Systemic Sclerosis (SSc)
Conditions
Keywords
Systemic sclerosis, Stem cell transplant, Mycophenolate mofetil
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.
Interventions
DRUGMycophenolate Mofetil 1000 mg twice daily
MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT and continued for 6 months.
Sponsors
Ottawa Hospital Research Institute
The Ottawa Hospital Academic Medical Organization (TOHAMO) Innovation Fund Grant.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Intervention model description
Consenting participants with SSc treated with AHSCT will receive (in 1:1 ratio) either: * MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT or * No immunoprophylaxis (current standard of care).
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
*
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility: | 18 months | The number of participants enrolled at a single center over 18 months. |
| Efficacy : | At day 0,90,180 and 270 | -Proportion of patients showing an increase in mRSS of \>7 between timepoints . |
| Safety : AEs/SAEs | 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: | 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
| Measure | Time frame | Description |
|---|---|---|
| Feasibility: Consent rate: | Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization. | Proportion of eligible patients who provide consent . |
| Feasibility: Retention rate: Proportion of participants retained | Day 270 (clinic visit #5) | Proportion of participants retained |
| Feasibility: Study completion rate: | Day 270 | Proportion of participants who completed proposed clinical outcomes at all pre determined time points |
| Feasibility: Adherence rate: | Day 180 and Day 270 | Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits. |
| Clinical outcomes: | day 0, 90, 180 and 270 | 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 . |
| Auto-antibody testing (clinical) | Prior to AHSCT and Day 180 (visit# 4) | Proportion of patients with a change in auto-antibody testing |
| Clinical outcome: | day 0,90,180 and 270 | 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. |
Countries
Canada
Contacts
CONTACTClinical Research Assistant
PRINCIPAL_INVESTIGATORNancy Maltez, Dr
The Ottawa Hospital
Outcome results
None listed