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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00278486
Recruitment Status : Terminated (First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.)
First Posted : January 18, 2006
Last Update Posted : April 8, 2013
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Tracking Information
First Submitted Date  ICMJE January 15, 2006
First Posted Date  ICMJE January 18, 2006
Last Update Posted Date April 8, 2013
Study Start Date  ICMJE August 2004
Actual Primary Completion Date April 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 20, 2008)
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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 15, 2006)
  • 1. Toxicity
  • 2. Efficacy: disease assessment by visual acuity, ophthalmologic examination and ERG
  • 3. Survival
Change History Complete list of historical versions of study NCT00278486 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Hematopoietic Stem Cell Transplantation in Autoimmune-Related Retinopathy(ARRON)
Official Title  ICMJE Immune Ablation and Hematopoietic Stem Cell Transplantation in Patients With Autoimmune-Related Retinopathy and Optic Neuropathy (ARRON) Syndrome (Not Associated With Cancer)
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 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Retinal Disease
Intervention  ICMJE Biological: Hematopoietic stem cell transplantation.
Autologous hematopoietic stem cell transplantation will be performed.
Study Arms  ICMJE Experimental: stem cell transplantation
Intervention: Biological: Hematopoietic stem cell transplantation.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 9, 2012)
2
Original Enrollment  ICMJE
 (submitted: January 15, 2006)
10
Actual Study Completion Date  ICMJE April 2012
Actual Primary Completion Date April 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00278486
Other Study ID Numbers  ICMJE DI ARRON 04
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Richard Burt, MD, Northwestern University
Study Sponsor  ICMJE Richard Burt, MD
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Richard Burt, MD Northwestern University
PRS Account Northwestern University
Verification Date April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP