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Clinical Study of the Efficacy and Safety of the Application of Allogeneic Mesenchymal (Stromal) Cells of Bone Marrow, Cultured Under the Hypoxia in the Treatment of Patients With Severe Pulmonary Emphysema

Phase 1
Withdrawn
Conditions
Pulmonary Emphysema
Interventions
Biological: Mesenchymal stem cells
Registration Number
NCT01849159
Lead Sponsor
Federal Research Clinical Center of Federal Medical & Biological Agency, Russia
Brief Summary

Actively developing stem cells (SCs) transplantation techniques cause natural interest to the problem of regeneration in the lungs. Numerous experimental studies proved the benefits of different types of SCs in experimental models of pulmonary emphysema (PE).

G. Zhen et al. have shown that the transplantation of mesenchymal stem cells (MSCs) to rats with papain-induced emphysema leads to their migration into the lungs, differentiation into type 2 alveolocytes, and inhibition of apoptosis and prevention PE.

K. Schweitzer et al. have proved the activity of inflammation in the airways, alveolocytes and endothelial cells apoptosis decreased after adipose SCs intravenous administration to mice with emphysema caused by chronic exposure to tobacco smoke or VEGF receptors blockade. The study of E.P. Ingenito et al. found that endobronchial installed MSCs engraft into the alveolar wall and peribronchial interstitium and release integrins, extracellular matrix components (collagen IV, laminin and fibrillin), platelet-derived growth factor receptor and transforming growth factor β2.

Our study also found reliable deterrent effect of allogeneic bone marrow MSCs on the development of elastase-induced emphysema in rats at different terms of transplantation.

After the success of pilot studies have started clinical trials. Currently, the website http://www. ClinicalTrials.gov reported three studies evaluating the efficacy and safety of MSC transplantation in patients with COPD and emphysema. Two of them have already been completed and the results of the first pilot project published.

Authors on the example of 4 patients showed a complete absence of adverse effects, improved quality of life and stability of functional parameters at 12 months after starting treatment One of the problems of MSC transplantation in patients with respiratory failure is an accelerated apoptosis of transplanted cells under the influence of proinflammatory cytokines and oxidative stress. Since it is proved that preconditioning MSCs under hypoxia increases their survival in hypoxic conditions, increases the expression of growth factors and antiinflammatory cytokines, we suppose that MSCs grown in hypoxic medium may have a significant positive effect on the disease.

Detailed Description

Not available

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  • HRCT-confirmed diagnosis of lung emphysema by two independent radiologists
  • post-bronchodilator FEV1/FVC ratio < 0.7
  • post-bronchodilator FEV1 % predicted value ≥ 20% and < 50%
  • age 35 and 75 years of, of either sex, and of any race
  • current or ex-smoker, with a cigarette smoking history ≥ 10 pack-years
Exclusion Criteria
  • • asthma or other clinically relevant lung disease other than COPD (e.g. restrictive lung diseases, sarcoidosis, tuberculosis, idiopathic pulmonary fibrosis, or lung cancer)

    • α1-Antitrypsin deficiency
    • Presence of bullae (more than 10 cm in the diameter)
    • active infection within 4 weeks of screening
    • significant exacerbation of COPD or has required mechanical ventilation within 4 weeks of screening
    • clinically relevant uncontrolled medical condition not associated with COPD
    • documented history of uncontrolled heart failure
    • pulmonary hypertension due to left heart condition
    • Subject has evidence of active malignancy, or prior history of active malignancy
    • Subject has a life expectancy of < 6 months

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
MSC groupMesenchymal stem cellsIntravenous infusion of MSC suspension, pre-conditioned under 1% oxygen, in the amount of 200 mln. cells per 400 mL of sodium chloride physiological solution. Infusions will be performed every 2 months for 1 year
Primary Outcome Measures
NameTimeMethod
Safety compared with placebo1 year

Mortality (Baseline and 2 years after procedure) Adverse effects and reactions to the treatment(Baseline and 2 years after procedure).

Vital signs (pulse rate, systolic and diastolic arterial blood pressure) (Baseline and 2 years after procedure)

Secondary Outcome Measures
NameTimeMethod
Dynamics of the physical capacity (by the 6-min test results)2 years
Dynamics of the blood gas composition (PaO2, PaCO2)2 years
DLCO change from baseline at 6, 12, 24 months2 years
Change from baseline in the lung tissue density measured by CT-densitometry at6, 12, 24 months2 years
Dynamics of serum level IL-6, TNF-α, Leptin2 years
Number and frequency of exacerbations2 years
Body mass index2 years
Change from baseline in the functional parameters (FEV1, TLC, RV, FEV1/FVC) at 6,12,18,24 months2 years
Quality of life indices by the questionnaire (SF-36)2 years

Trial Locations

Locations (1)

Federal Research Clinical Center of Federal Medical and Biological Agency of Russia

🇷🇺

Moscow, Moscow Region, Russian Federation

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