Focal Segmental GlomerulosclerosisPhase 1STEFOGNCT02693366
Stem Cell Therapy for Patients With Focal Segmental Glomerulosclerosis
A Phase 1 study testing Bone marrow stem cell in people with Focal Segmental Glomerulosclerosis and Chronic Kidney Diseases, run by Universidade Federal do Rio de Janeiro at 1 site in Brazil.
Trial summary
The purpose of this study is to analyze the safety, renal function, metabolic disorders and quality of life data in patients with focal segmental glomerulosclerosis treated with endovascular infusion of bone marrow derived mononuclear cells. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Focal Segmental Glomerulosclerosis, Chronic Kidney Diseases
- What is being tested?
- Bone marrow stem cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteBrazil
- Study dates
- 25 Jun 2015to 16 May 2018
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with diagnosis of primary focal segmental glomerulosclerosis after having been previously treated with corticosteroids and immunosuppressive drugs and have not reached satisfactory answer. Will also be considered candidates those patients who performed late diagnosis and therefore no more clinical indication to perform therapy with corticosteroids and immunosuppressants. In both cases, showing irreversible loss of renal function with filtration rate between 40 - 20 ml/min.
- Patient should use the classical nephroprotective medication: angiotensin-converting enzyme inhibitor or angiotensin-receptor blocker, or both.
Who cannot join
- Acute urinary tract infection;
- Urinary infection with tuberculosis bacillus or fungi;
- Patients with poorly anatomical formations of the urinary tract, polycystic kidney disease and other congenital or acquired kidney diseases.
- Blood pressure greater than 160 mm Hg systolic and 100 mmHg diastolic, in measurements taken during the last 3 outpatient visits;
- Who has performed examination with iodinated contrast the last 3 months
- Use of potentially nephrotoxic drugs;
Show all 24Show fewer
- Use of corticosteroid therapy in immunosuppressive doses or more than 0.3 mg/kg/day
- Inability to obtain vascular access for endovascular procedure
- Sepsis (defined according to the Society of Critical Care Medicine, American College of Chest Physicians, 1992);
- Malignancies
- Autoimmune disorders,
- Neurodegenerative diseases;
- Acute heart failure or decompensated;
- Primary hematologic diseases;
- Osteopathies reflecting increased risk for spinal puncture;
- Coagulopathies;
- Liver failure;
- History of stroke or myocardial infarction in the last 6 months;
- Pregnancy or breastfeeding;
- History and serology of chronic infectious diseases, including HIV, Hepatitis C virus, Hepatitis B virus
- Participation in another clinical trial last year
- Cognitive impairment to understand all procedures
- Prolonged travel plans or domicile changes to other states that generate unable to attend the follow-up visits;
- Any other clinically significant active disease in the opinion of the principal investigator
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Universitary Hospital Clementino Fraga Filho - UFRJ
Rio de Janeiro, 21941913
About this study
Official title: Safety Study of the Endovascular Infusion of Bone Marrow Derived Mononuclear Cells in Patients With Focal Segmental Glomerulosclerosis
The purpose of this study is to analyze the safety, renal function, metabolic disorders and quality of life data in patients with focal segmental glomerulosclerosis treated with endovascular infusion of bone marrow derived mononuclear cells.
Read the detailed description
Will be studied five patients with progressive chronic kidney disease and estimated clearance between 40 and 20 ml / min. Patients will be followed by clinical and laboratory examination for 3 months prior to the procedure. These previous results serve as a control for comparison with a second time when the same patients receive treatment with stem cells being subsequently followed up for 9 months a total of one year of clinical follow-up.
