Rationale: Pulmonary emphysema is a component of Chronic Obstructive Pulmonary Disease (COPD)
characterized by chronic inflammation with neutrophils and monocytes mediating the tissue
destruction under the regulation of various types of lymphocytes. Bone marrow-derived
mesenchymal stromal cells have potential to halt the progressive inflammatory response as
indicated by the investigator's pilot study (CCMO NL28562.000.09) .
Objective: To determine whether patients with emphysema develop anti-inflammatory and tissue
repair responses by treatment with allogeneic bone marrow-derived mesenchymal stromal cells
(MSC) from healthy donors.
Study design: an explorative double-blind, placebo-controlled randomized (2:1) trial in 30
patients with moderate to severe emphysema who are scheduled for two separate sessions for
surgical lung volume reduction (LVRS). The study treatment is intravenous allogeneic MSC or
placebo treatment in between the first and second surgical session. Randomisation will
allocate 10 patients to receive 2 x 106 /kg body weight MSC in a range of 1.5 x 106 MSC/ kg
to 2.5 x 106 MSC/ kg (at a maximum of 200 x106 MSC per study participant) iv (or 5 patients
to receive placebo) at week 4 and 3 before the second LVRS, and will allocate 10 patients to
receive 2 x 106 /kg body weight MSC in a range of 1.5 x 106 MSC/ kg to 2.5 x 106 MSC/ kg (at
a maximum of 200 x106 MSC per study participant) iv (or 5 patients to placebo) at week 12 and
11 before the second LVRS.
Main study parameters/endpoints: the study has a co-primary endpoint. First, the difference
in expression of CD31 on cells per micrometer alveolar septae present in lung tissue
harvested at the second LVRS from patients who received MSC at 3 and 4 weeks prior to LVRS2
or placebo. Second, the difference between MSC and placebo treatment in change in CO
diffusion capacity over a period of 3 years following LVRS2.
Phase:
Phase 2
Details
Lead Sponsor:
Leiden University Medical Center
Collaborators:
Erasmus Medical Center VU University Medical Center