Mycophenolate After Stem Cell Transplant for Systemic Sclerosis (IMPACT-SSc)
Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial
A pilot single center randomized trial to test whether a full-scale RCT evaluating the role of post-AHSCT maintenance immunosuppression with MMF in preventing disease relapse is feasible .
We are studying whether a brief course of treatment with MMF after AHSCT will serve to prevent disease relapse.
研究概览
详细说明
This study is performed determine the feasibility of a larger pragmatic RCT to evaluate the effectiveness of post- Autologous Hemopoietic Stem Cell Transplant (AHSCT) maintenance therapy with mycophenolate mofetil (MMF) in the prevention of Systemic sclerosis (SSc) relapse.
The study will include 6 participants who will be randomized at 1:1 ratio to treatment (MMF) or control arm (no treatment) for 6 months.
The participants will have 6 visits in total : An initial clinic visit will occur prior to stem cell collection. Post-AHSCT, in-person follow-up visits are scheduled on days 30, 90, 180, 270, and 300, with windows of +/- 1 week for days 30 and 90 and +/- 2 weeks for days 180, 270 and 300.
These visits will be coordinated at an established multidisciplinary (Rheumatology + Cell Therapy) clinic that are part of the patient's clinical care. At each visit, the participants will be asked to complete health questionnaires and laboratory tests will be performed as part of the standard of care of AHSCT recipients.
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名：Clinical Research Assistant
- 电话号码：81630 613 738 8400
- 邮箱：nasaidi@ohri.ca
学习地点
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Ontario
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Ottawa、Ontario、加拿大
- The Ottawa Hospital
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参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
- Consenting participants 18 years of age or older who are initiating AHSCT at The Ottawa Hospital for management of SSc.
Exclusion Criteria:
- Exclusion criteria are focused on patient safety, namely inability to receive treatment with
MMF due to contraindications as determine at time of randomization (day 90 / clinic visit #3) including:
- Renal insufficiency (eGFR <25 mL/minute/1.73m2) or
- Neutropenia (ANC <1.0 x 103/mcL) or
- Thrombocytopenia (platelet count <50 000/mcL)
- Previously documented intolerance.
- Pregnant or breastfeeding - Women of childbearing potential who are not willing to use a highly effective method of contraception for the duration of study participation will be excluded
学习计划
研究是如何设计的？
设计细节
- 主要用途：预防
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Mycophenolate mofetil (MMF)
MMF 1000mg p.o. b.i.d.
starting at day 90 post-AHSCT.
This proposed MMF regimen is used in the management of rheumatic diseases and described in current literature and clinical guidelines.
Duration of 6 months
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MMF 1000mg p.o. b.i.d.
starting at day 90 post-AHSCT and continued for 6 months.
|
|
无干预：Control arm
No scleroderma prophylaxis (current standard of care)
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Feasibility:
大体时间：18 months
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The number of participants enrolled at a single center over 18 months.
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18 months
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Safety : AEs/SAEs
大体时间：Day 90 (treatment start) , Day 180, day 270
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The proportion of patients experiencing a serious AE OR an AE greater that grade 2 (see below) OR an AE requiring discontinuation of the study drug in a participant who has received at least one dose of the study intervention
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Day 90 (treatment start) , Day 180, day 270
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Efficacy :
大体时间：At day 0,90,180 and 270
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-Proportion of patients showing an increase in mRSS of >7 between timepoints .
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At day 0,90,180 and 270
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Efficacy:
大体时间：At day 0,90,180 and 270
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-Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
|
At day 0,90,180 and 270
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Feasibility: Consent rate:
大体时间：Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
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Proportion of eligible patients who provide consent .
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Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
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Feasibility: Retention rate: Proportion of participants retained
大体时间：Day 270 (clinic visit #5)
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Proportion of participants retained
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Day 270 (clinic visit #5)
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Feasibility: Study completion rate:
大体时间：Day 270
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Proportion of participants who completed proposed clinical outcomes at all pre determined time points
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Day 270
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Feasibility: Adherence rate:
大体时间：Day 180 and Day 270
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Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.
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Day 180 and Day 270
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Auto-antibody testing (clinical)
大体时间：Prior to AHSCT and Day 180 (visit# 4)
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Proportion of patients with a change in auto-antibody testing
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Prior to AHSCT and Day 180 (visit# 4)
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Clinical outcomes:
大体时间：day 0, 90, 180 and 270
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Proportion of patients showing a change between timepoints in Modified Rodnan Skin Score (mRSS): Total score range 0 to 51.
Higher scores indicate greater skin thickening and more severe skin involvement .
|
day 0, 90, 180 and 270
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Clinical outcome:
大体时间：day 0,90,180 and 270
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Proportion of patients showing a change in Scleroderma Health Assessment Questionnaire (SHAQ) Disability Index between timepoints. Score range: 0 to 3. Higher scores indicate greater functional disability and worse health status. |
day 0,90,180 and 270
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Clinical outcome:
大体时间：Day 0,90,180 and 270
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Proportion of patients showing a change in global score between timepoints Patient and physician global score: Visual analog scale (0 to 10)
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Day 0,90,180 and 270
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合作者和调查者
调查人员
- 首席研究员：Nancy Maltez, Dr、The Ottawa Hospital
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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