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

Last updated: December 3, 2015
Sponsor: Royan Institute
Overall Status: Trial Status Unknown

Phase

1

Condition

Kidney Failure (Pediatric)

Nephropathy

Kidney Disease

Treatment

N/A

Clinical Study ID

NCT02382874
Royan-Kidney-005
  • Ages 2-14
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Age 2-14 years at onset of signs or symptoms of FSGS 2- Primary FSGS involving eithernative kidneys or primary FSGS recurring after renal transplantation. Biopsy confirmed asprimary 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 noreduction in protein excretion at 12 to 16 weeks are considered to be steroid resistant --Arelapse is return of proteinuria to ≥3.5 g/day in someone who had undergone a complete orpartial remission- IS resistance: no response to IS in 8-12 weeks) 6-At least one monthfrom last immunization received has passed 7- Ability to understand and willingness to signconsent by patient legal guardian

Exclusion

Exclusion Criteria:

  • 1- Are immunodeficient or have clinically significant chronic lymphopenia 2-Have anactive infection or positive PPD test result 3-Have serologic evidence of current or pastHIV, Hepatitis B, or Hepatitis C infection 4-Have any complicating medical issues thatinterfere with study conduct or cause increased risk such as malignancies, systemicautoimmune disease, diabetes, blood disease, liver disease, etc.

5- Positive genetic mutation testing for WT1, Podocin, Nephrin

Study Design

Total Participants: 5
Study Start date:
May 01, 2015
Estimated Completion Date:
October 31, 2017

Study 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.

Connect with a study center

  • Royan Institute

    Tehran,
    Iran, Islamic Republic of

    Active - Recruiting

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