Clinical trial NCT01239862: Safety of Stem Cells Intrabronchial Instillation for Silicosis
Official title: Phase-1 Study of Autologous Bone Marrow Cells Intrabronchial Instillation for Patients Silicosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Chronic and Accelerated Silicosis
Keywords: pulmonary, silicosis, chronic and accelerated silicosis, autologous transplantation
bone marrow cells, stem cells, cell therapy
Lead sponsor: Universidade Federal do Rio de Janeiro
Sponsor class: OTHER
Collaborators: name: Ministry of Science and Technology, Brazil
name: Ministry of Health, Brazil
name: National Research Council, Brazil
First posted: 2010-11-11
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The aim of this study was to analyze the safety, pulmonary function, and quality of life data of patients with silicosis treated with intrabronchial instillation of bone marrow derived mononuclear cells (BMDMC, 2x107) through bronchoscopy.

Detailed description: This study will perfume the safety (Phase I) study of 10 patients with silicosis treated with intrabronchial instillation of autologous bone marrow derived mononuclear cells (BMDMC, 2x107) through bronchoscopy. The inclusion criteria is: age between 18-50, chronic and accelerated silicosis, characterized with a fibrotic increase in the last two years, FEV1 \<60% and \> 40%, FVC \> 60% and SaO2 \>90%, while the exclusion criteria were: smoking, active tuberculosis or other infections, cancer, auto-immune disorders, hematological, hepatic or cardiac diseases, and pregnancy. All patients will be subjected to clinical examination, answered questionnaires of quality of life (SGRQ and SF36) and dyspnea score (Borg), performed high resolution CT of thorax, pulmonary function tests with DLCO and 6-minute walk test and lung perfusion scintigraphy before and 7, 30, 60, 180 and 360 days after treatment.

For each patient, 2×107 cells will be labeled with 99mTc. Briefly, 500 μl of sterile SnCl2 solution is added to the cells and the mixture is incubated at room temperature for 10 min. Forty-five mCi of 99mTc is then added and incubation continued for another 10 min. After centrifugation (500×g for 5 min), the supernatant is removed and the cells are washed in saline solution. The pellet was also resuspended in saline solution. Viability of the labeled cells was assessed by the trypan blue exclusion test, and was estimated to be greater than 93% in all cases. Labeling efficiency (%) was calculated by the activity in the pellet divided by the sum of the radioactivity in the pellet plus supernatant and was estimated to be greater than 90% in all cases. Whole body and planar scans will be performed 2 and 24h after instillation. Perfusion scintigraphy will be performed before and 30, 60, 120 and 180 days after BMDMC therapy. For regional analysis the right and left lungs will be divided into three zones: base, midzone, and apex.

Eligibility criteria: Inclusion Criteria:age between 18-50, chronic and accelerated silicosis, characterized with a fibrotic increase in the last two years, FEV1 \<60% and \> 40%, FVC \> 60% and SaO2 \>90%

Exclusion Criteria: smoking, active tuberculosis or other infections, cancer, auto-immune disorders, hematological, hepatic or cardiac diseases, and pregnancy

Primary outcomes:
measure: Absence of lung deficits during the procedure and/or in the 4 months follow-up
timeFrame: 4 months
Secondary outcomes:
measure: Improvement of pulmonary deficits
timeFrame: 1 year
Other outcomes:
Locations:
city: Rio de Janeiro
country: Brazil
facility: Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
state: RJ
Study officials:
References:
citation: Morales MM, Souza SA, Loivos LP, Lima MA, Szklo A, Vairo L, Brunswick TH, Gutfilen B, Lopes-Pacheco M, Araujo AJ, Cardoso AP, Goldenberg RC, Rocco PR, Fonseca LM, Lapa e Silva JR. Pilot safety study of intrabronchial instillation of bone marrow-derived mononuclear cells in patients with silicosis. BMC Pulm Med. 2015 Jun 11;15:66. doi: 10.1186/s12890-015-0061-8.