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. Identifier: NCT01099930
Recruitment Status : Completed
First Posted : April 8, 2010
Last Update Posted : September 28, 2016
Multiple Sclerosis Scientific Research Foundation
Information provided by (Responsible Party):
Ottawa Hospital Research Institute

March 1, 2010
April 8, 2010
September 28, 2016
August 2001
November 2012   (Final data collection date for primary outcome measure)
3 year MS activity free survival [ Time Frame: 3 year follow-up post transplant ]
Same as current
Complete list of historical versions of study NCT01099930 on Archive Site
  • Transplant related morbidity [ Time Frame: 3 year follow-up post transplant ]
  • Transplant related mortality [ Time Frame: 3 years ]
  • Time to MS treatment failure [ Time Frame: 3 years ]
  • rate of immune reconstitution following treatment [ Time Frame: 3 years ]
Same as current
Not Provided
Not Provided
Autologous Stem Cell Transplant for Multiple Sclerosis
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
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.
Not Provided
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

  • Other: Standard Therapy
    Patient will receive standard therapy as decided upon by their treating neurologist.
  • 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.
    Intervention: Other: Standard Therapy
  • Experimental: Stem Cell Transplantation
    Patients will undergo stem cell transplantation for the treatment of Multiple Sclerosis
    Intervention: Other: immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT),
Atkins HL, Bowman M, Allan D, Anstee G, Arnold DL, Bar-Or A, Bence-Bruckler I, Birch P, Bredeson C, Chen J, Fergusson D, Halpenny M, Hamelin L, Huebsch L, Hutton B, Laneuville P, Lapierre Y, Lee H, Martin L, McDiarmid S, O'Connor P, Ramsay T, Sabloff M, Walker L, Freedman MS. Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial. Lancet. 2016 Aug 6;388(10044):576-85. doi: 10.1016/S0140-6736(16)30169-6. Epub 2016 Jun 9.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
June 2016
November 2012   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
18 Years to 50 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
Not Provided
Not Provided
Ottawa Hospital Research Institute
Ottawa Hospital Research Institute
Multiple Sclerosis Scientific Research Foundation
Principal Investigator: Harold L Atkins, MD Ottawa Hospital Research Institute
Principal Investigator: Mark S Freedman, MD Ottawa Hospital Research Institute
Ottawa Hospital Research Institute
September 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP