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Maximizing Outcome of Multiple Sclerosis Transplantation (MOST)

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ClinicalTrials.gov Identifier: NCT03342638
Recruitment Status : Terminated (PI Sabbatical)
First Posted : November 17, 2017
Results First Posted : January 11, 2021
Last Update Posted : January 11, 2021
Sponsor:
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, Relapsing-Remitting
Interventions Drug: Cyclophosphamide
Drug: Mesna
Drug: rATG
Drug: Methylprednisolone
Drug: G-CSF
Biological: IVIg
Biological: Autologous Stem Cells
Enrollment 66
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Control Arm IVIg Arm
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) will be administered post-transplant.

Cyclophosphamide: Potent immunosuppressive agent; an alkylating agent

Mesna: Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

rATG: A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

Autologous Stem Cells: Infusion of participant's own stem cells

Hematopoietic Stem Cell Therapy performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. IVIg and G-CSF administered post-transplant.

Cyclophosphamide:immunosuppressive agent;alkylating agent

Mesna: Prophylaxis decrease for hemorrhagic cystitis

rATG: lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes & stem cells and release them into the bloodstream

IVIg: Immunoglobulin G (IgG) products manufactured from pooled human plasma and typically contain more than 95% unmodified IgG, which has intact Fc-dependent effector functions and only trace amounts of immunoglobulin A (IgA) or immunoglobulin M (IgM).

Autologous Stem Cells: Infusion of one's own stem cells

Period Title: Overall Study
Started 34 32
Completed 33 31
Not Completed 1 1
Reason Not Completed
Withdrawal by Subject             1             0
Physician Decision             0             1
Arm/Group Title Control Arm IVIg Arm Total
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) will be administered post-transplant.

Cyclophosphamide: Potent immunosuppressive agent; an alkylating agent

Mesna: Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

rATG: A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

Autologous Stem Cells: Infusion of participant's own stem cells

Hematopoietic Stem Cell Therapy performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. IVIg and G-CSF administered post-transplant.

Cyclophosphamide:immunosuppressive agent;alkylating agent

Mesna: Prophylaxis decrease for hemorrhagic cystitis

rATG: lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes & stem cells and release them into the bloodstream

IVIg: Immunoglobulin G (IgG) products manufactured from pooled human plasma and typically contain more than 95% unmodified IgG, which has intact Fc-dependent effector functions and only trace amounts of immunoglobulin A (IgA) or immunoglobulin M (IgM).

Autologous Stem Cells: Infusion of one's own stem cells

Total of all reporting groups
Overall Number of Baseline Participants 34 32 66
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 34 participants 32 participants 66 participants
36.3
(23 to 50)
35.3
(20 to 48)
35.8
(20 to 50)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 32 participants 66 participants
Female
24
  70.6%
19
  59.4%
43
  65.2%
Male
10
  29.4%
13
  40.6%
23
  34.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 32 participants 66 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
2
   5.9%
0
   0.0%
2
   3.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   5.9%
2
   6.3%
4
   6.1%
White
28
  82.4%
28
  87.5%
56
  84.8%
More than one race
0
   0.0%
1
   3.1%
1
   1.5%
Unknown or Not Reported
2
   5.9%
1
   3.1%
3
   4.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 34 participants 32 participants 66 participants
34 32 66
Diagnosis of Multiple Sclerosis using revised McDonald criteria  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 32 participants 66 participants
34
 100.0%
32
 100.0%
66
 100.0%
1.Primary Outcome
Title Efficacy - Rate of Disease Activity
Hide Description Defined as no relapse (defined as acute neurologic deterioration occurring after engraftment and lasting more than 24 hours, accompanied by objective worsening on neurological examination that are documented by a neurologist and not explained by fever, infection, stress, heat or related pseudoexacerbation; supportive confirmation by enhancement on MRI is preferred), no disease progression (defined as a 1.0-point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 6 months apart and not due to a non-MS disease process), and no new or enhancing lesions on MRI
Time Frame 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
Study Terminated due to PI taking Sabbatical: Data Not Collected
Arm/Group Title Control Arm IVIg Arm
Hide Arm/Group Description:

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) will be administered post-transplant.

Cyclophosphamide: Potent immunosuppressive agent; an alkylating agent

Mesna: Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

rATG: A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

Autologous Stem Cells: Infusion of participant's own stem cells

Hematopoietic Stem Cell Therapy performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. IVIg and G-CSF administered post-transplant.

Cyclophosphamide:immunosuppressive agent;alkylating agent

Mesna: Prophylaxis decrease for hemorrhagic cystitis

rATG: lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes & stem cells and release them into the bloodstream

IVIg: Immunoglobulin G (IgG) products manufactured from pooled human plasma and typically contain more than 95% unmodified IgG, which has intact Fc-dependent effector functions and only trace amounts of immunoglobulin A (IgA) or immunoglobulin M (IgM).

Autologous Stem Cells: Infusion of one's own stem cells

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame up to 1 year post transplant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Control Arm IVIg Arm
Hide Arm/Group Description

Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) will be administered post-transplant.

Cyclophosphamide: Potent immunosuppressive agent; an alkylating agent

Mesna: Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

rATG: A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

Autologous Stem Cells: Infusion of participant's own stem cells

Hematopoietic Stem Cell Therapy performed as follows: Autologous stem cells will be infused after conditioning with cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. IVIg and G-CSF administered post-transplant.

Cyclophosphamide:immunosuppressive agent;alkylating agent

Mesna: Prophylaxis decrease for hemorrhagic cystitis

rATG: lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens found on the surface of T cells

Methylprednisolone: Steroid

G-CSF: Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes & stem cells and release them into the bloodstream

IVIg: Immunoglobulin G (IgG) products manufactured from pooled human plasma and typically contain more than 95% unmodified IgG, which has intact Fc-dependent effector functions and only trace amounts of immunoglobulin A (IgA) or immunoglobulin M (IgM).

Autologous Stem Cells: Infusion of one's own stem cells

All-Cause Mortality
Control Arm IVIg Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   0/33 (0.00%)   0/31 (0.00%) 
Hide Serious Adverse Events
Control Arm IVIg Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   0/33 (0.00%)   0/31 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Control Arm IVIg Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   0/33 (0.00%)   0/31 (0.00%) 
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: NCT03342638    
Other Study ID Numbers: DIAD.MOST.2017
First Submitted: November 9, 2017
First Posted: November 17, 2017
Results First Submitted: October 30, 2020
Results First Posted: January 11, 2021
Last Update Posted: January 11, 2021