Mycophenolate After Stem Cell Transplant for Systemic Sclerosis

NCT07734012 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 20260406-01H
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Systemic Sclerosis (SSc)

Keywords

Systemic sclerosis; Stem cell transplant; Mycophenolate mofetil

Outcome Measures

Primary Outcomes (3)

• Feasibility:

  The number of participants enrolled at a single center over 18 months.

  18 months

• Efficacy :

  * 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.

  at each visit

• 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

Secondary Outcomes (6)

• 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

• Clinical outcomes:

  day 0, 90, 180 and 270

Study Arms (2)

Intervention Arm

EXPERIMENTAL

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

Control Arm

NO INTERVENTION

No scleroderma prophylaxis (current standard of care)

Interventions

Mycophenolate Mofetil 1000 mg twice daily DRUG

MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT and continued for 6 months.

Intervention Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Consenting participants 18 years of age or older who are initiating AHSCT at The Ottawa Hospital for management of SSc.

You may not qualify if:

• 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

Sponsors & Collaborators

• Ottawa Hospital Research Institute: lead
• The Ottawa Hospital Academic Medical Organization (TOHAMO) Innovation Fund Grant.: collaborator

Study Sites (1)

The Ottawa Hospital

Ottawa, Ontario, Canada

Location

MeSH Terms

Conditions

Scleroderma, Systemic

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Nancy Maltez, Dr

  The Ottawa Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Research Assistant

CONTACT

613 738 8400; nasaidi@ohri.ca

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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).

Study Record Dates

• First Submitted: July 24, 2026
• First Posted: July 29, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: July 29, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)