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NCT Number: NCT07734012

Mycophenolate After Stem Cell Transplant for Systemic Sclerosis

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.

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Key information

Conditions

Systemic Sclerosis (SSc) Connective Tissue Diseases Scleroderma, Systemic Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Ottawa Hospital

Ottawa, Ontario, Canada

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

  1. Feasibility:

    Time frame: 18 months

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

  2. Efficacy :

    Time frame: At day 0,90,180 and 270

    -Proportion of patients showing an increase in mRSS of >7 between timepoints .

  3. 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

  4. 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

  1. Feasibility: Consent rate:

    Time frame: Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.

    Proportion of eligible patients who provide consent .

  2. Feasibility: Retention rate: Proportion of participants retained

    Time frame: Day 270 (clinic visit #5)

    Proportion of participants retained

  3. Feasibility: Study completion rate:

    Time frame: Day 270

    Proportion of participants who completed proposed clinical outcomes at all pre determined time points

  4. Feasibility: Adherence rate:

    Time frame: Day 180 and Day 270

    Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.

  5. Clinical outcomes:

    Time frame: 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 .

  6. Auto-antibody testing (clinical)

    Time frame: Prior to AHSCT and Day 180 (visit# 4)

    Proportion of patients with a change in auto-antibody testing

  7. Clinical outcome:

    Time frame: 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.

  8. Clinical outcome:

    Time frame: Day 0,90,180 and 270

    Proportion of patients showing a change in global score between timepoints Patient and physician global score: Visual analog scale (0 to 10)

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Research Assistant

CONTACT

[email protected]

613 738 8400 ext. 81630

Sponsors and collaborators

Lead sponsor

Ottawa Hospital Research Institute

Other

Collaborators

  • The Ottawa Hospital Academic Medical Organization (TOHAMO) Innovation Fund Grant.

Registry information

Official study title

Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial

Acronym: IMPACT-SSc

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Jul 29, 2026
Registry last updated
Aug 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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