Conversion From Immediate Release Tacrolimus to Envarsus XR® in Simultaneous Pancreas-Kidney Recipients: Assessment of Functional, Safety and Quality of Life Outcomes (CIRTEN SPK)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Change in patient and physician-assessed degree of tremors based on Fahn-Tolosa-Marin Tremor Rating Scale
研究概览
简要总结
This is a Phase II/III, Single-center, Prospective, Open-label, Single Arm Study of 20 Simultaneous Kidney Pancreas recipients who received a transplant at least 3 months, but no more than 5 years prior, with a history of tremors following transplantation.
详细描述
Accrual objective: Enrollment of 2-3 participants per month and all participants within an 18 month period. With minimum of 1 year follow-up post-conversion, we anticipate the entire study would be completed within 2 ½ years.
Study design: This is a Phase II/III, Single-center, Prospective, Open-label, Single Arm Study of 20 Simultaneous Kidney Pancreas recipients who received a transplant at least 3 months, but no more than 5 years prior, with a history of tremors following transplantation.
Study duration: There will be a minimum of 1 year follow-up post-conversion, and it is expected that the entire study would be completed within 2 ½ years.
Study aims:
- Determine if EnvarsusXR® reduces tremor severity compared to IR tacrolimus
- Determine if EnvarsusXR® improves renal function or glycemic control compared to IR tacrolimus
- Compare the efficacy of EnvarsusXR® by comparing the outcomes (patient, pancreas graft and kidney graft survival) of patients treated with Envarsus XR to those of contemporary and historical controls at our center treated with IR tacrolimus, with the goal of showing non-inferiority.
- Compare insulin resistance and insulin sensitivity by defined measures post conversion between T1D and T2D recipients.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult, 18-70 years of age
- •Participant must be able to understand and provide consent
- •History of Diabetes Type 1 or Insulin-Dependent Diabetes Type 2 with Chronic Kidney Disease (CKD)
- •Recipient of a Simultaneous Pancreas Kidney (SPK) transplant, 3- 60 months prior to screening, per Principal Investigator's discretion.
- •Have a history of tremors following transplantation
- •Stable pancreas allograft function as evidenced by no requirement of exogenous insulin or oral anti-diabetic agents and stable pancreatic enzymes
- •Stable kidney allograft function
- •Currently taking Immediate-Release (IR) tacrolimus
- •Women of child-bearing potential (WOCP) must have a negative pregnancy test at the time of study entry
排除标准
- •Currently maintained on an extended-release tacrolimus immunosuppressive regimen
- •Previous history of tremors prior to transplantation
- •Solitary pancreas transplant recipients
- •History of solid organ transplant other than a kidney or pancreas
- •Uncontrolled concomitant infection at the discretion of the investigator
- •Presence of Donor Specific Antibodies
研究组 & 干预措施
Envarsus XR
Envarsus XR (extended release) will be administered orally, once-daily, for 6 months.
干预措施: Envarsus XR (Drug)
结局指标
主要结局
Change in patient and physician-assessed degree of tremors based on Fahn-Tolosa-Marin Tremor Rating Scale
时间窗: Over 6 months
FTM-TRS will be used as a tool for essential tremor scoring. (0: no tremor, 1: barely perceptible tremor, 2: \< 2 cm, 3: 2-4 cm, and 4: \> 4 cm).
Change in patient and physician-assessed degree of tremors based on Quality of Life in Essential Tremor (QUEST) Questionnaire
时间窗: Over 6 months
QUEST Questionnaire, a scoring tool will be used to measure the quality of life among patients with Essential Tremor. It consists of 30 items which are rated on a five-point scale (score 0-4), corresponding to the frequency (never, rarely, sometimes, frequently, always) with which tremor was perceived to affect a function or to be associated with various feelings and attitudes. A few items (e.g. 'I had to quit my job because of tremor') could only be answered at the extremes (yes/no; true/false), and these were scored either 0 or 4.
Scores on Quality of Life in Essential Tremor Questionnaires
时间窗: Over 6 months
Use QUEST (Quality of Life in Essential Tremor Questionnaire) scoring tool
Scores on Modified Transplant Symptom Occurrence and Symptom Distress Scale (MTSOSD) Questionnaire
时间窗: Over 6 months
MTSOSD Questionnaire scores will be used to assess the presence and distress of side effects associated with the use of immunosuppressive drugs after transplantation.
次要结局
- Comparable or improved glycemic control as measured by HbA1c(Over 6 months)
- Comparable or improved glycemic control as measured by mixed meal tolerance test(Over 6 months)
- Tacrolimus doses and levels(Over 6 months)
- Renal function by serum creatinine(Over 6 months)
- Mycophenolate doses and levels(Over 6 months)
- Renal function by eGFR (estimated Glomerular Filtration Rate)(Over 6 months)