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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT00230035
Date of registration: 28/09/2005
Prospective Registration: Yes
Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Public title: Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant (LIST)
Scientific title: A Randomized, Open Label, Phase II Multicenter Study of Non-Myeloablative Autologous Transplantation With Auto-CD34+HPC Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Systemic Lupus Erythematosus
Date of first enrolment: September 2005
Target sample size: 0
Recruitment status: Withdrawn
URL:  http://clinicaltrials.gov/show/NCT00230035
Study type:  Interventional
Study design:  Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Betty Diamond, MD
Address: 
Telephone:
Email:
Affiliation:  Department of Medicine, Columbia University
Name:     Keith Sullivan, MD
Address: 
Telephone:
Email:
Affiliation:  Division of Cellular Therapy, Department of Medicine, Duke University
Name:     Ann Traynor, MD
Address: 
Telephone:
Email:
Affiliation:  Division of Hematology and Oncology, University of Massachusetts Medical School
Name:     Richard Burt, MD
Address: 
Telephone:
Email:
Affiliation:  Division of Immunotherapy, Northwestern University
Name:     Kenneth Kalunian, MD
Address: 
Telephone:
Email:
Affiliation:  Division of Rheumatology, Allergy, and Immunology, University of California, Los Angeles
Name:     Bevra Hahn, MD
Address: 
Telephone:
Email:
Affiliation:  Division of Rheumatology, Department of Medicine, University of California, Los Angeles
Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female subjects between the ages of 18 and 60 years, inclusive

- Meet at least 4 of 11 American College of Rheumatology (ACR) Revised Classification
Criteria for SLE

- Have at least one of the following conditions defining severe steroid refractory
disease:

a) Lupus nephritis - Subjects must have severe disease, defined as meeting criteria
for BILAG renal category A, and be corticosteroid dependent while receiving at least
6 months of pulse CTX at doses of 500 to 1000 mg/m2 every 4 weeks or MMF at of 2
g/day or greater. If nephritis is to constitute the sole eligibility, a renal biopsy
performed within 11 months of the date of screening must show ISN/RPS 2003
classification of lupus nephritis Class III or IV disease. A renal biopsy must
demonstrate the potential of a reversible (non-fibrotic) component. b) Visceral organ
involvement other than nephritis - Subjects must be without mesenteric vasculitis.
The subject must be BILAG cardiovascular/respiratory category A, vasculitis category
A, or neurologic category A, and be corticosteroid dependent while receiving at least
3 months of oral (2 to 3 mg/kg/day or greater) or IV CTX (500 mg/m2 or greater every
4 weeks). c) Cytopenias that are immune-mediated - Subjects must be BILAG hematologic
category A and be corticosteroid dependent while receiving at least one of the
following: azathioprine at 2 mg/kg/day or greater for at least 3 months, MMF at 2
g/day or greater for more than 3 months, CTX at 500 mg/m2 or greater intravenously
every 4 weeks or 2 mg/kg/day orally for at least 3 months, cyclosporine at 3
mg/kg/day or greater for at least 3 months, or have had a splenectomy. d)
Mucocutaneous disease - Subjects must meet BILAG mucocutaneous category A and be
corticosteroid dependent while receiving at least 1 of the following: azathioprine at
2 mg/kg/day or greater for at least 3 months; methotrexate at 15 mg/week or greater
for at least 3 months; CTX at 500 mg/m2 or greater intravenously every 4 weeks or 2
mg/kg/day or greater orally for at least 3 months, cyclosporine at 3 mg/kg/day or
greater for at least 3 months, or MMF at doses 2 g/day or greater for at least 3
months. e) Arthritis/myositis - Subjects must meet BILAG musculoskeletal category A
and be corticosteroid dependent while receiving at least one of the following:
azathioprine at 2 mg/kg/day or greater for at least 3 months, methotrexate at 15
mg/week or greater for at least 3 months, CTX at 500 mg/m2 or greater intravenously
every 4 weeks or 2 mg/kg/day or greater orally for at least 3 months, MMF at 2 g/day
or greater for at least 3 months, or cyclosporine at 3 mg/kg/day or greater for at
least 3 months.

- Have the ability and willingness to provide written informed consent. In case of
lupus cerebritis, a person designated by the subject may give consent.

- Must be ANA positive

Exclusion Criteria:

- HIV positive status

- Any active systemic infection

- Hepatitis B surface antigen positive

- Hepatitis C PCR positive

- Use of immunosuppressive agents for other indications other than SLE

- Any comorbid illness that in the opinion of the investigator would jeopardize the
ability of the subject to tolerate therapy

- For lupus nephritis: renal biopsy, performed within 11 months of the screening date,
showing Class I, II, or V disease or Class III or IV disease in conjunction with
total sclerosis of 50% or more of the glomeruli

- Ongoing cancer. Patients with localized basal cell or squamous skin cancer are not
excluded.

- Pregnancy, unwillingness to use acceptable means of birth control, or unwilling to
accept or comprehend irreversible sterility as a side effect of therapy

- Psychiatric illness or mental deficiency not due to active lupus cerebritis making
compliance with treatment or informed consent impossible

- Hemoglobin adjusted diffusion capacity test (DLCO) less than 30% at screening

- Resting left ventricular ejection fraction (LVEF) 40% or less as evaluated by
echocardiogram

- History of an allergic reaction or hypersensitivity to Escherichia coli recombinant
proteins, CTX, or any part of the investigative or control therapy

- SGOT/SGPT greater than 2 x the upper limit of normal, unless due to active lupus

- ANC 1000 or greater if not due to active SLE

- Subdural hematoma or any active intracranial bleeding documented within 30 days of
the screening visit

- Failure to be approved for participation in this study by the SCSLE Protocol
Eligibility Review Committee

- Positive tuberculin skin test

- Presence of mesenteric vasculitis



Age minimum: 18 Years
Age maximum: 60 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Systemic Lupus Erythematosus
Intervention(s)
Drug: Azathioprine
Drug: Corticosteroids
Drug: Growth colony stimulating factor (G-CSF)
Drug: Intravenous immunoglobulin
Drug: Leflunomide
Drug: Methotrexate
Drug: Methylprednisolone
Drug: Mycophenolate mofetil
Drug: Rabbit anti-thymocyte globulin
Drug: Rituximab
Procedure: Autologous CD34+HPC transplantation (HSCT)
Procedure: Leukapheresis
Procedure: Non-myeloablative high dose immunosuppressive therapy conditioning (HDIT)
Procedure: Plasmapheresis
Primary Outcome(s)
Mortality resulting from treatment, underlying disease, or unrelated causes [Time Frame: At Month 30]
Secondary Outcome(s)
Secondary ID(s)
DAIT SCSLE-01
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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