Clinical Trial Detail

The purpose of this study is to test the safety and effect of an experimental treatment for severe cases of neurological autoimmune diseases. We want to learn what effects, good or bad, this treatment has on study participants’ diseases.

We are studying high-dose chemotherapy (drugs called carmustine, etoposide, cytosine arabinoside and melphalan) and antithymocyte globulin, followed by autologous or syngeneic stem cell transplant. (Autologous transplant is a procedure in which a patient receives his or her own stem cells; syngeneic transplant is a procedure in which a patient receives stem cells donated by his or her healthy identical twin.)

Treatment on this study will last about 2-3 months. Study participants will be admitted to the University of Washington Medical Center and stay in the hospital for about 3 weeks following the transplant. After that, participants will have follow-up visits at the Seattle Cancer Care Alliance outpatient clinic for about 1-2 months. Study participants will be expected to remain in Seattle for approximately 3 months after treatment for study follow-up.

Although preferable, not all study testing will need to be completed at the transplant center. Participants will return to the transplant center or be seen by their doctors for follow-up exams every 3 months for the first year after transplant, then annually for 5 years.

Diseases included in this study (patients with other related diseases may also be eligible):

• Primary CNS Vasculitis

• Rasmussen’s Encephalitis

• Autoimmune Peripheral Neuropathy

• Autoimmune Cerebellar Degeneration

• Gait Ataxia with Late Age Onset Polyneuropathy (GALOP)

• Stiff Person Syndrome

• Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

• Myasthenia Gravis

• Lambert Eaton Myasthenic Syndrome (LEMS)

• HTLV-1- Associated Myelopathy (HAM)/ Tropical Spastic Paraparesis (TSP)

• Opsoclonus/ Myoclonus (OM ; Anti-Ri)

• Neuromyelitis Optica (NMO)

• Multiple Sclerosis (MS)

1. Age 70 years or younger

2. Diagnosed with an autoimmune disorder of the central or peripheral nervous system

3. Evidence of disease activity

4. Must have had at least 2 previous lines of standard treatment that failed

Other eligibility criteria may apply.

1. Age 71 years or older

2. Pregnant or planning to become pregnant within 1 year of the procedure

3. Tested positive for HIV antibodies (HIV-positive)

4. Lung, heart, liver or kidney impairment (according to study guidelines)

5. Active uncontrolled infection

6. Evidence of myelodysplasia

Other exclusion criteria may apply.