Status:
RECRUITING
Treatment With Human Umbilical Cord-derived Mesenchymal Stromal Cells in Systemic Sclerosis
Lead Sponsor:
Marie Hudson, MD
Collaborating Sponsors:
Medical University of South Carolina
McGill University Health Centre/Research Institute of the McGill University Health Centre
Conditions:
Sclerosis, Systemic
Mesenchymal Stem Cells
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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).
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 ...
Eligibility Criteria
Inclusion
- SSc according to American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) 2103 classification criteria for systemic sclerosis
- Severe disease defined as:
- i) 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
Exclusion
- 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;
- 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
Key Trial Info
Start Date :
January 5 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2025
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT04356287
Start Date
January 5 2023
End Date
December 1 2025
Last Update
April 16 2024
Active Locations (1)
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1
Sir Mortimer B. Davis Jewish General Hospital
Montreal, Quebec, Canada, H3T 1E2