Bone marrow aspiration and subsequent cell preparation were accomplished on the same day as the endovascular infusion of autologous Bone Marrow derived Mononuclear stem cells (BMDMCs) in both renal arteries. Collection was performed under spinal anesthesia and light sedation, through puncture and repeated aspirations at the posterior iliac crest region. A total of 80 mL of bone marrow aspirate was collected from each patient, and after removal of bone and fatty residues, mononuclear cells were isolated by a Ficoll-Paque Plus (Amersham Biosciences, São Paulo, Brazil).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 millicurie (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 will be also resuspended in saline solution. Viability of the labeled cells will be assessed by the trypan blue exclusion test, and estimated to be greater than 93% in all cases.The labeling efficiency (%) will be calculated by the activity in the pellet divided by the sum of the radioactivity in the pellet plus supernatant and estimated to be greater than 90% in all cases.
After the collection of the stem cells, the patient will be submitted to puncture the femoral artery using the Seldinger technique under local anesthesia, followed by catheterization of the ostium of the renal arteries with minimum use of nonionic iodinated contrast. With the routing of diagnostic catheter or guide, the solution numbering about 30 to 100 million of dissolved plasma cells will be divided and injected into two renal arteries. The infusion volume is about 5 ml in each kidney. Whole body and planar scans will be performed 2 and 24h after infusion to determine the migration and cell viability. The patient will remain hospitalized for more 48 hours for clinical monitoring and collection of laboratorial tests.
- Being tested
- Bone marrow stem cell
- Keywords
- bone marrow cells, Focal Segmental Glomerulosclerosis, Chronic Kidney Diseases, stem cell, cell therapy
- Medical subject headings
- Glomerulosclerosis, Focal Segmental, Renal Insufficiency, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone marrow stem cellOther
Endovascular infusion of bone marrow derived cells in both renal arteries.
| Study group | Role | What they receive |
|---|---|---|
| Autologous Cell Therapy | Experimental | We are conducting a prospective, non-randomized, single-center longitudinal study in five patients with progressive chronic kidney disease and estimated clearance between 40 and 20 ml / min. Patients will be followed by clinical and laboratory examination for 3 months prior to the procedure. These previous results serve as a control for comparison with a second time when the same patients receive treatment with stem cells being subsequently followed up for 9 months a total of one year of clinical follow-up. Receives: Other: Bone marrow stem cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Kidney injury
Increase of serum creatinine of about 0.5 mg / dL when levels are less than 3.0 mg / dl and 1.0 mg / dl baseline levels when are greater than or equal to 3.0 mg / dL) when confirmed with the second examination.
- Acute: evaluated within 15 days of cell therapy;
- Subacute: evaluated 15-90 days of cell therapy
Measured: 9 months
Chronic kidney disease
Doubling of serum creatinine based on the third month after the cell therapy or the need to start dialysis
Measured: 9 months
Potential differentiation disorders of transplanted cells
Analyzed by clinical and imaging tests such abdominal ultrasound and chest radiography
Measured: 9 months
Systemic inflammatory potential of mononuclear cells administration in renal circulation
Laboratory tests: C-reactive protein, erythrocyte sedimentation rate, blood count and urinary sediment
Measured: 9 months
Death
Measured: 9 months
Secondary outcomes (6)
Renal function
The estimated creatinine clearance assessment by MDRD formula
Measured: 9 months
Bone metabolism
Evaluation of bone metabolism by serum phosphorus (mg/dL), calcium (mg/dL), parathormone (pg/ml), 25 (OH) vit. D (ng/ml).
Measured: 9 months
Balance assessment electrolyte and acid-base
Balance assessment electrolyte and acid-base by serum sodium (mEq/l), potassium (mEq/l), uric acid (mg/dl) and bicarbonate
Measured: 9 months
The lipid profile assessment and anemia
The lipid profile assessment (LDL- cholesterol, HDL-cholesterol and triglyceride) and anemia measured by hemoglobin (g/dL) and hematocrit.
Measured: 9 months
Quality of life questionnaire
Clinical improvement of the patient, with subjective assessment of general health and well being through SF36 quality of life questionnaire
Measured: 9 months
Imaging tests
Imaging tests: Renal scintigraphy with 99mTc-DTPA and DMSA
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2016 (estimated)
- Study start25 Jun 2015
- Primary completionFeb 2018
- Study completion16 May 2018
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 Focal Segmental GlomerulosclerosisStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
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, Rio de Janeiro State Research Supporting Foundation (FAPERJ)
- Responsible party
- Principal investigator, Marcelo Marcos Morales, Universidade Federal do Rio de Janeiro
- Investigators
- Marcelo Marcos Morales, MD,PHD, Principal investigator, Universidade Federal do Rio de Janeiro
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02693366
- Sponsor's protocol ID
- CAAE:01498412.5.0000.5257
- Acronym
- STEFOG
- Status last verified
- Jan 2020
- First submitted
- 30 Jan 2016
- Last updated
- 28 Jan 2020
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Botelho BF, Barreira AL, Filippo MG, Asensi KD, Faccioli LAP, Dos Santos Salgado AB, de Salles EF, Marques CEC, Silva PL, Dos Santos Goldenberg RC, Maiolino A, Gutfilen B, de Souza SAL, Junior ML, Morales MM. Safety and Biodistribution of an Autologous Bone Marrow-Derived Mononuclear Cell Infusion into Renal Arteries in Patients with Focal Segmental Glomerulosclerosis: A Phase 1 Study. Stem Cells Int. 2024 Jul 9;2024:2385568. doi: 10.1155/2024/2385568. eCollection 2024.
PubMed 39015674
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Study of the Endovascular Infusion of Bone Marrow Derived Mononuclear Cells in Patients With Focal Segmental Glomerulosclerosis
- ClinicalTrials.gov ID
- NCT02693366
- Acronym / sponsor ID
- STEFOG / CAAE:01498412.5.0000.5257
- Keywords
- bone marrow cells, Focal Segmental Glomerulosclerosis, Chronic Kidney Diseases, stem cell, cell therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (estimated)
- 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, Rio de Janeiro State Research Supporting Foundation (FAPERJ)
- Study start
- 25 Jun 2015 (actual)
- Primary completion
- Feb 2018 (actual)
- Study completion
- 16 May 2018 (actual)
- First posted
- 26 Feb 2016 (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 purpose of this study is to analyze the safety, renal function, metabolic disorders and quality of life data in patients with focal segmental glomerulosclerosis treated with endovascular infusion of bone marrow derived mononuclear cells.
Detailed description
Will be studied five patients with progressive chronic kidney disease and estimated clearance between 40 and 20 ml / min. Patients will be followed by clinical and laboratory examination for 3 months prior to the procedure. These previous results serve as a control for comparison with a second time when the same patients receive treatment with stem cells being subsequently followed up for 9 months a total of one year of clinical follow-up.
Bone marrow aspiration and subsequent cell preparation were accomplished on the same day as the endovascular infusion of autologous Bone Marrow derived Mononuclear stem cells (BMDMCs) in both renal arteries. Collection was performed under spinal anesthesia and light sedation, through puncture and repeated aspirations at the posterior iliac crest region. A total of 80 mL of bone marrow aspirate was collected from each patient, and after removal of bone and fatty residues, mononuclear cells were isolated by a Ficoll-Paque Plus (Amersham Biosciences, São Paulo, Brazil).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 millicurie (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 will be also resuspended in saline solution. Viability of the labeled cells will be assessed by the trypan blue exclusion test, and estimated to be greater than 93% in all cases.The labeling efficiency (%) will be calculated by the activity in the pellet divided by the sum of the radioactivity in the pellet plus supernatant and estimated to be greater than 90% in all cases.
