Focal Segmental GlomerulosclerosisPhase 1NCT02382874
Allogenic AD-MSC Transplantation in Idiopathic Nephrotic Syndrome (Focal Segmental Glomerulosclerosis)
A Phase 1 study testing Intravenous injection in people with Focal Segmental Glomerulosclerosis, run by Royan Institute at 1 site in Iran.
Trial summary
Idiopathic Focal Segmental Glumero Sclerosis (FSGS) is not very common but important manifestation of kidney disease. FSGS has poor prognosis among the patterns of Idiopathic Nephrotic Syndrome (INS). Read more about this study
- Who is it for?
- Ages 2 to 14 years
- Conditions the study is for
- Focal Segmental Glomerulosclerosis
- What is being tested?
- Intravenous injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteIran
- Study dates
- May 2015to Oct 2017
- 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 2 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 2-14 years at onset of signs or symptoms of FSGS 2- Primary FSGS involving either native kidneys or primary FSGS recurring after renal transplantation. Biopsy confirmed as primary FSGS (including all subtypes).
- 3-Estimated GFR ≥ 25 ml/min/1.73 m2 4- Up/c > 1.0 (g protein/g creatinine) on first am void 5- Steroid and IS resistance as defined by primary physician (patients with little or no reduction in protein excretion at 12 to 16 weeks are considered to be steroid resistant --A relapse is return of proteinuria to ≥3.5 g/day in someone who had undergone a complete or partial remission- IS resistance: no response to IS in 8-12 weeks) 6-At least one month from last immunization received has passed 7- Ability to understand and willingness to sign consent by patient legal guardian
Who cannot join
- 1- Are immunodeficient or have clinically significant chronic lymphopenia 2-Have an active infection or positive PPD test result 3-Have serologic evidence of current or past HIV, Hepatitis B, or Hepatitis C infection 4-Have any complicating medical issues that interfere with study conduct or cause increased risk such as malignancies, systemic autoimmune disease, diabetes, blood disease, liver disease, etc.
- 5- Positive genetic mutation testing for WT1, Podocin, Nephrin
Trial locations and contacts
1 site in 1 country.
Iran1 site, 1 recruiting
- Recruiting
Royan Institute
Tehran
Nasser Aghdami, MD,PhD, (+98)2123562000 ext. 516, nasser.aghdami@royaninstitute.org
Leila Arab, MD, (+98)2123562000 ext. 414, Leara91@gmail.com
About this study
Official title: Allogenic Adipose Derived Mesenchymal Stromal Cells Transplantation to Improve Kidney Function in Refractory Primary Nephrotic Syndrome (Focal Segmental Glomerulosclerosis,FSGS) ,a Phase I Clinical Trial
Idiopathic Focal Segmental Glumero Sclerosis (FSGS) is not very common but important manifestation of kidney disease.
FSGS has poor prognosis among the patterns of Idiopathic Nephrotic Syndrome (INS).
Even with treatment (Steroid therapy and cytotoxic immunosuppressant therapy), many patients eventually still require dialysis.
Cell therapy is useful in treatment of INS and mesenchymal stromal/stem cell is one of the cells that useful in the treatment of glomerulus disease.
Intravenous injection of allogeneic adipose derived mesenchymal stromal/stem cell will be done in 5 patients with refractory INS(FSGS). They will be followed 1, 2, 4 weeks and then monthly until a year following injection day.
Read the detailed description
Primary FSGS thought to be a part of the immune-mediated disease. Main challenge is to decrease protein excretion in urine. Only 30% of children with FSGS achieve complete remission with steroids and other 30-40% patients experience remission (partial and complete) with cytotoxic immunosuppressant drugs. Even with these treatments, many patients eventually still require dialysis. Other treatment strategy doesn't exceed beyond symptomatic treatment and delaying the progression. Also risk of recurrence after kidney transplantation is 20-50%.
Cell therapy is one of the treatment strategies and mesenchymal stromal/stem cell is one modality that notice in glomerulus disease.
