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

This study has been terminated.
(inactive)
Sponsor:
Information provided by (Responsible Party):
Mitchell Cairo, New York Medical College
ClinicalTrials.gov Identifier:
NCT00684255
First received: May 22, 2008
Last updated: February 6, 2014
Last verified: February 2014
  Purpose

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.


Condition Intervention Phase
Systemic Lupus Erythematosus
Systemic Sclerosis
Procedure: Reduced Intensity Allogeneic Transplant
Drug: Fludarabine
Drug: Busulfan
Drug: Campath
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)

Resource links provided by NLM:


Further study details as provided by New York Medical College:

Primary Outcome Measures:
  • Toxicity [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.

  • Immune Reconstitution. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    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. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Probability of progression free and overall survival will be measured.


Enrollment: 1
Study Start Date: August 2007
Study Completion Date: July 2008
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Reduced Intensity Regimen for Refractory SLE
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Procedure: Reduced Intensity Allogeneic Transplant
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).
Drug: Fludarabine
Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2
Drug: Busulfan
Busulfan 3.2 mg/kg Days _8, -7, -6, -5
Drug: Campath
Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2
Other Name: Alemtuzumab
Experimental: Reduced Intensity Regimen for SSc
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
Procedure: Reduced Intensity Allogeneic Transplant
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).
Drug: Fludarabine
Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2
Drug: Busulfan
Busulfan 3.2 mg/kg Days _8, -7, -6, -5
Drug: Campath
Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2
Other Name: Alemtuzumab

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.

  Eligibility

Ages Eligible for Study:   7 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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%
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00684255

Locations
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
New York Medical College
Investigators
Principal Investigator: Mitchell Cairo, MD Columbia University
  More Information

Additional Information:
No publications provided

Responsible Party: Mitchell Cairo, Principal Investigator, New York Medical College
ClinicalTrials.gov Identifier: NCT00684255     History of Changes
Other Study ID Numbers: AAAB1324, CHNY-01-511
Study First Received: May 22, 2008
Results First Received: December 22, 2008
Last Updated: February 6, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by New York Medical College:
Autoimmune Disease
Reduced Intensity Transplant

Additional relevant MeSH terms:
Lupus Erythematosus, Systemic
Scleroderma, Systemic
Scleroderma, Diffuse
Sclerosis
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases
Skin Diseases
Pathologic Processes
Busulfan
Fludarabine phosphate
Fludarabine
Alemtuzumab
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists
Antimetabolites, Antineoplastic
Antimetabolites

ClinicalTrials.gov processed this record on August 01, 2014