After the collection of the stem cells, the patient will be submitted to puncture the femoral artery using the Seldinger technique under local anesthesia, followed by catheterization of the ostium of the renal arteries with minimum use of nonionic iodinated contrast. With the routing of diagnostic catheter or guide, the solution numbering about 30 to 100 million of dissolved plasma cells will be divided and injected into two renal arteries. The infusion volume is about 5 ml in each kidney. Whole body and planar scans will be performed 2 and 24h after infusion to determine the migration and cell viability. The patient will remain hospitalized for more 48 hours for clinical monitoring and collection of laboratorial tests.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Cell Therapy | Experimental | Other: Bone marrow stem cellWe are conducting a prospective, non-randomized, single-center longitudinal study in five patients with progressive chronic kidney disease and estimated clearance between 40 and 20 ml / min. Patients will be followed by clinical and laboratory examination for 3 months prior to the procedure. These previous results serve as a control for comparison with a second time when the same patients receive treatment with stem cells being subsequently followed up for 9 months a total of one year of clinical follow-up. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Patients with diagnosis of primary focal segmental glomerulosclerosis after having been previously treated with corticosteroids and immunosuppressive drugs and have not reached satisfactory answer. Will also be considered candidates those patients who performed late diagnosis and therefore no more clinical indication to perform therapy with corticosteroids and immunosuppressants. In both cases, showing irreversible loss of renal function with filtration rate between 40 - 20 ml/min.
- Patient should use the classical nephroprotective medication: angiotensin-converting enzyme inhibitor or angiotensin-receptor blocker, or both.
Key exclusion criteria (24 listed)
- Acute urinary tract infection;
- Urinary infection with tuberculosis bacillus or fungi;
- Patients with poorly anatomical formations of the urinary tract, polycystic kidney disease and other congenital or acquired kidney diseases.
- Blood pressure greater than 160 mm Hg systolic and 100 mmHg diastolic, in measurements taken during the last 3 outpatient visits;
- Who has performed examination with iodinated contrast the last 3 months
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 |
|---|---|---|
| Bone marrow stem cellEndovascular infusion of bone marrow derived cells in both renal arteries. | Other | Autologous Cell Therapy |
Primary outcome
Kidney injury
Time frame: 9 months
Increase of serum creatinine of about 0.5 mg / dL when levels are less than 3.0 mg / dl and 1.0 mg / dl baseline levels when are greater than or equal to 3.0 mg / dL) when confirmed with the second examination. * Acute: evaluated within 15 days of cell therapy; * Subacute: evaluated 15-90 days of cell therapy
Chronic kidney disease
Time frame: 9 months
Doubling of serum creatinine based on the third month after the cell therapy or the need to start dialysis
Potential differentiation disorders of transplanted cells
Time frame: 9 months
Analyzed by clinical and imaging tests such abdominal ultrasound and chest radiography
Systemic inflammatory potential of mononuclear cells administration in renal circulation
Time frame: 9 months
Laboratory tests: C-reactive protein, erythrocyte sedimentation rate, blood count and urinary sediment
Death
Time frame: 9 months
6 secondary outcomes
Renal function
Time frame: 9 months
Bone metabolism
Time frame: 9 months
Balance assessment electrolyte and acid-base
Time frame: 9 months
The lipid profile assessment and anemia
Time frame: 9 months
Quality of life questionnaire
Time frame: 9 months
Imaging tests
Time frame: 9 months
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 28 Jan 2020.
- Derived from this study: PubMed 39015674Botelho BF, Barreira AL, Filippo MG, Asensi KD, Faccioli LAP, Dos Santos Salgado AB, de Salles EF, Marques CEC, Silva PL, Dos Santos Goldenberg RC, Maiolino A, Gutfilen B, de Souza SAL, Junior ML, Morales MM. Safety and Biodistribution of an Autologous Bone Marrow-Derived Mononuclear Cell Infusion into Renal Arteries in Patients with Focal Segmental Glomerulosclerosis: A Phase 1 Study. Stem Cells Int. 2024 Jul 9;2024:2385568. doi: 10.1155/2024/2385568. eCollection 2024.
- Individual participant dataSharing planned: Undecided
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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.
Marcelo Marcos Morales, MD,PHD principal investigator
Universidade Federal do Rio de Janeiro
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 Bone marrow stem cell in people with Focal Segmental Glomerulosclerosis and Chronic Kidney Diseases, 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?
The sponsor lists 2 inclusion and 24 exclusion criteria. 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 28 Jan 2020. 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?
Kidney injury (time frame: 9 months). The study lists 5 primary outcomes in all. 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 NCT02693366, 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 28 Jan 2020.