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Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica (SCT-NMO)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Jodie Burton MD, MSc, FRCPC, University of Calgary Identifier:
First received: April 18, 2011
Last updated: December 30, 2015
Last verified: December 2015
Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity. Based on recent outcomes of stem cell transplant trials and reports in autoimmune diseases including MS, and based on the mechanisms of NMO, we anticipate that stem cell transplantation may provide lasting disease stability for NMO patients. The hypothesis of the present trial is that autologous hematopoetic stem cell transplantation in patients with NMO will provide lasting benefit in relapse prevention. Specifically, we anticipate a 50% reduction in the proportion of patients experiencing relapse over a three year period. We will be following patients for a total of five years after transplantation.

Condition Intervention Phase
Neuromyelitis Optica
Procedure: AHSCT
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica

Resource links provided by NLM:

Further study details as provided by University of Calgary:

Primary Outcome Measures:
  • Proportion relapse-free at three years [ Time Frame: 3 years ]
    The proportion of surviving patients who are relapse-free at three years after transplant

Secondary Outcome Measures:
  • Proportion relapse-free at five years [ Time Frame: 5 years ]
    The proportion of surviving patients relapse-free at year five

  • Relapse count [ Time Frame: Annually over 5 years ]
    Number of NMO relapse events

  • Disability progression [ Time Frame: Over 5 years ]
    Time to progression of EDSS by one step

  • Retinal nerve fiber layer (RFNL) status [ Time Frame: 5 years ]
    Change in RNFL by optical coherence tomography over trial

  • 25 foot timed walk test [ Time Frame: 5 years ]
    Change in 25 ft timed walk test over trial

  • PASAT [ Time Frame: Annually over 5 years ]
    Annual and change from baseline to end of trial in Paced Auditory Serial Addition Test to assess cognitive function.

  • Hospitalization [ Time Frame: Over 5 years ]
    Number of hospitalizations, days in hospital over trial period

  • Overall survival [ Time Frame: Over 5 years ]
    Survival over trial period

  • Time to next relapse [ Time Frame: Over 5 years ]
    Time to next relapse after transplant

Enrollment: 3
Study Start Date: March 2011
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: March 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AHSCT
All patients undergo autologous hematopoietic stem cell transplantation in a two stage process.
Procedure: AHSCT

AHSCT Procedure:

  1. Mobilization and Harvesting:

    • Cyclophosphamide
    • Rituximab
    • GSCF
    • Dexamethasone
    • Apheresis
  2. Conditioning and Infusion (3-4 weeks after Mobilization and Harvesting):

    • Cyclophosphamide
    • MESNA
    • Rabbit ATG
    • Rituximab
    • Methylprednisolone
    • Stem Cell infusion
    • GSCF

Detailed Description:
Patients who are deemed eligible will be enrolled and undergo a two stage transplant process followed by neurological assessments every 6 months for the following 5 years assessing EDSS, visual metrics, MRI, AQP-4 antibodies, MSFC and SF36.

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age between 18-65, inclusive
  • Diagnosis of NMO using Wingerchuk 2006 NMO Criteria
  • EDSS 0-6.5
  • Treatment with a minimum of one NMO therapy in past 12 months
  • One objective and documented relapse in the past 12 months and two relapse events in the past 24 months despite medical therapy
  • ECOG performance status 0-3
  • Platelets ≥100 x 109/L
  • ALT ≤3 x ULN
  • Total bilirubin ≤2.0 x ULN, except in patients with Gilbert syndrome or in patients in whom the bilirubin rise is of non-hepatic origin
  • Serum creatinine <1.5 x ULN or creatinine clearance ≥50 cc/min
  • Patients must reside in Alberta, Canada for the duration of the transplant period of the trial

Exclusion Criteria:

  • Any illness that would jeopardize the ability of the patient to complete study protocol
  • Prior malignancy unless non-melanoma skin cancer, carcinoma in-situ of the cervix (CIN) or breast, or malignancy treated more than 5 years previously with no evidence of recurrent disease since initial treatment
  • Pregnant or lactating females. Women of childbearing potential must have a negative serum or urine β-hCG pregnancy test at screening
  • Inability or unwillingness to pursue effective means of birth control
  • FEV1/FVC < 50% of predicted
  • DLCO < 50% of predicted
  • Resting LVEF < 50 %
  • Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications
  • Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams
  • Unable or unwilling to provide written informed consent for participation
  • Active infection except asymptomatic bacteriuria
  • Any use of investigational therapies within 4 weeks prior to initiation of study treatment
  • Patients dependent on prednisone who cannot be successfully tapered to a maximum of 0.5mg/kg/d prior to mobilization therapy
  Contacts and Locations
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Please refer to this study by its identifier: NCT01339455

Canada, Alberta
Foothills Medical Centre, University of Calgary
Calgary, Alberta, Canada, T2N 2T9
Sponsors and Collaborators
University of Calgary
Principal Investigator: Jodie M Burton, MD,MSc,FRCPC Department of Clinical Neurosciences, Hotchkiss Brain Institute, University of Calgary
Principal Investigator: Jan Storek, MD,PhD Department of Medicine, University of Calgary
  More Information

Responsible Party: Jodie Burton MD, MSc, FRCPC, Assistant Professor, University of Calgary, University of Calgary Identifier: NCT01339455     History of Changes
Other Study ID Numbers: CHREB ID# 23282
Study First Received: April 18, 2011
Last Updated: December 30, 2015

Keywords provided by University of Calgary:
Neuromyelitis Optica
Devic's Disease
Stem Cell Transplant

Additional relevant MeSH terms:
Neuromyelitis Optica
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Optic Neuritis
Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
Eye Diseases
Autoimmune Diseases
Immune System Diseases processed this record on April 28, 2017