- ICH GCP
- US Clinical Trials Registry
- Clinical Trial 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)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
Illinois
-
Chicago, Illinois, United States, 60611
- Northwestern University, Feinberg School of Medicine
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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 Criteria:
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: stem cell transplantation
|
Autologous hematopoietic stem cell transplantation will be performed.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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)
Time Frame: 5 years after transplant
|
5 years after transplant
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- DI ARRON 04
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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