Umbilical Cord Derived Mesenchymal Stem Cells Transplantation for Active and Refractory Systemic Lupus Erythematosus

Phase 1

• Conditions: Systemic Lupus Erythematosus
• Interventions: Biological: human umbilical cord derived MSC transplantation for SLE

• Registration Number: NCT01741857
• Lead Sponsor: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School

Brief Summary

This study will explore safety and efficacy of allogeneic umbilical cord (UC) derived mesenchymal stem cells transplantation (MSCT) to treat patients with active and refractory systemic lupus erythematosus (SLE) who have been resistant to multiple standard treatments. The underlying hypothesis is that the active SLE condition is caused by an abnormal immune homeostasis that can be restored by MSCT.

Detailed Description

Inclusion Criteria:

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

2. Lupus nephritis with 24h urine protein≥1g;

3. 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 mycophenolate mofetil 2 g / day, for three months;

4. 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;

5. Willing to use contraception throughout the study and for 12 months following treatment

Study Timeline

• Study Start: 2012-01
• Status Verified: 2012-10
• Study First Submitted: 2012-11-26
• Study First Submitted QC: 2012-12-04
• Study First Posted: 2012-12-05
• Last Update Submitted: 2013-11-01
• Last Update Posted: 2013-11-05
• Primary Completion: 2013-12
• Study Completion: 2013-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 60 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 mycophenolate mofetil 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 months 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;
• Uncontrolled 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
human umbilical cord derived MSC	human umbilical cord derived MSC transplantation for SLE	human umbilical cord derived MSC transplantation for SLE

Primary Outcome Measures

Name	Time	Method
British Isles Lupus Assessment Group score (BILAG)	up to 12 months

Secondary Outcome Measures

Name	Time	Method
Renal function (GFR, Blood Urea Nitrogen, urinalysis)	pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation (for GFR assessed at baseline and 12 months after MSCT)
Lupus serology (Alb, ANA, dsDNA, C3, C4)	pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation

Trial Locations

Locations (1)

The Affiliated Drum Tower Hospital of Nanjing University Medical School; 🇨🇳 Nanjing, Jiangsu, China