (Phase 3) - AEs evaluated using NCI CTCAE criteria, v.5.0, including TEAEs, laboratory assessments and vital signs
- For Part II (Phase 3) - Change from baseline in the FACT-An, QUALMS, and EQ-5D-5L
- For Part II (Phase 3) - Evaluation of healthcare resource use (eg, hospitalization) associated with IP during study
研究者
GSM-CT
Scientific
Celgene Corp.
研究点 (52)
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