Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Hematopoietic Stem Cell Transplant in Devic's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00787722
Recruitment Status : Completed
First Posted : November 7, 2008
Results First Posted : February 28, 2020
Last Update Posted : February 28, 2020
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 Devic's Disease
Interventions Procedure: Hematopoietic Stem Cell Transplantation
Drug: Cyclophosphamide
Drug: G-CSF
Drug: rATG
Drug: Mesna
Drug: Rituximab
Drug: Methylprednisolone
Enrollment 13
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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

Period Title: Overall Study
Started 13
Completed 12
Not Completed 1
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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 Number of Baseline Participants 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants
<=18 years 0
Between 18 and 65 years 13
>=65 years 0
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 13 participants
42
(19 to 51)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants
Female
12
  92.3%
Male
1
   7.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
5
  38.5%
White
8
  61.5%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 13 participants
13
1.Primary Outcome
Title Survival
Hide Description survival rate will be evaluated at 6 months,1 year, 2 year, 3 year, 4 year, 5 year after the transplant
Time Frame 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 13
Measure Type: Count of Participants
Unit of Measure: Participants
6 months survival Number Analyzed 13 participants
13
 100.0%
1 year survival Number Analyzed 12 participants
12
 100.0%
2 year survival Number Analyzed 12 participants
12
 100.0%
3 year survival Number Analyzed 11 participants
11
 100.0%
4 year survival Number Analyzed 11 participants
11
 100.0%
5 year survival Number Analyzed 11 participants
11
 100.0%
2.Secondary Outcome
Title Quality of Life (QOL) Short Form - 36 (SF-36)
Hide Description 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.
Time Frame pre-transplant 12mo and 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 11
Median (Standard Deviation)
Unit of Measure: score on a scale
Pretransplant Number Analyzed 11 participants
30.83  (34.22)
1 Year Post Transplant Number Analyzed 11 participants
52.69  (55.08)
5 Year Post Transplant Number Analyzed 10 participants
61.63  (62.09)
3.Secondary Outcome
Title Post HSCT Immune -Modulating Medication and Relapse
Hide Description Number of immune - modulating medication and relapse evaluated 5 year - after the transplant
Time Frame Pre transplant and 6 months, 1 year, 2 year, 3 year, 4 year and 5 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation (HSCT)
Hide Arm/Group Description:

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 Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
Pre HSCT - Immunosuppression/Relapse Rate Number Analyzed 12 participants
12
 100.0%
6 Mos Post HSCT Immunosuppression/ Relapse Rate Number Analyzed 12 participants
1
   8.3%
1 Year Post HSCT Immunosuppression/Relapse Rate Number Analyzed 11 participants
1
   9.1%
2 Year Post HSCT Immunosuppression/Relapse Rate Number Analyzed 12 participants
3
  25.0%
3 Year Post HSCT Immunosuppression/Relapse Rate Number Analyzed 11 participants
0
   0.0%
4 Year Post HSCT Immunosuppression/Relapse Rate Number Analyzed 11 participants
1
   9.1%
5 Year Post HSCT Immunosuppression Relapse Rate Number Analyzed 11 participants
1
   9.1%
4.Secondary Outcome
Title Number of Patients Who Require No Device Assistance for Ambulation
Hide Description No Device Assistance Needed for Ambulation evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
Time Frame 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
Pre HSCT- No Assistive Required Number Analyzed 12 participants
6
  50.0%
6 Mos Post HSCT- No Assistive Device Required Number Analyzed 12 participants
9
  75.0%
1 Year Post HSCT- No Assistive Device Required Number Analyzed 12 participants
10
  83.3%
2 Year Post HSCT- No Assistive Device Required Number Analyzed 12 participants
9
  75.0%
3 Year Post HSCT- No Assistive Device Required Number Analyzed 11 participants
8
  72.7%
4 Year Post HSCT- No Assistive Device Required Number Analyzed 12 participants
8
  66.7%
5 Year Post HSCT- No Assistive Device Required Number Analyzed 11 participants
9
  81.8%
5.Secondary Outcome
Title Disability Score: Expanded Disability Status Scale (EDSS)
Hide Description

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.

Time Frame pretransplant 6 month, 5 year
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 13
Mean (Full Range)
Unit of Measure: score on a scale
Pretransplant Disability Score (EDSS) Number Analyzed 13 participants
4.4
(2.0 to 6.5)
1 Year Post Transplant Disability Score (EDSS) Number Analyzed 12 participants
2.8
(1 to 6.5)
5 Year Post Transplant Disability Score (EDSS) Number Analyzed 11 participants
3.3
(0 to 6.5)
6.Secondary Outcome
Title NMO-IgG Aquaporin- 4 Autoantibody Titer
Hide Description NMO-IgG aquaporin- 4 autoantibody titer will be tested pretransplant and post transplant.
Time Frame Pretransplant and 5 year Post Transplant
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

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 Number of Participants Analyzed 13
Measure Type: Count of Participants
Unit of Measure: Participants
Pretransplant NMO ASSAY positive Number Analyzed 12 participants
11
  91.7%
5 year post transplant NMO Assay positive Number Analyzed 11 participants
2
  18.2%
Time Frame 5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

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

All-Cause Mortality
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%)
Total   1/13 (7.69%)    
Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   1/13 (7.69%)    
Immune system disorders   
Unrelated Complications from Systemic Lupus Erythematosus   1/13 (7.69%)  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   9/13 (69.23%)    
General disorders   
Neutropenic Fevers  [1]  5/13 (38.46%)  5
Orthostatic Hypotension  [2]  2/13 (15.38%)  2
Hypertension, Hyperglycemia, Diarrhea  [3]  1/13 (7.69%)  1
Infections and infestations   
Infection  [4]  1/13 (7.69%)  1
Metabolism and nutrition disorders   
Hypophosphatemia  [5]  9/13 (69.23%)  9
Hypocalcemia  [6]  3/13 (23.08%)  3
Nausea and Vomiting  [7]  3/13 (23.08%)  3
Hypokalemia  [8]  2/13 (15.38%)  2
Indicates events were collected by systematic assessment
[1]
Neutropenic Fevers
[2]
orthostatic hypotension
[3]
hypertension, hyperglycemia and diarrhea
[4]
C.Diff
[5]
hypophosphatemia
[6]
hypocalcemia
[7]
nausea and vomiting
[8]
hypokalemia
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: NCT00787722    
Other Study ID Numbers: DIAD Devic's Disease Auto 2008
First Submitted: October 31, 2008
First Posted: November 7, 2008
Results First Submitted: November 18, 2019
Results First Posted: February 28, 2020
Last Update Posted: February 28, 2020