Mesenchymal Stem Cells Transplantation for Refractory Systemic Lupus Erythematosus (SLE)

Phase 1

• Conditions: Refractory Systemic Lupus Erythematosus
• Interventions: Biological: Allogeneic MSC (AlloMSC)

• Registration Number: NCT00698191
• Lead Sponsor: Nanjing Medical University

Brief Summary

This study will explore a new approach to treat patients with a medical condition known as systemic lupus erythematosus (SLE) who have been resistant to previous treatments using a new population of cells with capability to restore a normal immune system that will no longer attack the body.

The stated hypothesis is that the SLE condition is caused by an abnormal immune system that can be restored by replenishing the body with a new population of progenitor cells.

Detailed Description

The purpose of this study is to evaluate the safety and efficacy of allogeneic bone marrow derived mesenchymal stem cell (AlloMSC) transplantation in patients with refractory SLE. Patients with Lupus nephritis and refractory to corticosteroid or cyclophosphamide trials will be enrolled in this trial. The treatment intervention includes a 24 hour pretreatment with cyclophosphamide followed by AlloMSC transplantation. Patients will be admitted to the in-patient service for the 3-5 days for the transplant treatment and will be followed up in the outpatient clinic. All baseline lupus serology, renal function panels will be obtained at pre-treatment admission. Post-transplantation follow-up visits will be at monthly intervals for lupus serology and renal function tests, and every 3 months for analysis of T regulatory population. The transplanted patients will be evaluated by an integrated team of rheumatologists, hematologists and bone marrow transplant specialists every month for the entire duration of the trial (2 years) and every 6-12 months thereafter.

Study Timeline

• Study Start: 2007-03
• Status Verified: 2008-06
• Study First Submitted: 2008-06-13
• Study First Submitted QC: 2008-06-16
• Last Update Submitted: 2008-06-16
• Study First Posted: 2008-06-17
• Last Update Posted: 2008-06-17
• Primary Completion: 2011-12
• Study Completion: 2012-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 70 years old, SLEDAI≥8;

• Lupus nephritis with 24h urine protein≥1g;

• Refractory disease as determined by failure of the following regimens:

  • Trial of corticosteroids (oral prednisone more than 20 mg/day);
  • Trial of cyclophosphamide 0.4 ~ 0.6 / m2 every two weeks for six months, or other immunosuppressive drugs, such as MMF 2 g / day, for three months;

• Patients must sign an informed consent indicating that they are aware of the investigational nature of the study in keeping with the policy of the hospital;

• Willing to use contraception throughout the study and for 12 mos following treatment.

Exclusion Criteria

• Abnormal liver function (ALT higher than 3 times the normal value);
• End-stage renal failure;
• Severe heart and pulmonary failure, or other important organs damage;
• Undercontrolled infections
• Pregnant or breast feeding women, male or female who intended to recent pregnancy.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Allogeneic MSC (AlloMSC)	-

Primary Outcome Measures

Name	Time	Method
Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)	Monthly
Lupus serology (ANA, dsDNA, C3, C4)	Monthly
Renal function (GFR, BUN, urinalysis)	Monthly

Secondary Outcome Measures

Name	Time	Method
Percentage of systemic T regulatory population	Every 3 months

Trial Locations

Locations (1)

Department of Rheumatology and Immunology, Nanjing University Medical College Affiliated Drum Tower Hospital; 🇨🇳 Nanjing, Jiangsu, China