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.

Study Overview

• Status: Not yet recruiting
• Conditions: Systemic Sclerosis (SSc)
• Intervention / Treatment: Drug: Mycophenolate Mofetil 1000 mg twice daily

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 Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Clinical Research Assistant; Phone Number: 81630 613 738 8400; Email: nasaidi@ohri.ca

Study Locations

• Canada
  • Ontario
    • Ottawa, Ontario, Canada
      • The Ottawa Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: Mycophenolate Mofetil 1000 mg twice daily; MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT and continued for 6 months.
No Intervention: Control arm; No scleroderma prophylaxis (current standard of care)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility:	The number of participants enrolled at a single center over 18 months.	18 months
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	Day 90 (treatment start) , Day 180, day 270
Efficacy :	-Proportion of patients showing an increase in mRSS of >7 between timepoints .	At day 0,90,180 and 270
Efficacy:	-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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility: Consent rate:	Proportion of eligible patients who provide consent .	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	Day 270 (clinic visit #5)
Feasibility: Study completion rate:	Proportion of participants who completed proposed clinical outcomes at all pre determined time points	Day 270
Feasibility: Adherence rate:	Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.	Day 180 and Day 270
Auto-antibody testing (clinical)	Proportion of patients with a change in auto-antibody testing	Prior to AHSCT and Day 180 (visit# 4)
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 .	day 0, 90, 180 and 270
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.	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)	Day 0,90,180 and 270

Collaborators and Investigators

• Sponsor: Ottawa Hospital Research Institute
• Collaborators: The Ottawa Hospital Academic Medical Organization (TOHAMO) Innovation Fund Grant.
• Investigators: Principal Investigator: Nancy Maltez, Dr, The Ottawa Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: July 24, 2026
• First Submitted That Met QC Criteria: July 24, 2026
• First Posted (Actual): July 29, 2026

Study Record Updates

• Last Update Posted (Actual): August 7, 2026
• Last Update Submitted That Met QC Criteria: August 5, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: Stem cell transplant; Systemic sclerosis; Mycophenolate mofetil
• Additional Relevant MeSH Terms: Connective Tissue Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Scleroderma, Systemic; Organic Chemicals; Fatty Acids; Lipids; Acids, Acyclic; Carboxylic Acids; Caproates; Mycophenolic Acid
• Other Study ID Numbers: 20260406-01H

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes