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

Phase 1

Terminated

• Conditions: Systemic Lupus Erythematosus; Systemic Sclerosis
• Interventions: Procedure: Reduced Intensity Allogeneic Transplant; Drug: Fludarabine; Drug: Busulfan; Drug: Campath

• Registration Number: NCT00684255
• Lead Sponsor: New York Medical College

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.

Study Timeline

• Study Start: 2007-08
• Study First Submitted: 2008-05-22
• Study First Submitted QC: 2008-05-23
• Study First Posted: 2008-05-26
• Primary Completion: 2008-07
• Study Completion: 2008-07
• Results First Submitted: 2008-12-22
• Results First Submitted QC: 2009-11-23
• Results First Posted: 2009-12-02
• Status Verified: 2014-02
• Last Update Submitted: 2014-02-06
• Last Update Posted: 2014-04-01

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 1

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Reduced Intensity Regimen for Refractory SLE	Reduced Intensity Allogeneic Transplant	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Reduced Intensity Regimen for Refractory SLE	Fludarabine	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Reduced Intensity Regimen for Refractory SLE	Campath	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Reduced Intensity Regimen for SSc	Reduced Intensity Allogeneic Transplant	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
Reduced Intensity Regimen for SSc	Busulfan	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
Reduced Intensity Regimen for SSc	Campath	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
Reduced Intensity Regimen for Refractory SLE	Busulfan	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
Reduced Intensity Regimen for SSc	Fludarabine	RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).

Primary Outcome Measures

Name	Time	Method
Toxicity	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.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Columbia University Medical Center; 🇺🇸 New York, New York, United States