NCT00849745

Nonmyeloablative Allo Stem Cell Transplant for Severe Autoimmune Diseases

Nonmyeloablative Allogeneic Peripheral Blood Stem Cell Transplantation for Severe Autoimmune Diseases

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 18 Years – 69 Years
• Healthy volunteers: Not accepted

Summary

Autoimmune diseases present a special challenge to clinicians and the aim of this protocol is to serve as a last-line effort for patients with unmanageable disease. The primary purpose of this study is to assess feasibility in terms of toxicity and engraftment of a less toxic, nonablative conditioning regimen of Campath-1H, moderate dose fludarabine, and cyclophosphamide for patients with severe autoimmune diseases.

Detailed description

Our targeted illnesses are: * Systemic lupus erythematosus (SLE): SLE can involve virtually any organ system, but most commonly involves various combinations of arthritis, dermatitis, glomerulonephritis, central nervous system manifestations and hematologic complications. Although the overall five and ten-year survival rates in SLE are 86% and 80%, respectively, these rates are reduced to 60% and 50%, respectively, in patients with poor prognosis SLE (proliferative glomerulonephritis with chronic changes, elevated serum creatinine, nephrotic syndrome, anemia, low serum C3, inadequate response to treatment). * Systemic sclerosis (SSc): SSc is a condition divided into two forms (diffuse and limited) characterized by excessive and often relentless fibrosis in skin and internal organs. Visceral involvement can manifest as esophageal hypomotility, interstitial lung disease, pulmonary hypertension and renal failure. There is no satisfactory treatment for systemic sclerosis (SSc), which in its diffuse form has a 5-year mortality of 40%, similar to many malignancies. In clinical trials, alpha-interferon did not demonstrate a clinically significant effect and low dose methotrexate showed conflicting results.

Conditions

• Systemic Lupus Erythematosus
• Systemic Sclerosis

Interventions

Type	Name	Description
PROCEDURE	Nonmyeloablative allogeneic stem cell transplant	Prior to receiving Campath-1H, patients will be premedicated with Benadryl 50 mg IV or PO, and acetaminophen 650 mg orally. Hydrocortisone 100 mg IV is given on the first day of Campath. The preparative regimen will begin on day -5 and consist of 4 days of daily fludarabine at 30 mg/m2/d infused over 30 minutes, cyclophosphamide 500 mg/m2/d infused over 1 hour, 5 days of Campath-1H at 20 mg/d in 250 ml of D5 normal saline or normal saline infused over 3 hours. The mixed dosage of chemotherapy may be rounded off to within +/- 5% of the calculated dose, and doses of fludarabine and cyclophosphamide will be based on adjusted ideal body weight. IV hydration and diuretics will be used to maintain adequate urine output during and after administration of cyclophosphamide.

Timeline

• Start date: 2003-01-01
• Primary completion: 2015-07-01
• Completion: 2015-07-01
• First posted: 2009-02-24
• Last updated: 2016-06-23

Locations

1 site across 1 country: United States