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Sclerosis, SystemicPhase 1/2CARE-SScNCT04356287

Treatment With Human Umbilical Cord-derived Mesenchymal Stromal Cells in Systemic Sclerosis

A Phase 1/2 study testing UCMSC in people with Sclerosis, Systemic and Mesenchymal Stem Cells, run by Marie Hudson, MD at 1 site in Canada.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The purpose of this study is to test the safety and efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells (UCMSC) for the treatment of Systemic Sclerosis (SSc). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Sclerosis, Systemic, Mesenchymal Stem Cells
What is being tested?
UCMSC
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
18 participants
Where
1 siteCanada
Study dates
5 Jan 2023to Dec 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • SSc according to American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) 2103 classification criteria for systemic sclerosis
  • Severe disease defined as:
  • disease duration of 2 years or less with an mRss of > 20 and (ESR > 25 mm and/or hemoglobin < 11 g/dL, not explained by other causes than SSc), or ii) mRss >15 without any restriction as to disease duration plus at least one major organ involvement as defined by: a) respiratory involvement consisting of lung diffusion capacity for carbon monoxide (DLCO) and/or forced vital capacity (FVC) < 80% predicted and evidence of interstitial lung disease (chest X-ray and/or high resolution computed tomography (HRCT) scan); b) renal involvement consisting of past renal crisis and/or stage 2 or 3 chronic kidney disease (glomerular filtration rate between 30-89 mL/min) not explained by other causes than SSc; c) cardiac involvement consisting of reversible congestive heart failure, atrial or ventricular rhythm disturbances such as recurrent episodes of atrial fibrillation or flutter, recurrent atrial paroxysmal tachycardia, conduction abnormalities (2nd or 3rd degree atrioventricular block), and/or mild to moderate pericardial effusion. All causes of organ involvement should be attributed to SSc.
  • Inadequate response (determined by patient and physician judgement) or adverse events necessitating discontinuation of standard therapy (usually consisting of methotrexate 25 mg subcutaneous (or as tolerated) per week and/or mycophenolate mofetil 2-3 gm/d (or as tolerated) for at least 3 months
  • Ineligibility or unwillingness to undergo autologous hematopoietic stem cell transplant

Who cannot join

  • Age < 18 years
  • Pregnancy or unwillingness to use adequate contraception
  • Life-threatening end-organ damage defined as:
  • FVC < 45% and/or DLCO (corrected for hemoglobin) < 30% predicted;
  • Left ventricular ejection fraction < 40% by cardiac echocardiography;
  • Pulmonary hypertension with baseline resting systolic pulmonary arterial pressures > 50 mmHg by cardiac echocardiography, or mean pulmonary artery pressure > 25 mmHg (and pulmonary wedge pressure < 15 mmHg) on right heart catheterization;
Show all 17Show fewer
  • stage 4 or more chronic kidney disease (glomerular filtration rate < 30 ml/min)
  • Liver failure defined as an abnormal transaminase level (aspartate aminotransferase (ASAT), alanine aminotransaminase (ALAT) > 3 normal) unless related to activity of the disease
  • Concurrent neoplasms or myelodysplasia
  • Uncontrolled hypertension
  • Uncontrolled acute or chronic infection (HIV, HTLV-1/2 (Human T-lymphotropic virus), hepatitis B surface Ag positive, hepatitis C positive) or high risk thereof
  • Significant malnutrition with BMI < 18 kg/m2
  • Severe concomitant psychiatric disorder
  • Bone marrow insufficiency defined as neutropenia < 0.5 x 109 cell/L, thrombocytopenia < 30 x 109 cell/L, anemia < 8g/dL, CD4+ T lymphopenia < 200 x 106 cell/L due to other diseases than SSc (CD4 - cluster of differentiation 4)
  • History of poor compliance
  • Concurrent enrolment in any other protocol using an investigational drug
  • Inability to provide informed consent

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Marie Hudson, MD contact

514-340-8222 ext. 23476

marie.hudson@mcgill.ca

Canada1 site, 1 recruiting

  • Sir Mortimer B. Davis Jewish General Hospital

    Montreal, Quebec, H3T 1E2

    Marie Hudson, MD MPH, 1-514-340-8222 ext. 23476, marie.hudson@mcgill.ca

    Recruiting

Other trials in Canada

About this study

Official title: Phase I/II Randomized Controlled Trial of Umbilical Cord-derived mesenChymAl stRomal cElls in Systemic Sclerosis

The purpose of this study is to test the safety and efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells (UCMSC) for the treatment of Systemic Sclerosis (SSc).

