Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Feasibility:
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 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
- •Consenting participants 18 years of age or older who are initiating AHSCT at The Ottawa Hospital for management of SSc.
排除标准
- •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
研究组 & 干预措施
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
干预措施: Mycophenolate Mofetil 1000 mg twice daily (Drug)
Control arm
No scleroderma prophylaxis (current standard of care)
结局指标
主要结局
Feasibility:
时间窗: 18 months
The number of participants enrolled at a single center over 18 months.
Efficacy :
时间窗: at each visit
* Proportion of patients showing an increase in mRSS of \>7 between timepoints OR * Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
Safety : AEs/SAEs
时间窗: Day 90 (treatment start) , Day 180, day 270
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
Efficacy :
时间窗: At day 0,90,180 and 270
-Proportion of patients showing an increase in mRSS of \>7 between timepoints .
Efficacy:
时间窗: At day 0,90,180 and 270
-Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
次要结局
- Feasibility: Consent rate:(Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.)
- Feasibility: Retention rate: Proportion of participants retained(Day 270 (clinic visit #5))
- Feasibility: Study completion rate:(Day 270)
- Feasibility: Adherence rate:(Day 180 and Day 270)
- Auto-antibody testing (clinical)(Prior to AHSCT and Day 180 (visit# 4))
- Clinical outcomes:(day 0, 90, 180 and 270)
- Clinical outcome:(day 0,90,180 and 270)
- Clinical outcome:(Day 0,90,180 and 270)