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

Launched by MARIE HUDSON, MD · Apr 21, 2020

Keywords

ClinConnect Summary

This clinical trial is exploring a new treatment for people with systemic sclerosis, a serious autoimmune disease that affects the skin and internal organs. The study is investigating whether using special cells derived from human umbilical cords, called mesenchymal stromal cells (UCMSC), is safe and effective for patients with severe forms of this disease. The researchers are currently looking for participants aged between 65 and 74 who have been diagnosed with systemic sclerosis and have not responded well to standard treatments.

To take part in the trial, potential participants should have a diagnosis of systemic sclerosis and meet certain criteria that indicate their condition is severe. For example, they may need to have significant skin involvement or issues with major organs like the lungs, heart, or kidneys. Participants can expect to receive the UCMSC treatment and will be closely monitored throughout the study to assess their health and any changes in their condition. It’s important to note that individuals with certain serious health issues or who are pregnant cannot participate in this trial. If you or someone you know is interested, it’s a good idea to discuss it with a healthcare provider for more personalized information.

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Eligibility criteria

• Inclusion Criteria:
• • 1. SSc according to American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) 2103 classification criteria for systemic sclerosis
• 2. 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.
• • 3. 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
• • 4. Ineligibility or unwillingness to undergo autologous hematopoietic stem cell transplant
• Exclusion Criteria:
• • 1. Age \< 18 years
• • 2. Pregnancy or unwillingness to use adequate contraception
• 3. 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)
• • 4. Liver failure defined as an abnormal transaminase level (aspartate aminotransferase (ASAT), alanine aminotransaminase (ALAT) \> 3 normal) unless related to activity of the disease
• • 5. Concurrent neoplasms or myelodysplasia
• • 6. Uncontrolled hypertension
• • 7. 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
• • 8. Significant malnutrition with BMI \< 18 kg/m2
• • 9. Severe concomitant psychiatric disorder
• • 10. 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)
• • 11. History of poor compliance
• • 12. Concurrent enrolment in any other protocol using an investigational drug
• • 13. Inability to provide informed consent