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Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)

Primary Purpose

Systemic Lupus Erythematosus, Systemic Sclerosis

Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Reduced Intensity Allogeneic Transplant
Fludarabine
Busulfan
Campath
Sponsored by
New York Medical College
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Lupus Erythematosus focused on measuring Autoimmune Disease, Reduced Intensity Transplant

Eligibility Criteria

7 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Diffuse Systemic Sclerosis and variants as per ACR criteria
  • Medically refractory disease
  • Adequate Organ Function - Pulmonary function
  • Renal function, Cardiac function defined as:
  • SGOT (AST) or SGPT (ALT) <5 x upper limit of normal
  • Diagnosis of SLE - Medically refractory disease

Exclusion Criteria:

  • Karnofsky/Lansky <60%

Sites / Locations

  • Columbia University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Reduced Intensity Regimen for Refractory SLE

Reduced Intensity Regimen for SSc

Arm Description

RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).

RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).

Outcomes

Primary Outcome Measures

Toxicity
Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.

Secondary Outcome Measures

Chimerism
Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.
Immune Reconstitution.
Peripheral blood for immune reconstitution for T-cell, B-cell and NK cells to be obtained for measurement of cell.
Progression Free and Overall Survival.
Probability of progression free and overall survival will be measured.

Full Information

First Posted
May 22, 2008
Last Updated
February 6, 2014
Sponsor
New York Medical College
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1. Study Identification

Unique Protocol Identification Number
NCT00684255
Brief Title
Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)
Official Title
Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)
Study Type
Interventional

2. Study Status

Record Verification Date
February 2014
Overall Recruitment Status
Terminated
Why Stopped
inactive
Study Start Date
August 2007 (undefined)
Primary Completion Date
July 2008 (Actual)
Study Completion Date
July 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
New York Medical College

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated.
Detailed Description
This is to test whether a reduced intensity will result in a high degree of mixed or complete donor chimerism and stabilization of autoimmune disease in a select group of patients with medically refractory SLE or SSc.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Lupus Erythematosus, Systemic Sclerosis
Keywords
Autoimmune Disease, Reduced Intensity Transplant

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Reduced Intensity Regimen for Refractory SLE
Arm Type
Experimental
Arm Description
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Arm Title
Reduced Intensity Regimen for SSc
Arm Type
Experimental
Arm Description
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
Intervention Type
Procedure
Intervention Name(s)
Reduced Intensity Allogeneic Transplant
Intervention Description
Eeduced intensity allogeneic stem cell transplantation with a fludarabine/busulfan/alemtuzumab conditioning regimen is anticipated to result in mixed and/or complete donor chimerism and potentially alter the natural history and outcome of patients with medically refractory Systemic Lupus Erythematosus (SLE) or Systemic Sclerosis (SSc).
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Intervention Description
Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2
Intervention Type
Drug
Intervention Name(s)
Busulfan
Intervention Description
Busulfan 3.2 mg/kg Days _8, -7, -6, -5
Intervention Type
Drug
Intervention Name(s)
Campath
Other Intervention Name(s)
Alemtuzumab
Intervention Description
Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2
Primary Outcome Measure Information:
Title
Toxicity
Description
Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Chimerism
Description
Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.
Time Frame
1 year
Title
Immune Reconstitution.
Description
Peripheral blood for immune reconstitution for T-cell, B-cell and NK cells to be obtained for measurement of cell.
Time Frame
1 year
Title
Progression Free and Overall Survival.
Description
Probability of progression free and overall survival will be measured.
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diffuse Systemic Sclerosis and variants as per ACR criteria Medically refractory disease Adequate Organ Function - Pulmonary function Renal function, Cardiac function defined as: SGOT (AST) or SGPT (ALT) <5 x upper limit of normal Diagnosis of SLE - Medically refractory disease Exclusion Criteria: Karnofsky/Lansky <60%
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitchell Cairo, MD
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.childrensnyp.org
Description
(Click on "Morgan Stanley Children's Hospital" and then "Clinical Services" and then "Blood & Marrow Transplantation")

Learn more about this trial

Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)

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