We will evaluate safety and efficacy of intravenous injection of allogeneic AD-MSC (adipose-derived mesenchymal stromal cell) in 5 refractory INS patients.They will be followed 1, 2, 4 weeks and then monthly until a year following injection day.
- Conditions
- Focal Segmental Glomerulosclerosis
- Being tested
- Intravenous injection
- Keywords
- Idiopathic nephrotic syndrome, adipose derived mesenchymal stromal cell, Focal segmental glomerulosclerosis
- Medical subject headings
- Glomerulosclerosis, Focal Segmental, Nephrosis, Lipoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous injectionBiological
Intravenous injection of AD-MSC to the patients with FSGS
| Study group | Role | What they receive |
|---|---|---|
| AD-MSC | Experimental | The patients with FSGS who underwent intravenous injection of AD-MSC. Receives: Biological: Intravenous injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Liver function
increase of liver enzymes 2 weeks after cell injection.
Measured: 2 weeks
Serum creatinine
Decrease of serum creatinine 2 weeks after cell injection.
Measured: 2 weeks
Proteinuria
Reduction in proteinuria to <200 to 300 mg/day will be assessed by Changes in 24 hour urine protein analysis.
Measured: 12 hour
Secondary outcomes (3)
Renal function
will be assessed by change in serum levels of Cr, Urea and GFR. Time Frame: 12 hr after injection,1,2 weeks then monthly until a year after 1st injection.
Measured: 12 hours
Increase in anti inflammatory factors
It will be assessed by change in serum levels of IL-2, 10. Time frame: 12 hour after injection then monthly until a year 1st injection.
Measured: 12 hours
Increase in Treg
It will be assessed by change in serum levels of Treg. Time frame: 12 hr after injection,1 week, 3, 6,12 months after 1st injection.
Measured: 12hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2015 (estimated)
- Study startMay 2015
- Primary completionJun 2017 (estimated)
- Study completionOct 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Royan Institute
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Hamid Gourabi, PhD, Study chair, Head of Royan InstituteNasser Aghdami, MD,PhD, Study director, Head of department of regenerative medicine&cell therapy center,Royan InstituteHassan Otukesh, MD, Study director, Department Of Pediatric Nephrology and Dialysis, Ali aghar Children Hospital, Iran University of Medical Sciences, Tehran, IranRozita Hosseini, MD, Principal investigator, Department Of Pediatric Nephrology and Dialysis, Ali aghar Children Hospital, Iran University of Medical Sciences, Tehran, IranSoroosh Shekarchian, MD, Principal investigator, Department of Regenerative Biomedicine at the Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02382874
- Sponsor's protocol ID
- Royan-Kidney-005
- Status last verified
- Nov 2015
- First submitted
- 3 Mar 2015
- Last updated
- 4 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogenic Adipose Derived Mesenchymal Stromal Cells Transplantation to Improve Kidney Function in Refractory Primary Nephrotic Syndrome (Focal Segmental Glomerulosclerosis,FSGS) ,a Phase I Clinical Trial
- ClinicalTrials.gov ID
- NCT02382874
- Acronym / sponsor ID
- Royan-Kidney-005
- Conditions
- Focal Segmental Glomerulosclerosis
- Keywords
- Idiopathic nephrotic syndrome, adipose derived mesenchymal stromal cell, Focal segmental glomerulosclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Royan InstituteOther government
- Study start
- May 2015
- Primary completion
- Jun 2017 (estimated)
- Study completion
- Oct 2017 (estimated)
- First posted
- 9 Mar 2015 (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.
Idiopathic Focal Segmental Glumero Sclerosis (FSGS) is not very common but important manifestation of kidney disease.
FSGS has poor prognosis among the patterns of Idiopathic Nephrotic Syndrome (INS).
Even with treatment (Steroid therapy and cytotoxic immunosuppressant therapy), many patients eventually still require dialysis.
Cell therapy is useful in treatment of INS and mesenchymal stromal/stem cell is one of the cells that useful in the treatment of glomerulus disease.
