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

New York Medical College

Status and phase: Terminated; Phase 1

Conditions: Systemic Sclerosis; Systemic Lupus Erythematosus

Treatments: Drug: Busulfan; Drug: Fludarabine; Procedure: Reduced Intensity Allogeneic Transplant; Drug: Campath

Study type: Interventional

Funder types: Other

Identifiers

NCT00684255

AAAB1324

CHNY-01-511 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 1 patient

Sex: All

Ages: 7 to 50 years old

Volunteers: No 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%

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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1 participants in 2 patient groups

Reduced Intensity Regimen for Refractory SLE

Experimental group

Description:

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

Treatment:

Drug: Campath

Drug: Busulfan

Drug: Fludarabine

Procedure: Reduced Intensity Allogeneic Transplant

Reduced Intensity Regimen for SSc

Experimental group

Description:

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

Treatment:

Drug: Campath

Drug: Busulfan

Drug: Fludarabine

Procedure: Reduced Intensity Allogeneic Transplant