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


Phase 1
18 Years
60 Years
Not Enrolling
Both
Retinal Disease

Thank you

Trial Information

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


Inclusion Criteria:



1. Age between 18-60.

2. 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.

3. 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 Criteria:

1. Absence of light perception lasting more than 6 months

2. Any illness that in the opinion of the investigators would jeopardize the ability of
the patient to tolerate aggressive chemotherapy.

3. 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.

4. Positive pregnancy test.

5. 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.

6. Failure to willingly accept or comprehend irreversible sterility as a side effect of
therapy.

7. FEV1/FVC < 60% of predicted after bronchodilator therapy (if necessary).

8. DLCO < 50% of predicted.

9. Active ischemic heart disease and/or those who have had a myocardial infarction
within 6 months.

10. Resting LVEF < 40 %.

11. Bilirubin > 2.0 mg/dl

12. Serum creatinine > 2.0 mg/dl.

13. Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron
compounds/medications.

14. Presence of metallic objects implanted in the body that would preclude the ability of
the patient to safely have MRI exams.

15. Diagnosis of primary progressive MS.

16. Platelet count < 100,00/ul

17. Psychiatric illness, mental deficiency or cognitive dysfunction making compliance
with treatment or informed consent impossible.

18. Active infection except asymptomatic bacteruria.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Standard Snellen acuity clinical testing and improvement visual fields is done by using Humphrey Automated Machine with 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)

Outcome Time Frame:

5 years after transplant

Safety Issue:

Yes

Principal Investigator

Richard Burt, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Northwestern University

Authority:

United States: Food and Drug Administration

Study ID:

DI ARRON 04

NCT ID:

NCT00278486

Start Date:

August 2004

Completion Date:

April 2012

Related Keywords:

  • Retinal Disease
  • Retinal Diseases

Name

Location

Northwestern University, Feinberg School of MedicineChicago, Illinois  60611