Stem Cell Transplantation for Stiff Person Syndrome (SPS) (SPS)
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| ClinicalTrials.gov Identifier: NCT02282514 |
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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
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| 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 |
|---|---|
 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 |
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[1]
Conditioning Regimen
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| Arm/Group Title | Hematopoietic Stem Cell Transplantation | |
|---|---|---|
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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 |
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| Overall Number of Baseline Participants | 23 | |
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Baseline Analysis Population Description |
[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 23 participants | |
| <=18 years |
0 0.0%
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| Between 18 and 65 years |
23 100.0%
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| >=65 years |
0 0.0%
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Age, Continuous
Mean (Full Range) Unit of measure: Years |
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| Number Analyzed | 23 participants | |
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48.3
(27 to 60)
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 23 participants | |
| Female |
21 91.3%
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| Male |
2 8.7%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 23 participants | |
| American Indian or Alaska Native |
0 0.0%
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| Asian |
2 8.7%
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|
| Native Hawaiian or Other Pacific Islander |
0 0.0%
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| Black or African American |
3 13.0%
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| White |
17 73.9%
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| More than one race |
0 0.0%
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| Unknown or Not Reported |
1 4.3%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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| United States | Number Analyzed | 23 participants |
| 23 | ||
| Name/Title: | Kathleen Quigley |
| Organization: | Northwestern University |
| Phone: | 312-695-8192 |
| EMail: | k-quigley@northwestern.edu |
| 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 |