Status:

TERMINATED

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

Lead Sponsor:

Richard Burt, MD

Conditions:

Retinal Disease

Eligibility:

All Genders

18-60 years

Phase:

PHASE1

Brief 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...

Eligibility Criteria

Inclusion

  • Age between 18-60.
  • Diagnosis of ARRON syndrome. Diagnostic criteria described below.
  • Unexplained visual loss over weeks to months. The visual loss includes both visual acuity and field loss define as follows:
  • Visual acuity: 20/40 or less
  • OR
  • Visual field: perimetric mean deviation -5b
  • Positive antibody to retina or optic nerve.
  • OR
  • A response to immunosuppressive drugs or immune modulators (response is defined by improvement of vision or decrease the rate of decline of visual loss).
  • Absence of malignancy {negative physical examination, gastrointestinal endoscopies, mammography and gynecologic examination (for female), and serum PSA measurement (for male) within a year}.
  • Negative MRI of brain.
  • The patient has failed at least 3 months of corticosteroids (prednisone 0.5mg/kg to start), IVIG and at least one other immunosuppressive drug such as methotrexate, Imuran, cyclosporine, etc. Failure is defined by decline of visual acuity (by standard Snellen acuity clinical testing) or visual field (by Humphrey Automated Machine with the 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)

Exclusion

  • Absence of light perception lasting more than 6 months
  • Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
  • Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
  • Positive pregnancy test.
  • Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an IUD (intrauterine device); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
  • Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
  • FEV1/FVC \< 60% of predicted after bronchodilator therapy (if necessary).
  • DLCO \< 50% of predicted.
  • Active ischemic heart disease and/or those who have had a myocardial infarction within 6 months.
  • Resting LVEF \< 40 %.
  • Bilirubin \> 2.0 mg/dl
  • Serum creatinine \> 2.0 mg/dl.
  • Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications.
  • Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams.
  • Diagnosis of primary progressive MS.
  • Platelet count \< 100,00/ul
  • Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible.
  • Active infection except asymptomatic bacteruria.

Key Trial Info

Start Date :

August 1 2004

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

April 1 2012

Estimated Enrollment :

2 Patients enrolled

Trial Details

Trial ID

NCT00278486

Start Date

August 1 2004

End Date

April 1 2012

Last Update

April 8 2013

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Northwestern University, Feinberg School of Medicine

Chicago, Illinois, United States, 60611