High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic Diseases

Who is this study for? Patients with Autoimmune Diseases
What treatments are being studied? Anti-Thymocyte Globulin+Autologous Hematopoietic Stem Cell Transplantation+Carmustine+Cytarabine+Etoposide+Laboratory Biomarker Analysis+Melphalan+Peripheral Blood Stem Cell Transplantation+Prednisone+Syngeneic Bone Marrow Transplantation
Status: Active_not_recruiting
Location: See all (3) locations...
Intervention Type: Drug, Other, Biological, Procedure
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a stem cell transplant works in treating patients with autoimmune neurologic disease that did not respond to previous therapy. In autoimmune neurological diseases, the patient's own immune system 'attacks' the nervous system which might include the brain/spinal cord and/or the peripheral nerves. Giving high-dose chemotherapy, including carmustine, etoposide, cytarabine, melphalan, and antithymocyte globulin, before a stem cell transplant weakens the immune system and may help stop the immune system from 'attacking' a patient's nervous system. When the patient's own (autologous) stem cells are infused into the patient they help the bone marrow make red blood cells, white blood cells, and platelets so the blood counts can improve.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 71
Healthy Volunteers: f
View:

• Patients with an autoimmune disorder of the central or peripheral nervous system will be eligible; this will include:

‣ Primary Central Nervous System (CNS) vasculitis

⁃ Rasmussen's encephalitis

⁃ Autoimmune peripheral neuropathy (anti-Hu \[Anna-1\], anti-GM1 \[GD1b\], anti-MAG, anti-ganglioside, anti-sulfatide)

⁃ Autoimmune cerebellar degeneration

⁃ Gait Ataxia with Late age Onset Polyneuropathy (GALOP)

⁃ Stiff Person Syndrome

⁃ Chronic Inflammatory Demyelinating Polyneuropathy

⁃ Myasthenia Gravis

⁃ Lambert-Eaton myasthenic syndrome

⁃ Human T-cell lymphotropic virus (HTLV)-1-associated myelopathy (HAM) / tropical spastic paraparesis (TSP)

⁃ Opsoclonus/myoclonus (anti-Ri)

⁃ Neuromyelitis optica

⁃ Multiple sclerosis

⁃ Other central or peripheral nervous system autoimmune diseases as approved by study neurologists and the Fred Hutchinson Cancer Research Center (FHCRC) faculty at Patient Care Conference (PCC)

• Patients must satisfy the criteria for a diagnosis of one of the severe neurological autoimmune disorders outlined

• Patients age =\< 70 years

• Evidence of disease activity as outlined (e.g. gadolinium enhancement on magnetic resonance imaging of the brain or clinical progression)

• Patients must have failed at least 2 lines of standard therapy as outlined for the specific diseases

• DONOR: Sibling of any patient enrolled on this protocol proven by ABO typing, human leukocyte antigen (HLA) typing and variable number tandem repeat (VNTR) analysis to be syngeneic with the patient (e.g. identical twin)

• DONOR: Willing to undergo multiple apheresis procedures (except donors \< 12 years who will undergo bone marrow harvests)

Locations
United States
Colorado
Colorado Blood Cancer Institute
Denver
Washington
Fred Hutch/University of Washington Cancer Consortium
Seattle
Swedish Medical Center-First Hill
Seattle
Time Frame
Start Date: 2008-06
Completion Date: 2030-01-31
Participants
Target number of participants: 53
Treatments
Experimental: Treatment (immunosuppressive therapy followed by transplant)
Patients receive carmustine IV on day -6, etoposide IV and cytarabine IV BID on days -5 to -2, melphalan IV on day -1 and antithymocyte globulin IV on days -2 and -1. Patients then undergo autologous or syngeneic stem cell transplant on day 0. Patients also receive prednisone PO QD on days 7-21, followed by 2 week taper.
Sponsors
Leads: Fred Hutchinson Cancer Center

This content was sourced from clinicaltrials.gov