Trial Outcomes & Findings for Hematopoietic Stem Cell Transplant in Devic's Disease (NCT NCT00787722)
NCT ID: NCT00787722
Last Updated: 2020-02-28
Results Overview
survival rate will be evaluated at 6 months,1 year, 2 year, 3 year, 4 year, 5 year after the transplant
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
13 participants
Primary outcome timeframe
6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
Results posted on
2020-02-28
Participant Flow
Participant milestones
| Measure |
Hematopoietic Stem Cell Transplantation
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Hematopoietic Stem Cell Transplant in Devic's Disease
Baseline characteristics by cohort
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
42 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplantPopulation: The reason that the number analyzed in one or more rows differs from the overall number analyzed is because one patient with coexistent SLE died of complications from active lupus 10 months after the transplant and another patient was only 3 years out from transplant.
survival rate will be evaluated at 6 months,1 year, 2 year, 3 year, 4 year, 5 year after the transplant
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Survival
6 months survival
|
13 Participants
|
|
Survival
1 year survival
|
12 Participants
|
|
Survival
2 year survival
|
12 Participants
|
|
Survival
3 year survival
|
11 Participants
|
|
Survival
4 year survival
|
11 Participants
|
|
Survival
5 year survival
|
11 Participants
|
SECONDARY outcome
Timeframe: pre-transplant 12mo and 5 yearsPopulation: The reason that the number analyzed differs in one or more rows is because 1 patient did not complete the form and was excluded. Another patient was not 5 years out from transplant so there is no 5 year data on the patient.
SF- 36 is a self-administered quality of life exam. The evaluation of the results was done by attributing scores to each question, which were then transformed into a scale ranging from 0 to 100, where 0 corresponds to the worst quality of life and 100 to the best.
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=11 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Quality of Life (QOL) Short Form - 36 (SF-36)
Pretransplant
|
30.83 score on a scale
Standard Deviation 34.22
|
|
Quality of Life (QOL) Short Form - 36 (SF-36)
1 Year Post Transplant
|
52.69 score on a scale
Standard Deviation 55.08
|
|
Quality of Life (QOL) Short Form - 36 (SF-36)
5 Year Post Transplant
|
61.63 score on a scale
Standard Deviation 62.09
|
SECONDARY outcome
Timeframe: Pre transplant and 6 months, 1 year, 2 year, 3 year, 4 year and 5 year after transplantPopulation: The reason that the number analyzed in one or more rows differs from the overall number analyzed is because one patient with coexistent SLE died of complications from active lupus 10 months after the transplant and another patient was only 3 years out from transplant.
Number of immune - modulating medication and relapse evaluated 5 year - after the transplant
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=12 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Post HSCT Immune -Modulating Medication and Relapse
Pre HSCT - Immunosuppression/Relapse Rate
|
12 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
6 Mos Post HSCT Immunosuppression/ Relapse Rate
|
1 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
1 Year Post HSCT Immunosuppression/Relapse Rate
|
1 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
2 Year Post HSCT Immunosuppression/Relapse Rate
|
3 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
3 Year Post HSCT Immunosuppression/Relapse Rate
|
0 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
4 Year Post HSCT Immunosuppression/Relapse Rate
|
1 Participants
|
|
Post HSCT Immune -Modulating Medication and Relapse
5 Year Post HSCT Immunosuppression Relapse Rate
|
1 Participants
|
SECONDARY outcome
Timeframe: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplantPopulation: The reason that the number analyzed in one or more rows differs from the overall number analyzed is because one patient with coexistent SLE died of complications from active lupus 10 months after the transplant and another patient was only 3 years out from transplant.
No Device Assistance Needed for Ambulation evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=12 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Number of Patients Who Require No Device Assistance for Ambulation
1 Year Post HSCT- No Assistive Device Required
|
10 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
Pre HSCT- No Assistive Required
|
6 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
6 Mos Post HSCT- No Assistive Device Required
|
9 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
2 Year Post HSCT- No Assistive Device Required
|
9 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
3 Year Post HSCT- No Assistive Device Required
|
8 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
4 Year Post HSCT- No Assistive Device Required
|
8 Participants
|
|
Number of Patients Who Require No Device Assistance for Ambulation
5 Year Post HSCT- No Assistive Device Required
|
9 Participants
|
SECONDARY outcome
Timeframe: pretransplant 6 month, 5 yearPopulation: The reason that the number analyzed in one or more rows differs from the overall number analyzed is because one patient with coexistent SLE died of complications from active lupus 10 months after the transplant and another patient was only 3 years out from transplant.
Disability scores (disease improvement defined by at least a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least three months apart. The EDSS scale ranges from 0 to 10 in 0.5 unit increments that represent higher levels of disability.
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Disability Score: Expanded Disability Status Scale (EDSS)
Pretransplant Disability Score (EDSS)
|
4.4 score on a scale
Interval 2.0 to 6.5
|
|
Disability Score: Expanded Disability Status Scale (EDSS)
1 Year Post Transplant Disability Score (EDSS)
|
2.8 score on a scale
Interval 1.0 to 6.5
|
|
Disability Score: Expanded Disability Status Scale (EDSS)
5 Year Post Transplant Disability Score (EDSS)
|
3.3 score on a scale
Interval 0.0 to 6.5
|
SECONDARY outcome
Timeframe: Pretransplant and 5 year Post TransplantPopulation: The reason that the number analyzed in one or more rows differs from the overall number analyzed is because one patient with coexistent SLE died of complications from active lupus 10 months after the transplant and another patient was only 3 years out from transplant.
NMO-IgG aquaporin- 4 autoantibody titer will be tested pretransplant and post transplant.
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 Participants
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
NMO-IgG Aquaporin- 4 Autoantibody Titer
Pretransplant NMO ASSAY positive
|
11 Participants
|
|
NMO-IgG Aquaporin- 4 Autoantibody Titer
5 year post transplant NMO Assay positive
|
2 Participants
|
Adverse Events
Hematopoietic Stem Cell Transplantation
Serious events: 1 serious events
Other events: 9 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 participants at risk
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Immune system disorders
Unrelated Complications from Systemic Lupus Erythematosus
|
7.7%
1/13 • Number of events 1 • 5 years
|
Other adverse events
| Measure |
Hematopoietic Stem Cell Transplantation
n=13 participants at risk
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Hematopoietic Stem Cell Transplantation: Infusion of participant's own stem cells
Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system
G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
rATG: A rabbit polyclonal antibody to lymphocytes
Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation
|
|---|---|
|
Infections and infestations
Infection
|
7.7%
1/13 • Number of events 1 • 5 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
69.2%
9/13 • Number of events 9 • 5 years
|
|
General disorders
Neutropenic Fevers
|
38.5%
5/13 • Number of events 5 • 5 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
23.1%
3/13 • Number of events 3 • 5 years
|
|
Metabolism and nutrition disorders
Nausea and Vomiting
|
23.1%
3/13 • Number of events 3 • 5 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
15.4%
2/13 • Number of events 2 • 5 years
|
|
General disorders
Orthostatic Hypotension
|
15.4%
2/13 • Number of events 2 • 5 years
|
|
General disorders
Hypertension, Hyperglycemia, Diarrhea
|
7.7%
1/13 • Number of events 1 • 5 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place