Systemic Lupus Erythematosus, Systemic Sclerosis
Conditions
Keywords
Autoimmune Disease, Reduced Intensity Transplant
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.
Interventions
PROCEDUREReduced 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).
DRUGFludarabine
Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2
DRUGBusulfan
Busulfan 3.2 mg/kg Days \_8, -7, -6, -5
DRUGCampath
Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2
Sponsors
New York Medical College
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 50 Years
Healthy volunteers
No
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%
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Progression Free and Overall Survival. | 1 year | Probability of progression free and overall survival will be measured. |
Countries
United States
Participant flow
Recruitment details
Study closed due to low accrual.
Participants by arm
| Arm | Count |
|---|---|
| Systemic Sclerosis (SSc) Reduced Intensity Regimen Medically Refractory Systemic Sclerosis (SSc) | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Systemic Sclerosis (SSc) |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
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.
Time frame: 1 year
Secondary
Chimerism
Percentage(%) of mixed and/or complete donor chimerism has been measured at different time points.
Time frame: 1 year
Secondary
Immune Reconstitution.
Peripheral blood for immune reconstitution for T-cell, B-cell and NK cells to be obtained for measurement of cell.
Time frame: 1 year
Secondary
Progression Free and Overall Survival.
Probability of progression free and overall survival will be measured.
Time frame: 1 year