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NCT00716066Active, not recruitingPhase 2

Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases

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

Source: ClinicalTrials.gov · Last updated 27 Feb 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Phase (registry)
Phase 2
Study type
Interventional
Enrollment
53 (actual)
First submitted
15 Jul 2008
Start date
Jun 2008
Primary completion date
30 Dec 2025
Lead sponsor
Fred Hutchinson Cancer Center
NCT number
NCT00716066

What you commit to

  • Study type: Interventional · Phase 2
  • Participants enrolled: 53
  • From Jun 2008 to the primary completion date, 30 Dec 2025
  • 3 sites in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Demonstrated lack of compliance with prior medical care

Other illness

  • Patients who are serologically positive for human immunodeficiency virus (HIV)
  • Active uncontrolled infection
  • Patients whose life expectancy is limited by illness other than their neurological condition
  • Active malignancy (excluding localized squamous cell or basal cell carcinoma of the skin)
  • DONOR: Concordant for autoimmune neurological disease(s) as determined by neurological evaluation

Lab values

  • 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

Age

  • Age \>= 71 years

Other

  • Pregnancy or expressed plans to become pregnant within 1 year of the procedure
  • Patients with evidence of myelodysplasia
  • DONOR: Inadequate documentation that donor and recipient are syngeneic
  • DONOR: Donors who do not fulfill criteria as apheresis donors as established by institutional guidelines

Who the study is looking for

  • 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)

Age in the registry: - to 71 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (3)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Colorado Blood Cancer Institute
Coordinator of this site? Confirm its status
DenverUnited StatesUnknown status
Fred Hutch/University of Washington Cancer Consortium
Coordinator of this site? Confirm its status
SeattleUnited StatesUnknown status
Swedish Medical Center-First Hill
Coordinator of this site? Confirm its status
SeattleUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.