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

September 27, 2016 updated by: Ottawa Hospital Research Institute

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • Ottawa Hospital Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 46 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
3 year MS activity free survival
Time Frame: 3 year follow-up post transplant
3 year follow-up post transplant

Secondary Outcome Measures

Outcome Measure
Time Frame
Transplant related morbidity
Time Frame: 3 year follow-up post transplant
3 year follow-up post transplant
Transplant related mortality
Time Frame: 3 years
3 years
Time to MS treatment failure
Time Frame: 3 years
3 years
rate of immune reconstitution following treatment
Time Frame: 3 years
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ottawa Hospital Research Institute

Collaborators

Multiple Sclerosis Scientific Research Foundation

Investigators

  • Principal Investigator: Mark S Freedman, MD, Ottawa Hospital Research Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2001

Primary Completion (Actual)

November 1, 2012

Study Completion (Actual)

June 1, 2016

Study Registration Dates

First Submitted

March 1, 2010

First Submitted That Met QC Criteria

April 6, 2010

First Posted (Estimate)

April 8, 2010

Study Record Updates

Last Update Posted (Estimate)

September 28, 2016

Last Update Submitted That Met QC Criteria

September 27, 2016

Last Verified

September 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 200037401H

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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