Clinical trial NCT02594839: Safety and Efficacy of Allogeneic Mesenchymal Stem Cells in Patients With Rapidly Progressive Interstitial Lung Disease
Official title: A Phase I-II Study to Evaluate Safety and Efficacy of Allogeneic Bone-Marrow Mesenchymal Stem Cells in Patients With Rapidly Progressive Interstitial Lung Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Idiopathic Interstitial Pneumonia
Interstitial Lung Disease
Idiopathic Pulmonary Fibrosis
Keywords: interstitial lung disease
idiopathic interstitial pneumonia
idiopathic pulmonary fibrosis
stem cells
Lead sponsor: Federal Research Clinical Center of Federal Medical & Biological Agency, Russia
Sponsor class: OTHER_GOV
First posted: 2015-11-03
Enrollment: 20
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The study evaluates the safety and the efficacy of the addition of intravenous transplantation of donor bone marrow mesenchymal stem cells in patients with idiopathic interstitial pneumonia or connective tissue disease associated with interstitial lung disease, which have actively progressing disease with rapid loss of pulmonary function on the background of routine treatment.

Detailed description: Despite significant progress in the treatment of interstitial lung disease, achieved thanks to new drugs, such as pirfenidone and nintedanib, there are many patients for whom these drugs are not available or poorly tolerated. In addition significant evidence of their effectiveness and safety is valid only for idiopathic pulmonary fibrosis. The transplantation of allogeneic stem cells is a promising direction in the modern medicine with the proven safety for different diseases. But the effectiveness of this therapy is still under research. We believe that in most severe cases of a rapidly progressive interstitial lung disease the transplantation of mesenchymal stem cells may be an effective technology due to their immunomodulatory properties.

Eligibility criteria: Inclusion Criteria:

* Male and female patients 20-80 years old
* Diagnosis of idiopathic interstitial pneumonia or secondary to connective tissue diseases interstitial lung disease, based on:

  * Clinical symptoms \> 12 months duration,
  * Histologically diagnosed or diagnostic chest HRCT features of interstitial pneumonia
* Forced Vital Capacity (FVC) ≥ 40% predicted and Diffusing Lung Capacity (DLCO) ≥20%
* Loss more than 10% of FVC (L) and DLCO during the last 12 months
* Signed informed consent.

Exclusion Criteria:

* Diagnosis of an interstitial lung disease other than idiopathic interstitial pneumonia or connective tissue disease associated with interstitial lung disease (sarcoidosis, pulmonary alveolar proteinosis, lymphangioleiomyomatosis, pulmonary amyloidosis, exposure-related lung disease etc)
* Obstructive lung disease: FEV1/FVC \< 0.70
* Clinically significant medical condition, in the opinion of the investigator, may compromise the results of the study
* Evidence of active infection within 4 week prior to enrollment
* History of malignancy \< 5 years prior to enrollment
* Unable to cooperate with any study procedures
* Pregnant or breast-feeding
* Treatment with antiinflammatory or antifibrotic drugs including oral steroids, cytostatic drugs, pirfenidone, D penicillamine, colchicine, tumor necrosis factor α blockers, imatinib, interferon γ, monoclonal antibodies \< 6 months prior to randomization.
* Active listing for transplant of any organ.
* Known history of alcohol abuse within 1 year prior to enrollment
* Participation in another clinical trial

Primary outcomes:
measure: Safety: Number of serious adverse events
timeFrame: 12 months
Secondary outcomes:
measure: DLCO changes from baseline
timeFrame: 12 months
measure: FVC changes from baseline
timeFrame: 12 months
measure: exercise capacity changes
timeFrame: 12 months
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Federal Research Clinical Center FMBA of Russia
Study officials:
References: