Trial Outcomes & Findings for Stem Cell Transplantation for Stiff Person Syndrome (SPS) (NCT NCT02282514)
NCT ID: NCT02282514
Last Updated: 2021-01-27
Results Overview
Number of Participants who Did Not Experience Treatment-Related Mortality
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
23 participants
Primary outcome timeframe
Mean 3.6 years
Results posted on
2021-01-27
Participant Flow
Participant milestones
| Measure |
Hematopoietic Stem Cell Transplantation
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Stem Cell Transplantation for Stiff Person Syndrome (SPS)
Baseline characteristics by cohort
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 Participants
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
23 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
48.3 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Mean 3.6 yearsNumber of Participants who Did Not Experience Treatment-Related Mortality
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 Participants
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Overall Survival
|
23 participants
|
SECONDARY outcome
Timeframe: Mean 3.6 yearsDecrease (50%) and complete discontinuation of muscle relaxation anti-spasmatic medications
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 Participants
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Reduction of Muscle Relaxation Anti-spasmatic Medications
|
14 Participants
|
SECONDARY outcome
Timeframe: mean 3.6 yearsImprovement is defined as a statistically significant change in SF-36 QOL score. The scale is 0-100. The lower the score the worse quality of life.
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 Participants
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Short-form 36 Quality of Life Questionnaire (SF-36 QOL)
Pre Treatment
|
37.75 units on a scale
Standard Deviation 12.81
|
|
Short-form 36 Quality of Life Questionnaire (SF-36 QOL)
Post Treatment
|
58.35 units on a scale
Standard Deviation 22.81
|
Adverse Events
Hematopoietic Stem Cell Transplantation
Serious events: 1 serious events
Other events: 5 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 participants at risk
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
General disorders
Non Treatment Related Death
|
4.3%
1/23 • Number of events 1 • Up to 4.5 years
|
Other adverse events
| Measure |
Hematopoietic Stem Cell Transplantation
n=23 participants at risk
Conditioning regimen will be 200 mg/kg of IV cyclophosphamide given in 4 equal fractions on days -5 through -2 with IV mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused IV before each dose of rATG. Autologous hematopoietic stem cells (HSCT) will be infused IV on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1.
HSCT: cells will be collected from blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous HSCT is to re-infuse immature cells that can re-establish blood production and patient's immune system.
Cyclophosphamide: Alkylating agent which causes prevention of cell division by forming adducts with DNA Mesna: Medication used to decrease the risk of hemorrhagic cystitis rATG: lymphocyte-specific immunosuppressive agent 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 Rituxan: Chimeric monoclonal antibody
|
|---|---|
|
Endocrine disorders
Hyperglycemia
|
21.7%
5/23 • Number of events 5 • Up to 4.5 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place