NCT04631211终止2 期
A Prospective, Multicenter, Randomized, Open-Label Phase 2, Parallel, Dose Ranging Multidose Study of Thrombosomes® vs Liquid Stored Platelets (LSP) in Bleeding Thrombocytopenic Patients
Cellphire Therapeutics, Inc.10 个研究点 分布在 3 个国家目标入组 21 人开始时间: 2021年3月5日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 21
- 试验地点
- 10
- 主要终点
- Primary Efficacy Endpoint
研究概览
简要总结
This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate, in a dose-escalation manner, the safety, and impact on bleeding, and the preliminary effect on coagulation measures of increasing doses of allogeneic Thrombosomes in comparison to standard of care, LSP.
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
- 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
- •Adults (≥18 years) with TCP as defined by BOTH (a) and (b):
- •a platlet count of ≤ 70,000 platelets/μL blood
- •ANY ONE OR MORE of (1-3):
- •confirmed diagnosis of hematologic malignancy, myeloproliferative disorder, myelodysplastic syndrome, or aplasia
- •undergoing chemotherapy, immunotherapy, radiation therapy or hematopoietic stem cell transplantation
- •refractory to platelet transfusion defined as two 1-hour CCI of <5,000 on consecutive transfusions of LSP or as defined by local site policy (Sacher, 2003)
- •WHO Bleeding Score of 2 or 3
- •Able to provide informed consent directly or through legally authorized representative, and comply with treatment and monitoring
- •Negative pregnancy test for women of childbearing potential
排除标准
- •Any disorder or condition related to any venous thrombosis, embolism, or ischemia within the past 3 months
- •Any disorder or condition related to arterial thrombosis including: ischemia, stroke, MI, or stent placement, within past 6 months
- •Any valve replacement and/or repair of left atrial appendance occlusion device
- •Sinusoidal obstruction syndrom (veno-occlusive disease) or cytopkine release syndrome associated with CAR-T cell therapy
- •Refusal to accept blood products
- •Liver enzyme blood levels greater than 3× the upper limit of normal (ULN)
- •Blood creatinine level greater than 3× ULN
- •Received platelet inhibitor drugs, cyclooxygenase-2 (COX-2) inhibitors, or nonsteroidal anti-inflammatory drugs within 5 days prior to infusion
- •Currently (at the time of randomization) receiving anticoagulant therapy or antiplatelet therapy. Low dose prophylaxis for line clots is not excluded.
- •Receipt of any pro-coagulant agents (e.g., DDAVP, recombinant Factor VIIa or prothrombin complex concentrates (PCC)) other than Tranexamic Acid (TXA) or Epsilon Aminocaproic Acid (EACA, Amicar), within 48 hours of first infusion, or with known hypercoagulable state
- •WHO Bleeding Score of 2 solely due to lumbar puncture, retinal bleeding or GI bleeding or WHO Bleeding Score of 3 solely due to lumbar puncture
- •Receiving L-asparaginase as part of a current cycle of treatment
- •Known inherited or acquired bleeding disorder including, but not limited to: acquired storage pool deficiency or paraproteinemia with platelet inhibition
- •Known inherited or acquired prothrombotic disorders, including antiphospholipid syndrome (Those with lupus anticoagulant or positive antiphospholipid serology without thrombosis are NOT excluded.)
- •On dialysis
- •Receipt of an investigational drug within 1 month before first infusion, other than for treatment of their underlying disease
- •Females pregnant or nursing or unwilling to use contraception during and for 30 days after taking the study product (females). Evidence of effective birth control may be used, at the discretion of the physician
- •Acute or chronic medical disorder that, in the opinion of the Investigator, would impair the ability of the patient to receive or respond to study treatment
- •Prior participation in this study with successful infusion of the investigational or control product
- •Currently enrolled in other trials not related to their primary disease process or involving platelet transfusions, platelet growth factors, or other pro-coagulant agents
结局指标
主要结局
Primary Efficacy Endpoint
时间窗: Evaluated at 24 hours post initial infusion
Cessation or decrease in bleeding at primary bleeding site, based upon the most severe bleeding location at Day 1 baseline taken with in 12 hours prior to infusion, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score evaluated at 24 hours post initial infusion.
次要结局
- Secondary Efficacy Endpoint assessed by Number of days alive and without WHO (World Health Organization) Grade 2a or greater bleeding(7 days after first Thrombosomes or LSP infusion)
- Secondary Efficacy Endpoint assessed for Number, timing, type and reason for administration of all blood products(7 days after first Thrombosomes or LSP infusion)
- Secondary Efficacy Endpoint assessed by 30 day mortality(30 days post first infusion (+/- 2 days))
- Secondary Efficacy Endpoint assessed by measures of coagulation(From baseline through last study visit (up to 30 days (+/- 2 days)))
- Secondary Efficacy Endpoint assessed by changes in markers of endothelial cell injury/repair(From baseline through last study visit (up to 30 days (+/- 2 days)))
- Secondary Efficacy Endpoint assessed by platelet count(24, 48, 72 hours and Day 7 post first infusion)
- Secondary Efficacy Endpoint assessed by measures of hematology(From baseline through last study visit (up to 30 days (+/- 2 days)))
- Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score(24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 post first infusion)
研究者
研究点 (10)
Loading locations...
相似试验
终止
2 期
Obeticholic Acid in Pediatric Subjects With Biliary AtresiaBiliary AtresiaNCT05321524Intercept Pharmaceuticals7
终止
2 期
Randomized Study on Multiple Cycles of Interleukin-7 in HIV Patients Immune Non-respondersHIVNCT01241643Cytheris SA92
已完成
2 期
A Clinical Study of KRN321 in Adult Subjects With Myelodysplastic SyndromeMDSNCT01497145Kyowa Kirin Co., Ltd.45
终止
2 期
A Phase 2 Study of ELX-02 in Patients With Nephropathic CystinosisNonsense MutationCystinosisGenetic DiseaseNCT04069260Eloxx Pharmaceuticals, Inc.3
终止
2 期
NLA101 in Adults Receiving High Dose Chemotherapy for AMLLeukemia, Myeloid, AcuteNCT03301597Nohla Therapeutics, Inc.146