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Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study

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: NCT00273364
Recruitment Status : Completed
First Posted : January 9, 2006
Results First Posted : August 12, 2020
Last Update Posted : August 12, 2020
Sponsor:
Collaborators:
Uppsala University
Sheffield Teaching Hospitals NHS Foundation Trust
University of Sao Paulo
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Multiple Sclerosis
Interventions Procedure: Hematopoietic Stem Cell Therapy
Drug: Standard treatment with a conventional drug
Enrollment 110
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG

Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen

Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)

Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)

Period Title: Overall Study
Started 55 55
Completed 55 55
Not Completed 0 0
Arm/Group Title Hematopoietic Stem Cell Transplantation Standard Therapy for MS Total
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG

Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen

Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)

Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)

Total of all reporting groups
Overall Number of Baseline Participants 55 55 110
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 55 participants 55 participants 110 participants
35.6
(18 to 54)
35.6
(19 to 52)
35.6
(18 to 54)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 55 participants 110 participants
Female
34
  61.8%
39
  70.9%
73
  66.4%
Male
21
  38.2%
16
  29.1%
37
  33.6%
Expanded Disability Status Scale (EDSS)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 52 participants 54 participants 106 participants
3.4  (1.2) 3.3  (1.0) 3.35  (.05)
[1]
Measure Description: The EDSS provides a total disability score on a scale that ranges from 0 (no disability) to 10 (worst neurologic disability).
[2]
Measure Analysis Population Description: The number analyzed in rows differs from the overall enrolled because EDSS were not analyzed on patients who did not receive intervention or who were lost to follow up.
1.Primary Outcome
Title Expanded Disability Status Scale (EDSS) Improvement
Hide Description The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability. Improvement in EDSS is defined by both a 0.5 or 1.0 points sustained for more than 6 months.
Time Frame Pre Treatment, 6 and 12 months Post Treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed in one or more rows differs from the overall number analyzed because not all patients had their EDSS evaluated and some patients were lost to followed up.
Arm/Group Title Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Hide Arm/Group Description:

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG

Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen

Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)

Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)

Overall Number of Participants Analyzed 52 54
Mean (Standard Deviation)
Unit of Measure: units on a scale
Pre Treatment Number Analyzed 52 participants 54 participants
3.4  (1.2) 3.3  (1.0)
6 Months Post Treatment Number Analyzed 52 participants 54 participants
2.5  (1.4) 3.7  (1.5)
12 Months Post Treatment Number Analyzed 50 participants 48 participants
2.4  (1.4) 4  (1.7)
Time Frame Adverse events were collected during transplant and at 1 year post Treatment
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG

Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen

Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)

Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)

All-Cause Mortality
Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Affected / at Risk (%) Affected / at Risk (%)
Total   0/52 (0.00%)      0/55 (0.00%)    
Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/52 (0.00%)      0/55 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Hematopoietic Stem Cell Transplantation Standard Therapy for MS
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   17/52 (32.69%)      0/55 (0.00%)    
General disorders     
Febrile Neutropenia (culture negative)   13/52 (25.00%)  13 0/55 (0.00%)  0
Metabolism and nutrition disorders     
Hypophosphatemia   17/52 (32.69%)  17 0/55 (0.00%)  0
Hypokalemia   13/52 (25.00%)  13 0/55 (0.00%)  0
Hypergylcemia   5/52 (9.62%)  5 0/55 (0.00%)  0
Indicates events were collected by systematic assessment
Limitations to the study are the relatively small number of patients who were treated compared with pharmaceutical sponsored trials, and the relatively small sample size resulted in small numbers of patients available to assess longer term outcomes.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Kathleen Quigley
Organization: Northwestern University
Phone: 312-695-8192
EMail: k-quigley@northwestern.edu
Layout table for additonal information
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00273364    
Obsolete Identifiers: NCT03133403
Other Study ID Numbers: DI MS.Randomized2004
First Submitted: January 5, 2006
First Posted: January 9, 2006
Results First Submitted: July 15, 2020
Results First Posted: August 12, 2020
Last Update Posted: August 12, 2020