Treatment With Human Umbilical Cord-derived Mesenchymal Stromal Cells in Systemic Sclerosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMarie Hudson, MD
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
SSc according to American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) 2103 classification criteria for systemic sclerosis
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
Disqualifiers
Age < 18 years
Pregnancy or unwillingness to use adequate contraception
FVC < 45% and/or DLCO (corrected for hemoglobin) < 30% predicted;
Left ventricular ejection fraction < 40% by cardiac echocardiography;
Trial design
Design model
Parallel
Treatments tested in this trial
UCMSC
Biological/VaccineEach infusion will consist of 1 million MSC/kg suspended in 50 mL of PlasmaLyte A.
Placebo
Other interventionEach infusion will consist of 50 mL of PlasmaLyte A.
Treatment groups
18 Participants
are divided into 3 treatment groupsGroup A: One infusion of UCMSCExperimental treatment 1 intervention
Group B: Two infusions of UCMSCExperimental treatment 1 intervention
Group C: Placebo infusionsPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
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 \[https://ctep.cancer.gov/protocolDevelopment/electronic\_applications/ctc.htm\]
Time frame
Month 1Secondary outcomes
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 \[Khanna et al., 2017\]
Time frame
Month 0 and Month 12Other outcomes
1
Safety at the time of infusion, 24 hours, 10 days +/- 24 hours, Month 6, Month 9 and Month 12 (adverse events)
Measure of safety of UCMSC in severe SSc using the NCI Common Terminology Criteria for Adverse Events (CTCAE) v5.0 classification \[https://ctep.cancer.gov/protocolDevelopment/electronic\_applications/ctc.htm\]
Time frame
24 hours, 10 days +/- 24 hours, Month 6, Month 9 and Month 122
Mortality occurring after randomization and up to study completion
Causes of death and their relation to SSc versus the study intervention will be evaluated by the Data and Safety Monitoring Committee (DSMC).
Time frame
1 year3
Modified Rodnan skin score
A measure of skin thickness \[Khanna et al., 2017\]
Time frame
Month 0, Month 3, Month 6 and Month 94
World Health Organization (WHO) performance status
WHO performance status \[Oken et al., 1982\] describes a patient's level of functioning in terms of their ability to care for themselves, daily activity, and physical ability (walking, working, etc.).
Time frame
Month 0, Month 3, Month 6, Month 9 and Month 12Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Marie Hudson, MD
Lead sponsor
Sir Mortimer B. Davis - Jewish General Hospital
Sponsor institution
Medical University of South Carolina
Collaborator
McGill University Health Centre/Research Institute of the McGill University Health Centre
Collaborator
Université de Montréal
Collaborator
Assistance Publique - Hôpitaux de Paris
Collaborator
Centre hospitalier de l'Université de Montréal (CHUM)
Collaborator
University Paris 7 - Denis Diderot
Collaborator
Source ClinicalTrials.gov