Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases

  • STATUS
    Recruiting
  • End date
    Dec 1, 2027
  • participants needed
    40
  • sponsor
    Fred Hutchinson Cancer Research Center
Updated on 26 April 2021
cancer
blood stem cell transplant
cytarabine
white blood cell count
melphalan
prednisone
MRI
antithymocyte globulin
etoposide
white blood cells
biomarker analysis
spinal cord
apheresis
carmustine
autoimmune disease
vasculitis
malignancy
polyneuropathy
cell counts
nervous
gait ataxia
ataxia
myelopathy
peripheral neuropathy
polyradiculoneuropathy
encephalitis autoimmune
multiple sclerosis
myoclonus
neuromyelitis optica
stiff person syndrome
syngeneic bone marrow transplantation
cerebellar degeneration
opsoclonus
variable number tandem repeat
anna-1
hematopoietic stem cell transplantation
abo typing
ganglioside
spinal cord disease
paraparesis

Summary

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.

Description

PRIMARY OBJECTIVES:

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.

SECONDARY OBJECTIVES:

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.

Details
Condition Cerebral Vasculitis, Myelopathy, Myasthenia Gravis, Eaton-Lambert Syndrome, Autoimmune disease, Stiff-Man Syndrome, Neuromyelitis Optica, Opsoclonus-Myoclonus Syndrome, nervous system disorder, Autoimmunity, Autoimmune Nervous System Disorder, Chronic inflammatory demyelinating polyradiculoneuropathy, Myasthenia Gravis generalised, Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis (Chronic Weakness), Spinal Cord Disorders, Neurologic Disorders, Autologous Transplant Autoimmune, Multiple Sclerosis Transplant, MS Stem Cell Transplant, Multiple Sclerosis Stem Cell Transplant, HCT for Neurologic Autoimmune Disorders, CIDP Transplant, Myasthenia Gravis Transplant, Cerebellar Degeneration, Rasmussen Subacute Encephalitis, central nervous system vasculitis, autoimmune diseases, autoimmune disorder, autoimmune disorders, lambert-eaton myasthenic syndrome, eaton lambert syndrome, stiff person syndrome
Treatment anti-thymocyte globulin, laboratory biomarker analysis, cytarabine, etoposide, prednisone, melphalan, autologous hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, carmustine, Syngeneic Bone Marrow Transplantation
Clinical Study IdentifierNCT00716066
SponsorFred Hutchinson Cancer Research Center
Last Modified on26 April 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age less than or equal to 71 yrs?
Gender: Male or Female
Do you have any of these conditions: Myasthenia Gravis (Chronic Weakness) or Opsoclonus-Myoclonus Syndrome or Chronic inflammatory demyelinating polyradiculoneuropathy or Autoimmune Nervo...?
Do you have any of these conditions: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) or eaton lambert syndrome or central nervous system vasculitis or Neurologic Disorders or HCT...?
Do you have any of these conditions: Autoimmune disease or Cerebellar Degeneration or Myasthenia Gravis or nervous system disorder or Spinal Cord Disorders or eaton lambert syndrome or Ch...?
Do you have any of these conditions: Autologous Transplant Autoimmune or Cerebellar Degeneration or Myasthenia Gravis Transplant or Cerebral Vasculitis or lambert-eaton myasthenic syndrom...?
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
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