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Autologous Stem Cell Transplant for Multiple Sclerosis (MS/BMT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01099930
Recruitment Status : Completed
First Posted : April 8, 2010
Last Update Posted : September 28, 2016
Sponsor:
Collaborator:
Multiple Sclerosis Scientific Research Foundation
Information provided by (Responsible Party):
Ottawa Hospital Research Institute

Study Description
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Brief Summary:
Multiple sclerosis is an autoimmune disease. We are studying whether high dose chemotherapy and autologous stem cell transplant can replace the autoreactive immune system and if this reduces clinical inflammatory disease in the central nervous system (CNS). A second goal is to examine whether there is long-term stabilization or improvement in disability scores if the inflammatory disease is controlled.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Other: immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT), Other: Standard Therapy Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Targeting Multiple Sclerosis as an Autoimmune Disease With Intensive Immunoablative Therapy and Immunological Reconstitution: A Potential Curative Therapy for Patients With a Predicted Poor Prognosis
Study Start Date : August 2001
Actual Primary Completion Date : November 2012
Actual Study Completion Date : June 2016

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Non-randomized control group
Patients meeting inclusion/exclusion criteria not consenting to treatment will be requested to consent to control group and followed while receiving standard of care.
 Other: Standard Therapy
Patient will receive standard therapy as decided upon by their treating neurologist.
Experimental: Stem Cell Transplantation
Patients will undergo stem cell transplantation for the treatment of Multiple Sclerosis
 Other: immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT),

Stem Cell Mobilization with Cyclophosphamide 4.5 gm/m2 and rhGCSF 10 ug/kg/d x 10 day.

Stem Cell Collection with Cobe Spectra Stem Cell Purification with Miltenyi CliniMACS Stem Cell Transplant Conditioning with Busulphan 9.6 mg/kg iv, Cyclophosphamide 200 mg/kg iv, rabbit ATG 5 mg/kg iv followed by CD34 selected autologous hematopoietic stem cell transplant


Outcome Measures
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Primary Outcome Measures :
  1. 3 year MS activity free survival [ Time Frame: 3 year follow-up post transplant ]

Secondary Outcome Measures :
  1. Transplant related morbidity [ Time Frame: 3 year follow-up post transplant ]
  2. Transplant related mortality [ Time Frame: 3 years ]
  3. Time to MS treatment failure [ Time Frame: 3 years ]
  4. rate of immune reconstitution following treatment [ Time Frame: 3 years ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age between 18 and 50 years
  • The diagnosis of active multiple sclerosis with relapses or progression and sustained accumulated impairment, made by a neurologist expert in the field
  • Patient considered at high risk of progression
  • EDSS Cerebellar Functional score greater than or equal to 3 OR EDSS Pyramidal Functional score greater than or equal to 3
  • EDSS between greater than or equal to 3 and less than or equal to 6
  • Evidence of current disease activity
  • Evidence of progression or continued relapses or worsening MRI after at least one year of therapy with interferon-B1, glatiramer acetate, Mitoxantrone, or other conventional dose immunosuppressive drug therapy
  • If a patient has previously received a cytotoxic agent (Mitoxantrone, Cyclophosphamide etc.) they must have normal bone marrow morphology and cytogenetics before being considered eligible for this study
  • MRI brain scan that satisfies the MRI criteria of Paty or Fazekas for the diagnosis of multiple sclerosis
  • No evidence of hepatic inflammation or fibrosis

Exclusion Criteria:

  • Patients with primary progressive multiple sclerosis
  • Patients with cardiac, renal, pulmonary, hepatic or other organ impairment that would limit their ability to receive dose intensive immunosuppressive therapy including high dose chemotherapy and ASCT
  • Patient with any active or chronic infection
  • Patients who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C
  • Patients with a previous history of a malignancy other than basal cell carcinoma of the skin or carcinoma in situ that has been in remission for more than one year
  • Patients whose life expectancy is severely limited by another co-morbid illness
  • Patients with evidence of myelodysplasia or other non-autoimmune cytopenia
  • Patients having received a cytotoxic agent within one month of enrolling in this study
  • Pregnancy or risk of pregnancy. This includes patients that are unwilling to practice active contraception during the time of chemotherapy
  • Patients with hypersensitivity to rabbit proteins
  • Patients unable to give written informed consent in accordance with research ethics board guidelines
  • Patients having previous exposure to natalizumab or alemtuzumab.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01099930


Locations
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Canada, Ontario
Ottawa Hospital Research Institute
Ottawa, Ontario, Canada, K1H 8L6
Sponsors and Collaborators
Ottawa Hospital Research Institute
Multiple Sclerosis Scientific Research Foundation
Investigators
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Principal Investigator: Harold L Atkins, MD Ottawa Hospital Research Institute
Principal Investigator: Mark S Freedman, MD Ottawa Hospital Research Institute
More Information
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Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Ottawa Hospital Research Institute
ClinicalTrials.gov Identifier: NCT01099930    
Other Study ID Numbers: 200037401H
First Posted: April 8, 2010    Key Record Dates
Last Update Posted: September 28, 2016
Last Verified: September 2016
Keywords provided by Ottawa Hospital Research Institute:
immunoablation
stem cell transplantation
Additional relevant MeSH terms:
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Multiple Sclerosis
Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
 Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases