Chronic and Accelerated SilicosisPhase 1SilicStemCellNCT01239862
Safety of Stem Cells Intrabronchial Instillation for Silicosis
A Phase 1 study testing Autologous cell transplantation in people with Chronic and Accelerated Silicosis, run by Universidade Federal do Rio de Janeiro at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Chronic and Accelerated Silicosis
- What is being tested?
- Autologous cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteBrazil
- Study dates
- Aug 2009to Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
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
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
Rio de Janeiro, Rio de Janeiro, 21949900
About this study
Official title: Phase-1 Study of Autologous Bone Marrow Cells Intrabronchial Instillation for Patients Silicosis
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.
Read the 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.
- Conditions
- Chronic and Accelerated Silicosis
- Being tested
- Autologous cell transplantation
- Keywords
- pulmonary, silicosis, chronic and accelerated silicosis, autologous transplantation, bone marrow cells, stem cells, cell therapy
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Silicosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous cell transplantationOther
Intrabronchial Instillation Of Bone Marrow Derived Mononuclear Cells
| Study group | Role | What they receive |
|---|---|---|
| Autologous cell transplantation | Experimental | We conducted a prospective, non-randomized, single-center longitudinal study in five patients. Inclusion criteria were age 18-50 years, chronic and accelerated silicosis, forced expiratory volume in 1s <60% and >40%, forced vital capacity ≥60% and arterial oxygen saturation >90%. BMDMCs were administered through bronchoscopy (2×107 cells) into both lungs. Physical examination, laboratory evaluations, quality of life questionnaires, thoracic computed tomography scans, lung function tests, and perfusion scintigraphy were performed before the beginning of treatment and up to 360 days after BMDMC (Bone Marrow Derived Mononuclear Cells) therapy. Additionally, whole-body and planar scans were evaluated 2 and 24 h after instillation. Receives: Other: Autologous cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absence of lung deficits during the procedure and/or in the 4 months follow-up
Measured: 4 months
Secondary outcomes (1)
Improvement of pulmonary deficits
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2010 (estimated)
- Study startAug 2009
- Primary completionMay 2011
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic and Accelerated SilicosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universidade Federal do Rio de Janeiro
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Science and Technology, Brazil, Ministry of Health, Brazil, National Research Council, Brazil
- Responsible party
- Principal investigator, Marcelo Marcos Morales, Universidade Federal do Rio de Janeiro
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01239862
- Sponsor's protocol ID
- CONEP5772008
- Acronym
- SilicStemCell
- Status last verified
- May 2015
- First submitted
- 10 Nov 2010
- Last updated
- 27 May 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 26059242
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase-1 Study of Autologous Bone Marrow Cells Intrabronchial Instillation for Patients Silicosis
- ClinicalTrials.gov ID
- NCT01239862
- Acronym / sponsor ID
- SilicStemCell / CONEP5772008
- Conditions
- Chronic and Accelerated Silicosis
- Keywords
- pulmonary, silicosis, chronic and accelerated silicosis, autologous transplantation, bone marrow cells, stem cells, cell therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Universidade Federal do Rio de JaneiroUniversity, hospital or other
- Collaborators
- Ministry of Science and Technology, Brazil, Ministry of Health, Brazil, National Research Council, Brazil
- Study start
- Aug 2009
- Primary completion
- May 2011 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 11 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous cell transplantation | Experimental | Other: Autologous cell transplantationWe conducted a prospective, non-randomized, single-center longitudinal study in five patients. Inclusion criteria were age 18-50 years, chronic and accelerated silicosis, forced expiratory volume in 1s <60% and >40%, forced vital capacity ≥60% and arterial oxygen saturation >90%. BMDMCs were administered through bronchoscopy (2×107 cells) into both lungs. Physical examination, laboratory evaluations, quality of life questionnaires, thoracic computed tomography scans, lung function tests, and perfusion scintigraphy were performed before the beginning of treatment and up to 360 days after BMDMC (Bone Marrow Derived Mononuclear Cells) therapy. Additionally, whole-body and planar scans were evaluated 2 and 24 h after instillation. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous cell transplantationIntrabronchial Instillation Of Bone Marrow Derived Mononuclear Cells | Other | Autologous cell transplantation |
Primary outcome
Absence of lung deficits during the procedure and/or in the 4 months follow-up
Time frame: 4 months
1 secondary outcome
Improvement of pulmonary deficits
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2015.
- Derived from this study: PubMed 26059242Morales 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.
Related trials for Chronic and Accelerated Silicosis
Not yet recruitingObservational study
Perioperative Risk Factors Related to the Prognosis of Lung Transplantation: A Retrospective Study
COPD, Interstitial Pneumonia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous cell transplantation in people with Chronic and Accelerated Silicosis, run by Universidade Federal do Rio de Janeiro at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Absence of lung deficits during the procedure and/or in the 4 months follow-up (time frame: 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01239862, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2015.