Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 29
- 试验地点
- 32
- 主要终点
- Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4
研究概览
简要总结
This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.
详细描述
Screening:
After the informed consent/assent form (ICF) is signed, the screening assessments will be performed in a single visit to establish the eligibility of the participant, based on inclusion and exclusion criteria, as well as aGvHD grading. Scheduling of the first week of ECP treatments and the arrangements for availability of typed and cross-matched donor packed red blood cells (PRBCs) for transfusion, if required, will be made in advance of participants entering the Treatment Period.
Treatment Period:
Once eligibility is established, participants will enter the 12-week ECP Treatment Period. The availability of typed and cross-matched donor PRBCs for transfusion during treatment, if needed, should be established prior to the scheduling of ECP treatments.
Participants will be allowed to continue standard aGvHD prophylaxis regimens (e.g., cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil) without the addition of new therapies. Participants will be allowed to discontinue prophylaxis regimens for reasons of toxicity, and will also be allowed to switch to another prophylaxis medication within the same class (e.g., the calcineurin inhibitors cyclosporine and tacrolimus) for reasons of toxicity.
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
- 1 Year 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Methoxsalen with ECP
Participants receive methoxsalen 20 µg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.
干预措施: Methoxsalen (Drug)
Methoxsalen with ECP
Participants receive methoxsalen 20 µg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.
干预措施: Extracorporeal Photopheresis (ECP) (Procedure)
结局指标
主要结局
Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4
时间窗: 4 weeks
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: * CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment * PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
次要结局
- Cumulative Dose of Daily Steroids(From diagnosis of aGvHD to 12 Weeks)
- Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8(8 weeks)
- Number of Participants With Adverse Events(16 weeks)
- Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index(16 weeks)
- Number of Patients With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria(at 4, 8 and 12 weeks)
- Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12(12 weeks)
- Overall Response Rate (ORR) According to the Modified Glucksberg Criteria(4 weeks, 8 weeks, and 12 weeks)
- Number of Patients With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria(at 4, 8 and 12 weeks)