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

February 6, 2014 updated by: Mitchell Cairo, New York Medical College

Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 years to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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%

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • 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 Names:
  • Alemtuzumab

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Toxicity
Time Frame: 1 year
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.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Chimerism
Time Frame: 1 year
Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.
1 year
Immune Reconstitution.
Time Frame: 1 year
Peripheral blood for immune reconstitution for T-cell, B-cell and NK cells to be obtained for measurement of cell.
1 year
Progression Free and Overall Survival.
Time Frame: 1 year
Probability of progression free and overall survival will be measured.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

New York Medical College

Investigators

  • Principal Investigator: Mitchell Cairo, MD, Columbia University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2007

Primary Completion (Actual)

July 1, 2008

Study Completion (Actual)

July 1, 2008

Study Registration Dates

First Submitted

May 22, 2008

First Submitted That Met QC Criteria

May 23, 2008

First Posted (Estimate)

May 26, 2008

Study Record Updates

Last Update Posted (Estimate)

April 1, 2014

Last Update Submitted That Met QC Criteria

February 6, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AAAB1324
  • CHNY-01-511 (Other Identifier: CU)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Systemic Lupus Erythematosus

Clinical Trials on Reduced Intensity Allogeneic Transplant

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Hungary

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe