NCT00278486

Hematopoietic Stem Cell Transplantation in Autoimmune-Related Retinopathy(ARRON)

Immune Ablation and Hematopoietic Stem Cell Transplantation in Patients With Autoimmune-Related Retinopathy and Optic Neuropathy (ARRON) Syndrome (Not Associated With Cancer)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Richard Burt, MD · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

ARRON is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the retina and/or optic nerve. In addition, the disease may affect the nerves in the ear or other parts of the body . The affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness. If the nerves to the ear are affected, reduced hearing or deafness may result. The likelihood of progression of your disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and rabbit ATG (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of ARRON syndrome. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and rabbit ATG is to destroy the cells in the immune system which are thought to be causing this disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.

Conditions

• Retinal Disease

Interventions

Type	Name	Description
BIOLOGICAL	Hematopoietic stem cell transplantation.	Autologous hematopoietic stem cell transplantation will be performed.

Timeline

• Start date: 2004-08-01
• Primary completion: 2012-04-01
• Completion: 2012-04-01
• First posted: 2006-01-18
• Last updated: 2013-04-08

Locations

1 site across 1 country: United States