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Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis

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: NCT00497952
Recruitment Status : Active, not recruiting
First Posted : July 9, 2007
Last Update Posted : July 18, 2022
Sponsor:
Information provided by (Responsible Party):
Talaris Therapeutics Inc.

Study Description
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Brief Summary:
The goal of this research study is to establish chimerism with the goal to halt disease progression in patients with Multiple Sclerosis.

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 ...

Detailed Description:

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis
Study Start Date : July 2007
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

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Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Stem cell engraftment [ Time Frame: One month to three years ]

Secondary Outcome Measures :
  1. Disease remission [ Time Frame: 3 Years ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinically definite MS according to the McDonald criteria
  • Confirmed diagnosis of relapsing-remitting MS.
  • Age between 18 and 55 years
  • Extended Disability Status Score (EDSS) between 0 and 5.0
  • Independently ambulatory (eligible for inclusion if subject was acutely non-ambulatory within the previous year and return of function is substantiated with EDSS score.)
  • Relapse within the last year or sustained disability progression of 1.0 for six months
  • Treatment with high dose, high frequency Interferon-β therapy, or failure to tolerate Interferon-β therapy
  • Diffusing capacity of the lung for carbon monoxide (DLCO)> 50% (unless cleared by physician)
  • Ejection fraction (EF) > 40% (unless cleared by cardiologist)

  • Required initial laboratory data (obtained within 30 days prior to transplant, unless otherwise specified)

    • HIV-1,2 antigen and antibody negative
    • HBsAg negative (chronic hepatitis B carriers without clinical evidence of liver disease can be considered on an individual basis if it is determined that the added risk is justified by the prognosis and lack of treatment alternatives)
    • Hepatitis C antibody negative (positive antibody allowed if antigen (RNA)-negative and no clinical evidence of cirrhosis)
    • Cytomegalovirus (CMV), hepatitis B, Human T-lymphotropic virus (HTLV)-1,2, Epstein-Barr virus (EBV), and Herpes antibody status known
    • Pregnancy test negative (women of childbearing potential only)

  • No life-threatening organ dysfunction.

    • Uncontrolled or severe cardiovascular disease, including recent (<6 months) myocardial infarction, angina (symptomatic despite optimal medical management), life-threatening arrhythmia or hypertension
  • Able to give informed consent

Exclusion Criteria:

  • Women who are of child bearing potential must have a negative pregnancy test (serum pregnancy test - human chorionic gonadotrophin (HCG)) within 48 hours of initiating total body irradiation and agree to use reliable contraception for 1 year following transplant.
  • Concomitant severe diseases (respiratory, renal, liver, cardiac failures, psychiatric disorders, neoplasms)
  • Recurrent urinary, pulmonary infections.
  • Active bacterial, viral, or fungal infection
  • Active peptic ulcer disease
  • Previous treatments with total lymphoid irradiation or total body irradiation
  • Interferon-neutralizing antibody positive with a titer greater than 20
  • Relapse in the month preceding enrollment
  • Poor compliance
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): NCT00497952


Sponsors and Collaborators
Talaris Therapeutics Inc.
Investigators
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Study Director: Suzanne T Ildstad, MD Talaris Therapeutics Inc.
More Information
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Responsible Party: Talaris Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT00497952    
Other Study ID Numbers: ICT-13080-122206
First Posted: July 9, 2007    Key Record Dates
Last Update Posted: July 18, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Talaris Therapeutics Inc.:
Relapsing-remitting multiple sclerosis
Marrow/Stem cell transplant
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