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Stem Cell Transplantation for Stiff Person Syndrome (SPS) (SPS)

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ClinicalTrials.gov Identifier: NCT02282514
Recruitment Status : Terminated (Could not predict who would respond, relapse or go into remission)
First Posted : November 4, 2014
Results First Posted : January 6, 2021
Last Update Posted : January 27, 2021
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Stiff-Person Syndrome
Interventions Biological: Autologous Hematopoietic Stem Cells
Drug: Cyclophosphamide
Drug: Mesna
Drug: rATG
Drug: Methylprednisolone
Drug: G-CSF
Drug: Rituxan
Enrollment 23
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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

Period Title: Overall Study
Started 23
Completed [1] 23
Not Completed 0
[1]
Conditioning Regimen
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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 Number of Baseline Participants 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
<=18 years
0
   0.0%
Between 18 and 65 years
23
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 23 participants
48.3
(27 to 60)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
Female
21
  91.3%
Male
2
   8.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   8.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
3
  13.0%
White
17
  73.9%
More than one race
0
   0.0%
Unknown or Not Reported
1
   4.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 23 participants
23
1.Primary Outcome
Title Overall Survival
Hide Description Number of Participants who Did Not Experience Treatment-Related Mortality
Time Frame Mean 3.6 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 23
Measure Type: Number
Unit of Measure: participants
23
2.Secondary Outcome
Title Reduction of Muscle Relaxation Anti-spasmatic Medications
Hide Description Decrease (50%) and complete discontinuation of muscle relaxation anti-spasmatic medications
Time Frame Mean 3.6 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 23
Measure Type: Count of Participants
Unit of Measure: Participants
14
  60.9%
3.Secondary Outcome
Title Short-form 36 Quality of Life Questionnaire (SF-36 QOL)
Hide Description Improvement 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.
Time Frame mean 3.6 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 23
Geometric Mean (Standard Deviation)
Unit of Measure: units on a scale
Pre Treatment 37.75  (12.81)
Post Treatment 58.35  (22.81)
Time Frame Up to 4.5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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

All-Cause Mortality
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%)
Total   1/23 (4.35%)    
Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   1/23 (4.35%)    
General disorders   
Non Treatment Related Death   1/23 (4.35%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   5/23 (21.74%)    
Endocrine disorders   
Hyperglycemia   5/23 (21.74%)  5
Indicates events were collected by systematic assessment
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: NCT02282514    
Other Study ID Numbers: DIAD.SPS.2014
First Submitted: October 30, 2014
First Posted: November 4, 2014
Results First Submitted: November 16, 2020
Results First Posted: January 6, 2021
Last Update Posted: January 27, 2021