Allogeneic Stem Cell Transplantation in Systemic Lupus Erythematosus

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00278590
• Date of registration: 16/01/2006
• Prospective Registration: No
• Primary sponsor: Northwestern University
• Public title: Allogeneic Stem Cell Transplantation in Systemic Lupus Erythematosus
• Scientific title: Allogeneic Stem Cell Transplantation in Patients With Systemic Lupus Erythematosus
• Date of first enrolment: July 2004
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT00278590
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Richard Burt, MD
• Affiliation: Northwestern University

Key inclusion & exclusion criteria

Recipient Inclusion Criteria:

- Ages 18 to 50 years old.

- Meet at least 4 of 11 American College of Rheumatology (ACR) Classification criteria
for SLE (see Appendix 1).

- Able to give informed consent.

- HLA matched sibling donor available.

- Meet one of following three:

1. For lupus nephritis, participants must fail pulse cyclophosphamide (500 to 1000
mg/m2 monthly for a minimum of 6 months). Failure is defined as meeting criteria
to be considered as BILAG (Appendix 4) renal category A. If indication for HSCT
is nephritis, a renal biopsy must be obtained and document either class III or IV
glomerulonephritis.

2. For visceral organ involvement other than nephritis, participants must be BILAG
cardiovascular/respiratory category A, vasculitis category A, or neurologic
category A and must fail at least 3 months of oral or IV cyclophosphamide and be
corticosteroid dependent. Steroid dependence being defined as at least 3 months
of steroid therapy and inability to wean corticosteroid to less than 20 mg/day of
prednisone or equivalent.

3. For cytopenias that are immune mediated, participants must be BILAG hematologic
category A. Participants must have an inability to maintain platelets > 15,000,
an inability to prevent active bleeding without transfusion, an inability to
maintain hemoglobin > 7.0, or an inability to prevent cardiovascular disease
without transfusion. In addition, participants must fail corticosteroids (either
oral prednisone > 0.5 mg/kg/day for more than 6 months or pulse
methylprednisolone for at least one cycle of three days), be refractory to IVIG,
and at least one of the following: azathioprine at 2 mg/kg/day for at least 3
months, mycophenolate mofetil 2 grams daily for more than 3 months,
cyclophosphamide intravenously or orally for at least 3 months, or cyclosporine
at least 3 mg/kg/day for at least 3 months, danazol for at least 3 months, or
splenectomy.

Recipient Exclusion Criteria:

- HIV positive.

- Ongoing malignancy except localized basal cell or squamous skin cancer. Other
malignancies for which the participant is judged to be cured by local surgical
therapy, such as head and neck cancer, or stage I or II breast cancer will be
considered on an individual basis by the investigators doing the final screening for
participant qualification.

- Positive pregnancy test, inability or unwillingness to pursue effective means of birth
control, failure to willingly accept or comprehend irreversible sterility as a side
effect of therapy.

- Psychiatric illness or mental deficiency making compliance with treatment or informed
consent impossible.

- DLCO < 45% of predicted unless attributed solely to active lupus.

- Resting LVEF < 40% unless due to active lupus.

- Known hypersensitivity to E. Coli derived proteins.

- Transaminases greater than 2 times normal unless due to active lupus.

- Any illness that in the opinion of the investigator would jeopardize the ability of
the patient to tolerate this treatment.

Donor Inclusion Criteria

- Donor must be a HLA identical sibling or HLA matched cord blood donor.

- If donor is HLA matched sibling, donor must be > 18 years of age and less than 50years
old.

- If multiple HLA matched donors are available, preference will be given to samesex,
same CMV status, and nulliparous donor, or in the case of cord blood higher nucleated
cell count.

- If donor is HLA matched cord blood, cord blood stem cells will be obtained from the
New York Blood Center Cord Blood Registry (Tel [phone]) which is an
internationally recognized registry or, if a match is not available, from Stemcyte
(626-821-9860) which is a commercial registry that specializes in minority donors or
from National Marrow Donor Program (NMDP). One unit of HLA matched cord blood unit
will be infused on day zero.

Donor Exclusion Criteria

- Physiologic age > 50 years old or <18 years old.

- HIV positive.

- Active ischemic heart disease or heart failure.

- Acute or chronic active hepatitis.

- Uncontrolled diabetes mellitus or any other illness that in the opinion of the
investigators would jeopardize the ability of the donor to tolerate stem cell
collection.

- Prior history of malignancy except localized basal cell or squamous skin cancer. Other
malignancies for which the patient is judged to be cured by local surgical therapy,
such as (but not limited to) head and neck cancer, or stage I or II breast cancer will
be considered on an individual basis.

- Positive pregnancy test.

- Positive ANA or anti-ds DNA.

- Psychiatric illness or mental deficiency making compliance with treatment or informed
consent impossible.

- Major hematological abnormalities such as platelet count less than 150,000/ul, ANC
less than 1000/ul.

- If donor is sibling must collect a minimum of 2. 106CD34+ cells/kg to proceed to
Transplant.

- If donor is cord blood unit(s) then a minimum number of nucleated cells available must
be more than 2 x 107 /kg. To achieve this number of nucleated cells, two units of HLA
matched cord blood may be utilized. (Wagner JE Blood. 2005 Feb 1;105(3):1343-7)



Age minimum: 18 Years
Age maximum: 50 Years
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Lupus Erythematosus

Intervention(s)

Biological: Allogeneic Stem Cell Transplantation

Primary Outcome(s)

Survival, disease free survival, number of relapses, organ function [Time Frame: Evaluation of response will be performed for 5 years.]

Secondary ID(s)

DI SLE.Allo2004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors