Skip to main content
OpenTrials
Completed

NCT Number: NCT01099930

Autologous Stem Cell Transplant for Multiple Sclerosis

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Multiple Sclerosis Autoimmune Diseases Autoimmune Diseases of the Nervous System Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Immune System Diseases Nervous System Diseases

Age range

18 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Ottawa Hospital Research Institute

Ottawa, Ontario, K1H 8L6, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT),

Other

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

Standard therapy

Other

Patient will receive standard therapy as decided upon by their treating neurologist.

Primary outcomes

  1. 3 year MS activity free survival

    Time frame: 3 year follow-up post transplant

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

Ottawa Hospital Research Institute

Other

Collaborators

  • Multiple Sclerosis Scientific Research Foundation

Registry information

Official study 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

Acronym: MS/BMT

Important dates

Study start
2001
Primary completion
2012
Study completion
2016
First posted
Apr 8, 2010
Registry last updated
Sep 28, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.