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Autoimmune DiseasePhase 2NCT00716066

Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases

A Phase 2 study testing Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Carmustine in people with Autoimmune Disease and Neurologic Autoimmune Disease, run by Fred Hutchinson Cancer Center at 3 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 71 years
What is being tested?
Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Carmustine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
53 participants
Where
3 sitesUnited States
Study dates
Jun 2008to 31 Jan 2030
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 71 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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)
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  • 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)

Who cannot join

  • Age >= 71 years
  • 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%
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  • 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 locations and contacts

3 sites in 1 country.

United States3 sites

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

  • Swedish Medical Center-First Hill

    Seattle, Washington, 98122-4307

Other trials in United States

About this study

Official title: 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

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.

Read the detailed description

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 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.

Being tested
Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation, Carmustine, Cytarabine, Etoposide
Keywords
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
Medical subject headings
Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Stiff-Person Syndrome, Vasculitis, Central Nervous System, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Lambert-Eaton Myasthenic Syndrome, Myasthenia Gravis, Neuromyelitis Optica

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Anti-Thymocyte GlobulinBiological

    Also called Antithymocyte Globulin, Antithymocyte Serum, ATG, ATGAM

    Given IV

  • Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Autologous Stem Cell Transplantation

    Undergo autologous or syngeneic stem cell transplantation

  • CarmustineDrug

    Also called BCNU, Becenum, Becenun, BiCNU

    Given IV

  • CytarabineDrug

    Also called .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone

    Given IV

  • EtoposideDrug

    Also called Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar

    Given IV

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • MelphalanDrug

    Also called Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard

    Given IV

  • Peripheral Blood Stem Cell TransplantationProcedure or surgery

    Also called PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation

    Undergo autologous or syngeneic stem cell transplantation

  • PrednisoneDrug

    Also called .delta.1-Cortisone, 1, 2-Dehydrocortisone, Adasone, Cortancyl

    Given PO

  • Syngeneic Bone Marrow TransplantationProcedure or surgery

    Undergo syngeneic bone marrow transplantation

Study groups in NCT00716066
Study groupRoleWhat they receive
Treatment (immunosuppressive therapy followed by transplant)ExperimentalPatients 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.
Receives: Biological: Anti-Thymocyte Globulin; Procedure: Autologous Hematopoietic Stem Cell Transplantation; Drug: Carmustine; Drug: Cytarabine; Drug: Etoposide; Other: Laboratory Biomarker Analysis; Drug: Melphalan; Procedure: Peripheral Blood Stem Cell Transplantation; Drug: Prednisone; Procedure: Syngeneic Bone Marrow Transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of grades 4-5 regimen-related toxicity

    Assessed by the Regimen Related Toxicity Scale. Using the National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0. The development of a grade 4 to 5 toxicity of any of the included major organ systems within the first 365 days after transplant will be defined as regimen-related toxicity.

    Measured: Up to 1 year post-transplant

Secondary outcomes (5)

  1. Transplant-related mortality

    Defined as death within the first 100 days of transplant due to transplant-related complications.

    Measured: Within 100 days post-transplant

  2. Disease responses

    Assessed by clinical, laboratory and radiologic evaluation

    Measured: Up to 5 years

  3. Engraftment kinetics

    Monitored for engraftment kinetics of granulocytes, platelets and red cells post-transplant.

    Measured: Over first 60 days post-transplant

  4. Number of subjects achieving greater than or equal to 4.0 x 10^6 CD34+ cells/kg, after up to two peripheral blood stem cell mobilizations

    Efficacy of peripheral blood stem cell mobilization as evaluated by total number of harvested CD34+cells/kg, for autologous transplant.

    Measured: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)

  5. Number of subjects with an exacerbation of autoimmune disease symptoms secondary to G-CSF (filgrastim) during peripheral blood stem cell mobilization

    Subjects are evaluated by standardized clinical neurologic tests specific to autoimmune disease type.

    Measured: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jul 2008 (estimated)
  2. Study startJun 2008
  3. Primary completion30 Dec 2025
  4. Study completion31 Jan 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
53 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00716066
Sponsor's protocol ID
2260.00
Other IDs
NCI-2010-00403, 2260.00, RG9213030
Status last verified
Feb 2026
First submitted
15 Jul 2008
Last updated
27 Feb 2026
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Carmustine in people with Autoimmune Disease and Neurologic Autoimmune Disease, run by Fred Hutchinson Cancer Center at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of grades 4-5 regimen-related toxicity (time frame: Up to 1 year post-transplant). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00716066.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2026.

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