Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis
|ClinicalTrials.gov Identifier: NCT00497952|
Recruitment Status : Active, not recruiting
First Posted : July 9, 2007
Last Update Posted : May 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|Multiple Sclerosis||Biological: hematopoetic stem cell infusion||Phase 1 Phase 2|
Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
While the cause of MS in not known, there is an autoimmune component that destroys nerve cells. Autoimmunity is a condition where an individual's immune system attacks his/her own cells. Bone marrow stem cell transplantation has been shown to halt autoimmunity. Stem cell transplant can be performed using the patient's own cells, or donor cells. The general consensus in the field is that donor transplant is most likely to halt disease progression. This study is designed to evaluate the safety of a donor transplant procedure as a therapy for relapsing remitting multiple sclerosis (RRMS).
Two factors limit the widespread application of traditional donor stem cell transplant: 1) preparing the patient for transplant (conditioning); and 2) graft-versus-host disease (GVHD). Traditional conditioning destroys the recipient's immune system and requires that the marrow transplant be successful because the patient is unable to fight off infection if the donor cells do not survive. GVHD occurs when donor immune cells recognize the recipient's cells as foreign tissue and attack them. Severe GVHD can result in death. This study utilizes a new approach to conditioning which leaves the patient's immune system intact. The transplant product is depleted of GVHD-producing cells but retains tolerance-promoting cells, called facilitating cells, which are intended to ensure the donor and recipient cells coexists peacefully. The toxicity of conditioning and transplantation is significantly reduced. The end result is a marrow system that contains recipient and donor cells, a state called mixed chimerism.
In this study, we will determine the appropriate cell dose to safely establish mixed chimerism following partial conditioning in patients with RRMS. The study takes a gradual approach to increasing the cell dose to achieve mixed chimerism. Each patient will receive a cell dose one unit above the dose received by the most recent safely transplanted patient. We believe this study will provide a breakthrough in the treatment of MS. The goal of this study is to evaluate the potential of safely establishing mixed chimerism to interrupt the autoimmune process and end the devastating effects of MS.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis|
|Study Start Date :||July 2007|
|Estimated Primary Completion Date :||December 2018|
|Estimated Study Completion Date :||December 2020|
Experimental: Multiple Sclerosis Patients
Recipients treated with a hematopoetic stem cell infusion from a living donor
Biological: hematopoetic stem cell infusion
Enriched hematopoietic stem cell infusion
- Stem cell engraftment [ Time Frame: One month to three years ]
- Disease remission [ Time Frame: 3 Years ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00497952
|Study Director:||Suzanne T Ildstad, MD||University of Louisville|