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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Enrollment
6
MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT and continued for 6 months.
The Ottawa Hospital
Ottawa, Ontario, Canada
Feasibility:
The number of participants enrolled at a single center over 18 months.
Time frame: 18 months
Efficacy :
-Proportion of patients showing an increase in mRSS of \>7 between timepoints .
Time frame: At day 0,90,180 and 270
Safety : AEs/SAEs
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
Time frame: Day 90 (treatment start) , Day 180, day 270
Efficacy:
-Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
Time frame: At day 0,90,180 and 270
Feasibility: Consent rate:
Proportion of eligible patients who provide consent .
Time frame: Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
Feasibility: Retention rate: Proportion of participants retained
Proportion of participants retained
Time frame: Day 270 (clinic visit #5)
Feasibility: Study completion rate:
Proportion of participants who completed proposed clinical outcomes at all pre determined time points
Time frame: Day 270
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Feasibility: Adherence rate:
Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.
Time frame: Day 180 and Day 270
Clinical outcomes:
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 .
Time frame: day 0, 90, 180 and 270
Auto-antibody testing (clinical)
Proportion of patients with a change in auto-antibody testing
Time frame: Prior to AHSCT and Day 180 (visit# 4)
Clinical outcome:
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.
Time frame: day 0,90,180 and 270
Clinical outcome:
Proportion of patients showing a change in global score between timepoints Patient and physician global score: Visual analog scale (0 to 10)
Time frame: Day 0,90,180 and 270