Hematopoietic Stem Cell Transplant in Devic's Disease

• Status: Completed
• Trial end date: 2018-11-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. 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 intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Northwestern University
• Treatments: Antilymphocyte Serum; Cyclophosphamide; Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Rituximab; Thymoglobulin

Criteria

Inclusion Criteria:

• Age between 16-65, at the time of pretransplant evaluation

• An established diagnosis of Devic's disease (more than one acute attack)

• NMO- IgG aquaporin-4 autoantibody positive

Exclusion Criteria:

• Paraplegia or quadriplegia and legal blindness (defined as visual acuity of 20/200 or less in the better eye with the best correction possible)

• 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 intrauterine device (IUD); 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

• forced expiratory volume at one (FEV1) / forced vital capacity (FVC) < 60% of predicted after bronchodilator therapy (if necessary)

• Diffusing capacity of lung for carbon monoxide (DLCO) < 50% of predicted

• Resting left ventricular ejection fraction (LVEF) < 50 %

• Serum creatinine > 2.0 mg/dl

• Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins

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

• Bilirubin > 2.0 mg/dl

• Platelet count < 100,000/ul or absolute neutrophil count (ANC) < 1000/ul

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

• Active infection except asymptomatic bacteriuria

• Inability to give informed consent

• HIV positive

• Transaminases > 3x of normal limits, liver cirrhosis