Read the detailed description

A single-center, three-arm, randomized, double-blind, placebo-controlled trial is proposed. A total of 18 SSc patients will be enrolled in 3 successive blocks of 6 patients each. After being informed about the study and potential risks, all patients giving written informed consent will be randomized to one of two treatment arms or a placebo arm (total of 6 patients per arm). Within each block, the 6 patients will be randomized in a 2:2:2 ratio in one of the following arms: placebo, 1 infusion of UCMSC (M0), or 2 infusions of UCMSC (M0, M3). Second infusions of UCMSC will be performed only in the absence of Treatment Related Severe Adverse Events (TRSAE). Randomization into blocks 2 and 3 will be staggered, to allow the detection of TRSAE prior to inclusion of patients in a subsequent block, i.e. the second block will be randomized only in the absence of TRSAE one month after the first infusion of all 6 patients in block one, and similarly for the third block.

Being tested
UCMSC, Placebo
Keywords
Systemic Sclerosis, Umbilical cord-derived mesenchymal stromal cells, Scleroderma, Systemic
Medical subject headings
Scleroderma, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • UCMSCBiological

    Also called umbilical cord derived mesenchymal stromal cells

    Each infusion will consist of 1 million MSC/kg suspended in 50 mL of PlasmaLyte A.

  • PlaceboOther

    Each infusion will consist of 50 mL of PlasmaLyte A.

Study groups in NCT04356287
Study groupRoleWhat they receive
One infusion of UCMSCExperimentalPatients receive one intravenous infusion of UCMSC at month 0 and one intravenous infusion of placebo at month 3. Each experimental infusion will consist of 1 million UCMSC/kg suspended in 50 ml of PlasmaLyte A. Each placebo infusion will consist of a similar volume of PlasmaLyte A.
Receives: Biological: UCMSC
Two infusions of UCMSCExperimentalPatients receive one intravenous infusion of UCMSC at month 0 and one intravenous infusion of UCMSC at month 3. Each experimental infusion will consist of 1 million UCMSC/kg suspended in 50 ml of PlasmaLyte A.
Receives: Biological: UCMSC
Placebo infusionsPlacebo comparatorPatients receive intravenous placebo infusions at months 0 and 3. Each placebo infusion will consist of 50 ml of PlasmaLyte A.
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Measure of safety one month after first infusion

    Treatment related severe adverse event using the NCI Common Terminology Criteria for Adverse Events (CTCAE) v5.0 classification [https://ctep.cancer.gov/protocolDevelopment/electronic_applications/ctc.htm]

    Measured: Month 1

Secondary outcomes (1)

  1. Change in modified Rodnan skin score (mRss) between Month 0 and Month 12

    A measure of skin thickness; difference between Month 12 and Month 0 on the mRss [Khanna et al., 2017]

    Measured: Month 0 and Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Apr 2020
  2. Study start5 Jan 2023
  3. Primary completionDec 2026 (estimated)
  4. Study completionDec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
18 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04356287
Sponsor's protocol ID
MP-05-2020-2251
Acronym
CARE-SSc
Status last verified
Jan 2026
First submitted
15 Apr 2020
Last updated
3 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Wang D, Zhang H, Liang J, Wang H, Hua B, Feng X, Gilkeson GS, Farge D, Shi S, Sun L. A Long-Term Follow-Up Study of Allogeneic Mesenchymal Stem/Stromal Cell Transplantation in Patients with Drug-Resistant Systemic Lupus Erythematosus. Stem Cell Reports. 2018 Mar 13;10(3):933-941. doi: 10.1016/j.stemcr.2018.01.029. Epub 2018 Mar 1.

    PubMed 29478901

  • Liang J, Zhang H, Kong W, Deng W, Wang D, Feng X, Zhao C, Hua B, Wang H, Sun L. Safety analysis in patients with autoimmune disease receiving allogeneic mesenchymal stem cells infusion: a long-term retrospective study. Stem Cell Res Ther. 2018 Nov 14;9(1):312. doi: 10.1186/s13287-018-1053-4.

    PubMed 30428931

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing UCMSC in people with Sclerosis, Systemic and Mesenchymal Stem Cells, run by Marie Hudson, MD at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 3 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Marie Hudson, MD (514-340-8222 ext. 23476, marie.hudson@mcgill.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Measure of safety one month after first infusion (time frame: Month 1). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04356287, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2026.

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