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Allogenic AD-MSC Transplantation in Idiopathic Nephrotic Syndrome (Focal Segmental Glomerulosclerosis)

Primary Purpose

Focal Segmental Glomerulosclerosis

Status

Unknown status

Phase

Phase 1

Locations

Iran, Islamic Republic of

Study Type

Interventional

Intervention

Intravenous injection

About this trial

This is an interventional treatment trial for Focal Segmental Glomerulosclerosis focused on measuring Idiopathic nephrotic syndrome, adipose derived mesenchymal stromal cell, Focal segmental glomerulosclerosis

Eligibility Criteria

2 Years - 14 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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

Sites / Locations

Royan InstituteRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AD-MSC

Arm Description

The patients with FSGS who underwent intravenous injection of AD-MSC.

Outcomes

Primary Outcome Measures

Liver function

increase of liver enzymes 2 weeks after cell injection.

Serum creatinine

Decrease of serum creatinine 2 weeks after cell injection.

Proteinuria

Reduction in proteinuria to <200 to 300 mg/day will be assessed by Changes in 24 hour urine protein analysis.

Secondary Outcome Measures

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.

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.

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.

Full Information

NCT ID

NCT02382874

First Posted

March 3, 2015

Last Updated

December 3, 2015

Sponsor

Royan Institute

1. Study Identification

Unique Protocol Identification Number

NCT02382874

Brief Title

Allogenic AD-MSC Transplantation in Idiopathic Nephrotic Syndrome (Focal Segmental Glomerulosclerosis)

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2015

Overall Recruitment Status

Unknown status

Study Start Date

May 2015 (undefined)

Primary Completion Date

June 2017 (Anticipated)

Study Completion Date

October 2017 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Royan Institute

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief 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). 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Focal Segmental Glomerulosclerosis

Keywords

Idiopathic nephrotic syndrome, adipose derived mesenchymal stromal cell, Focal segmental glomerulosclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

AD-MSC

Arm Type

Experimental

Arm Description

The patients with FSGS who underwent intravenous injection of AD-MSC.

Intervention Type

Biological

Intervention Name(s)

Intravenous injection

Intervention Description

Intravenous injection of AD-MSC to the patients with FSGS

Primary Outcome Measure Information:

Title

Liver function

Description

increase of liver enzymes 2 weeks after cell injection.

Time Frame

2 weeks

Title

Serum creatinine

Description

Decrease of serum creatinine 2 weeks after cell injection.

Time Frame

2 weeks

Title

Proteinuria

Description

Reduction in proteinuria to <200 to 300 mg/day will be assessed by Changes in 24 hour urine protein analysis.

Time Frame

12 hour

Secondary Outcome Measure Information:

Title

Renal function

Description

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.

Time Frame

12 hours

Title

Increase in anti inflammatory factors

Description

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.

Time Frame

12 hours

Title

Increase in Treg

Description

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.

Time Frame

12hours

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

14 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: - 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 Exclusion Criteria: - 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Nasser Aghdami, MD,PhD

Phone

(+98)21235000

Ext

516

nasser.aghdami@royaninstitute.org

First Name & Middle Initial & Last Name or Official Title & Degree

Leila Arab, MD

Phone

(+98)21235000

Ext

414

Leara91@gmail.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Hamid Gourabi, PhD

Organizational Affiliation

Head of Royan Institute

Official's Role

Study Chair

First Name & Middle Initial & Last Name & Degree

Nasser Aghdami, MD,PhD

Organizational Affiliation

Head of department of regenerative medicine&cell therapy center,Royan Institute

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Hassan Otukesh, MD

Organizational Affiliation

Department Of Pediatric Nephrology and Dialysis, Ali aghar Children Hospital, Iran University of Medical Sciences, Tehran, Iran

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Rozita Hosseini, MD

Organizational Affiliation

Department Of Pediatric Nephrology and Dialysis, Ali aghar Children Hospital, Iran University of Medical Sciences, Tehran, Iran

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Soroosh Shekarchian, MD

Organizational Affiliation

Department of Regenerative Biomedicine at the Cell Science Research Center, Royan Institute for Stem Cell Biology and Technology, ACECR, Tehran, Iran.

Official's Role

Principal Investigator

Facility Information:

Facility Name

Royan Institute

City

Tehran

Country

Iran, Islamic Republic of

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Nasser Aghdami, MD,PhD

Phone

(+98)2123562000

Ext

516

nasser.aghdami@royaninstitute.org

First Name & Middle Initial & Last Name & Degree

Leila Arab, MD

Phone

(+98)2123562000

Ext

414

Leara91@gmail.com

First Name & Middle Initial & Last Name & Degree

Reza Moghadasali, PhD

12. IPD Sharing Statement

Links:

URL

http://royaninstitute.org

Description

Related Info

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Allogenic AD-MSC Transplantation in Idiopathic Nephrotic Syndrome (Focal Segmental Glomerulosclerosis)

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