Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases

Study Purpose

This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a peripheral blood 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 peripheral blood 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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Eligible Ages N/A - 71 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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.
  • - 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)

    Exclusion Criteria:

    - Pregnancy or expressed plans to become pregnant within 1 year of the procedure.
  • - Patients who are serologically positive for human immunodeficiency virus (HIV) - Patients with pulmonary, cardiac, hepatic or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this should include patients with any of the following: - Severe pulmonary dysfunction associated with a carbon monoxide diffusing capacity (DLCO) (corrected for hemoglobin) < 60%, or requires supplemental oxygen; patients who are unable to perform pulmonary function test (because of underlying disease) will be excluded if the oxygen saturation is < 92% on room air.
  • - Uncontrolled malignant arrhythmias, or clinical evidence of congestive heart failure (New York class III-IV) or ejection fraction < 50% - Renal disease with estimated glomerular filtration rate (GFR) by creatinine clearance or iothalamate clearance < 50 ml/min/1.73 m^2 body surface area.
  • - Serum glutamate pyruvate transaminase (SGPT)/aspartate aminotransferase (AST) > 3 times normal or direct bilirubin greater than 2.5 mg/dL on two repeated tests.
  • - Active uncontrolled infection.
  • - Demonstrated lack of compliance with prior medical care.
  • - Patients whose life expectancy is limited by illness other than their neurological condition.
  • - Patients with evidence of myelodysplasia.
  • - Active malignancy (excluding localized squamous cell or basal cell carcinoma of the skin) - DONOR: Inadequate documentation that donor and recipient are syngeneic.
  • - DONOR: Donors who do not fulfill criteria as apheresis donors as established by institutional guidelines.
- DONOR: Concordant for autoimmune neurological disease(s) as determined by neurological evaluation

Trial Details

Trial ID:

This trial id was obtained from, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Fred Hutchinson Cancer Research Center
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

George Georges
Principal Investigator Affiliation Fred Hutch/University of Washington Cancer Consortium
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Recruiting
Countries United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Autoimmune Disease, Neurologic Autoimmune Disease, Autologous Transplant Autoimmune, Multiple Sclerosis Transplant, MS Stem Cell Transplant, Multiple Sclerosis Stem Cell Transplant, Stiff Person Syndrome, HCT for Neurologic Autoimmune Disorders, CIDP Transplant, Myasthenia Gravis Transplant, Autoimmune Nervous System Disorder, Central Nervous System Vasculitis, Cerebellar Degeneration, Chronic Inflammatory Demyelinating Polyneuropathy, Lambert Eaton Myasthenic Syndrome, Myasthenia Gravis, Neuromyelitis Optica, Opsoclonus Myoclonus Syndrome, Rasmussen Subacute Encephalitis
Additional Details


  • I. Evaluate the safety of high-dose carmustine, etoposide, Ara-c (cytarabine) and melphalan (BEAM) and Thymoglobulin (antithymocyte globulin) as a high-dose immunosuppressive treatment (HDIT) regimen in patients with severe, refractory neurological autoimmune disease.
  • I. Evaluate disease responses and the duration of response to HDIT and autologous hematopoietic stem cell transplantation (HSCT).
  • II. Determine the efficacy and safety of G-CSF (filgrastim) and prednisone or cyclophosphamide for hematopoietic stem mobilization in patients with neurological autoimmune diseases.
OUTLINE: Patients receive carmustine intravenously (IV) on day -6, etoposide IV and cytarabine IV twice daily (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 peripheral blood stem cell transplant on day 0. Patients also receive prednisone orally (PO) once daily (QD) on days 7-21, followed by 2 week taper. After completion of study treatment, patients are followed up at 3 months, 1 year, and then annually thereafter for up to 5 years.

Arms & Interventions


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 peripheral blood stem cell transplant on day 0. Patients also receive prednisone PO QD on days 7-21, followed by 2 week taper.


Biological: - Anti-Thymocyte Globulin

Given IV

Procedure: - Autologous Hematopoietic Stem Cell Transplantation

Undergo autologous or syngeneic peripheral blood stem cell transplantation

Drug: - Carmustine

Given IV

Drug: - Cytarabine

Given IV

Drug: - Etoposide

Given IV

Other: - Laboratory Biomarker Analysis

Correlative studies

Drug: - Melphalan

Given IV

Procedure: - Peripheral Blood Stem Cell Transplantation

Undergo autologous or syngeneic peripheral blood stem cell transplantation

Drug: - Prednisone

Given PO

Procedure: - Syngeneic Bone Marrow Transplantation

Undergo syngeneic bone marrow transplantation

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Colorado Blood Cancer Institute, Denver, Colorado




Colorado Blood Cancer Institute

Denver, Colorado, 80218

Site Contact

Richard A. Nash

[email protected]


Seattle, Washington




Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109

Site Contact

Bernie McLaughlin

[email protected]


Swedish Medical Center-First Hill, Seattle, Washington




Swedish Medical Center-First Hill

Seattle, Washington, 98122-4307

Site Contact

Bernie McLaughlin

[email protected]