A Phase 2, Open-Label, Single-Arm, Pilot Study of Safety and Efficacy of CC-4047 (Pomalidomide) in Patients With Advanced Chronic Graft-Versus-Host Disease Developing After Allogeneic Hematological Stem Cell Transplantation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Overall Response (Complete Response + Partial Response + Other)
研究概览
简要总结
This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease.
详细描述
Chronic Graft vs. Host Disease is a major complication after allogeneic hematopoietic stem cell transplantation developing in 30 - 70% of patients. It is a multisystem alloimmune and autoimmune disorder with a negative impact on quality of life and functional status, increased need for extended immunosuppression and is the leading cause of late transplant related mortality.
CC-4047 is a novel immune modulatory drug that is a thalidomide analog with a 4,000 fold greater inhibition of TNF-α production related to thalidomide. Several features of CC-4047 suggest that this drug may be useful in treating chronic GVHD including in vitro suppression of TNF-α production, increasing Th1 and stimulation of IL-12 and sIL-Rα.
This study is an open-label, single-arm, pilot study of efficacy and safety of CC-4047 in patients with advanced chronic GvHD who failed to achieve a response with high-dose corticosteroids or second line systemic immunosuppressive therapy.
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
- 未提供
排除标准
- •Pregnant or lactating females.
- •New immunosuppressive therapy started within the preceding 4 weeks.
- •Extracorporeal photopheresis within the preceding 3 months.
- •Hypersensitivity to any immune modulator drug (IMiD™).
- •Unable to take prophylactic anticoagulation.
- •Any condition which places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- •Acute, persistent, recurrent or late-onset acute GvHD defined by NIH criteria.
- •Any of the following laboratory values at registration:
- •absolute neutrophil count (ANC) less than 1.0 x 109/L,
- •platelets less than 75 x 109/L, or
- •creatinine clearance less than 50 mL/min (Cockroft-Gault formula).
- •Uncontrolled infection requiring systemic antibiotics.
- •Known human immunodeficiency virus (HIV), hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infection.
- •Known uncontrolled arrhythmias or symptomatic heart disease or left ventricular ejection fraction less than 40% (an ECHO should be performed as clinically indicated)
- •Recurrence of cancer for which the transplant was done except for presence of minimal residual disease by PCR.
- •Other cancer less than or equal to 2 years prior study-entry except:
- •Basal cell carcinoma of the skin,
- •Squamous cell carcinoma of the skin,
- •Carcinoma in situ of the cervix,
- •Carcinoma in situ of the breast, or
- •Prostate cancer (Tumor, Node, Metastasis [TNM] stage T1a or T1b)
研究组 & 干预措施
CC-4047 Arm
CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).
干预措施: CC-4047 (Drug)
结局指标
主要结局
Overall Response (Complete Response + Partial Response + Other)
时间窗: 1 year after last dose of CC-4047
* CR is defined as complete resolution in all of signs and symptoms at all affected organs and tissues * PR is defined as improvement in greater than or equal to 1 organ/tissue with no progression in any other affected organ/tissue * Improvement in chronic GvHD symptoms less than what meets the definition of a PR is defined as other * Progressive disease is defined as failure of therapy to control chronic GvHD despite increasing the dose of primary therapy or adding second line treatments * No response is defined as no change in disease.
次要结局
- Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation(30 days after last dose of CC-4047 or until resolution of event)