Trial Outcomes & Findings for Maximizing Outcome of Multiple Sclerosis Transplantation (NCT NCT03342638)
NCT ID: NCT03342638
Last Updated: 2021-01-11
Results Overview
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
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
66 participants
Primary outcome timeframe
5 years
Results posted on
2021-01-11
Participant Flow
Participant milestones
| Measure |
Control Arm
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
|
IVIg Arm
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 Study
STARTED
|
34
|
32
|
|
Overall Study
COMPLETED
|
33
|
31
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Control Arm
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
|
IVIg Arm
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 Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
Maximizing Outcome of Multiple Sclerosis Transplantation
Baseline characteristics by cohort
| Measure |
Control Arm
n=34 Participants
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
|
IVIg Arm
n=32 Participants
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
n=66 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
36.3 years
n=5 Participants
|
35.3 years
n=7 Participants
|
35.8 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
56 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=5 Participants
|
32 participants
n=7 Participants
|
66 participants
n=5 Participants
|
|
Diagnosis of Multiple Sclerosis using revised McDonald criteria
|
34 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
66 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 5 yearsPopulation: Study Terminated due to PI taking Sabbatical: Data Not Collected
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
Outcome measures
Outcome data not reported
Adverse Events
Control Arm
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
IVIg Arm
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place