NCT07345000尚未招募3 期
A Randomized, Controlled, Multicenter, Double-blind Phase III Study Evaluating the Efficacy and Safety of Romiplostim N01 Combined With Standard Immunosuppressive Therapy (IST) Versus Placebo Combined With IST in Treatment-naïve Subjects With Severe Aplastic Anemia
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 210
- 主要终点
- Complete Response (CR) rate at the 6-month time point of treatment.
研究概览
简要总结
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥15 years, regardless of sex (subjects ≥18 years old will be enrolled first; enrollment of subjects aged 15-18 years will commence after sufficient PK/PD data are obtained).
- •Diagnosis of SAA or VSAA according to the British Journal of Haematology (BJH) guidelines. The diagnostic criteria for SAA are as follows:
- •①Bone marrow cellularity <25% of normal; or between 25% and <50%, with residual hematopoietic cells comprising <30%.
- •②Peripheral blood counts must meet at least two of the following three criteria (based on the lowest values from tests within 28 days prior to the first dose):
- •Absolute neutrophil count (ANC) <0.5×10⁹/L
- •Platelet count (PLT) <20×10⁹/L
- •Absolute reticulocyte count (RET) <60×10⁹/L The diagnostic criterion for VSAA is: meeting the SAA criteria + ANC <0.2×10⁹/L.
- •Written informed consent
排除标准
- •History and/or concomitant presence of other primary or secondary bone marrow failure (BMF) syndromes, such as:
- •①Primary: Fanconi anemia, dyskeratosis congenita, congenital amegakaryocytic thrombocytopenia or Shwachman-Diamond syndrome, symptomatic paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), clonal cytopenia of undetermined significance (CCUS), antibody-mediated BMF, idiopathic cytopenia of undetermined significance (ICUS), etc.
- •Secondary: large granular lymphocyte (LGL) leukemia, infiltration of the bone marrow by other systemic malignancies, myelofibrosis, and acute hematopoietic arrest, etc.
- •Note: Asymptomatic PNH and hepatitis-associated SAA may be included if they meet all other inclusion criteria.
- •Evidence of clonal cytogenetic abnormalities at screening.
- •Participation in another clinical trial with investigational drugs or medical devices within 30 days prior to the first dose or within 5 half-lives of the investigational product (whichever is longer).
- •Previous use of any of the following agents prior to the first dose:
- •Alemtuzumab
- •Mycophenolate mofetil ④Sirolimus
- •Tacrolimus ⑥High-dose cyclophosphamide (≥45 mg/kg/day)
- •Cumulative cyclosporine A (CsA) therapy exceeding 4 weeks prior to the first dose. If cumulative use is ≤4 weeks, a washout period of >14 days prior to the first dose is required.
- •Cumulative use of thrombopoietin receptor agonists (TPO-RAs) for >14 days prior to the first dose, or cumulative use ≤14 days with a washout period of <14 days, including:
- •Romiplostim / Nplate® (romiplostim)
- •Eltrombopag ③Hetrombopag ④Recombinant human thrombopoietin, etc.
- •Previous history of hematopoietic stem cell transplantation.
- •Uncontrolled bleeding and/or infection after standard treatment prior to the first dose [defined as persistent signs/symptoms related to infection without improvement despite appropriate antibiotic and/or other therapy], or requiring intravenous (IV) antibiotic administration.
- •Concomitant active CMV and EBV infection (positive test).
结局指标
主要结局
Complete Response (CR) rate at the 6-month time point of treatment.
时间窗: During 6 months of therapy
Proportion of subjects achieving hematopoietic complete response at the 6-month. Hematopoietic complete response is defined as: hemoglobin ≥10 g/dL, absolute neutrophil count ≥1×10⁹/L, and platelet count ≥100×10⁹/L.
次要结局
未报告次要终点
研究者
相似试验
招募中
不适用
Paclitaxel-Coated Balloon Treatment of Chronic RhinosinusitisNCT07290140Airiver Medical, Inc.300
尚未招募
2 期
Nimotuzumab Combined With Nab-paclitaxel/Gemcitabine in the Perioperative Treatment of High-risk Resectable/Borderline Resectable Pancreatic CancerNCT07277452The First Affiliated Hospital with Nanjing Medical University156
招募中
2 期
A Phase II Study to Evaluate the Efficacy and Safety of D-2570 in Subjects With Active Psoriatic ArthritisNCT07315061InventisBio Co., Ltd222
尚未招募
2 期
CONVERT-HB1: Radical Prostatectomy After Systemic Therapy for High-volume Metastatic Hormone-sensitive Prostate CancerMetastatic Prostate CancerNCT07268794Fudan University112
招募中
3 期
A multicentric, randomized, double-blind, placebo-controlled trial to evaluate the efficacy of COLIRIOBCN070660 administered topically in patients with moderately severe to severe non-proliferative diabetic retinopathyDiabetic Retinopathy2023-505791-30-01Bcn Peptides S.A.100