Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial
Trial Snapshot
- Phase
- Phase 2
- Status
- Not yet recruiting
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Feasibility:
Study Overview
Brief Summary
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.
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Prevention
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
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
Intervention: Mycophenolate Mofetil 1000 mg twice daily (Drug)
Control arm
No scleroderma prophylaxis (current standard of care)
Outcomes
Primary Outcomes
Feasibility:
Time Frame: 18 months
The number of participants enrolled at a single center over 18 months.
Efficacy :
Time Frame: 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
Time Frame: 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 :
Time Frame: At day 0,90,180 and 270
-Proportion of patients showing an increase in mRSS of \>7 between timepoints .
Efficacy:
Time Frame: 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.
Secondary Outcomes
- 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)