Intravenous injection of allogeneic adipose derived mesenchymal stromal/stem cell will be done in 5 patients with refractory INS(FSGS). They will be followed 1, 2, 4 weeks and then monthly until a year following injection day.
Detailed description
Primary FSGS thought to be a part of the immune-mediated disease. Main challenge is to decrease protein excretion in urine. Only 30% of children with FSGS achieve complete remission with steroids and other 30-40% patients experience remission (partial and complete) with cytotoxic immunosuppressant drugs. Even with these treatments, many patients eventually still require dialysis. Other treatment strategy doesn't exceed beyond symptomatic treatment and delaying the progression. Also risk of recurrence after kidney transplantation is 20-50%.
Cell therapy is one of the treatment strategies and mesenchymal stromal/stem cell is one modality that notice in glomerulus disease.
We will evaluate safety and efficacy of intravenous injection of allogeneic AD-MSC (adipose-derived mesenchymal stromal cell) in 5 refractory INS patients.They will be followed 1, 2, 4 weeks and then monthly until a year following injection day.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AD-MSC | Experimental | Biological: Intravenous injectionThe patients with FSGS who underwent intravenous injection of AD-MSC. |
Who may qualify
- Ages
- 2 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Age 2-14 years at onset of signs or symptoms of FSGS 2- Primary FSGS involving either native kidneys or primary FSGS recurring after renal transplantation. Biopsy confirmed as primary FSGS (including all subtypes).
- 3-Estimated GFR ≥ 25 ml/min/1.73 m2 4- Up/c > 1.0 (g protein/g creatinine) on first am void 5- Steroid and IS resistance as defined by primary physician (patients with little or no reduction in protein excretion at 12 to 16 weeks are considered to be steroid resistant --A relapse is return of proteinuria to ≥3.5 g/day in someone who had undergone a complete or partial remission- IS resistance: no response to IS in 8-12 weeks) 6-At least one month from last immunization received has passed 7- Ability to understand and willingness to sign consent by patient legal guardian
Key exclusion criteria (2 listed)
- 1- Are immunodeficient or have clinically significant chronic lymphopenia 2-Have an active infection or positive PPD test result 3-Have serologic evidence of current or past HIV, Hepatitis B, or Hepatitis C infection 4-Have any complicating medical issues that interfere with study conduct or cause increased risk such as malignancies, systemic autoimmune disease, diabetes, blood disease, liver disease, etc.
- 5- Positive genetic mutation testing for WT1, Podocin, Nephrin
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 |
|---|---|---|
| Intravenous injectionIntravenous injection of AD-MSC to the patients with FSGS | Biological | AD-MSC |
Primary outcome
Liver function
Time frame: 2 weeks
increase of liver enzymes 2 weeks after cell injection.
Serum creatinine
Time frame: 2 weeks
Decrease of serum creatinine 2 weeks after cell injection.
Proteinuria
Time frame: 12 hour
Reduction in proteinuria to <200 to 300 mg/day will be assessed by Changes in 24 hour urine protein analysis.
3 secondary outcomes
Renal function
Time frame: 12 hours
Increase in anti inflammatory factors
Time frame: 12 hours
Increase in Treg
Time frame: 12hours
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2015.
- Related InfoLink provided by the sponsor.
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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.
Hamid Gourabi, PhD study chair
Head of Royan Institute
Nasser Aghdami, MD,PhD study director
Head of department of regenerative medicine&cell therapy center,Royan Institute
Hassan Otukesh, MD study director
Department Of Pediatric Nephrology and Dialysis, Ali aghar Children Hospital, Iran University of Medical Sciences, Tehran, Iran
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 Intravenous injection in people with Focal Segmental Glomerulosclerosis, run by Royan Institute at 1 site in Iran.
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 2 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: status not confirmed, as of the sponsor's last update on 4 Dec 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 Iran. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Nasser Aghdami, MD,PhD ((+98)21235000 ext. 516, nasser.aghdami@royaninstitute.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Liver function (time frame: 2 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Dec 2015.