Hematopoietic Stem Cell Transplant in Devic's Disease
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|ClinicalTrials.gov Identifier: NCT00787722|
Recruitment Status : Completed
First Posted : November 7, 2008
Results First Posted : February 28, 2020
Last Update Posted : February 28, 2020
|Condition or disease||Intervention/treatment||Phase|
|Devic's Disease||Procedure: Hematopoietic Stem Cell Transplantation Drug: Cyclophosphamide Drug: G-CSF Drug: rATG Drug: Mesna Drug: Rituximab Drug: Methylprednisolone||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Trial of High Dose Immunosuppressive Therapy With Hematopoietic Stem Cell Support in Devic's Disease|
|Actual Study Start Date :||October 10, 2009|
|Actual Primary Completion Date :||November 2018|
|Actual Study Completion Date :||November 2018|
Experimental: Hematopoietic Stem Cell Transplantation
Hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide, G-CSF, Mesna, rATG, rituximab, and methylprednisolone.
Procedure: Hematopoietic Stem Cell Transplantation
Infusion of participant's own stem cells
A medication used as chemotherapy and to suppress the immune system
A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
A rabbit polyclonal antibody to lymphocytes
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Other Name: Mesnex
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Other Name: Rituxan
A corticosteroid medication used to suppress the immune system and decrease inflammation
- Survival [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]survival rate will be evaluated at 6 months,1 year, 2 year, 3 year, 4 year, 5 year after the transplant
- Quality of Life (QOL) Short Form - 36 (SF-36) [ Time Frame: pre-transplant 12mo and 5 years ]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.
- Post HSCT Immune -Modulating Medication and Relapse [ Time Frame: Pre transplant and 6 months, 1 year, 2 year, 3 year, 4 year and 5 year after transplant ]Number of immune - modulating medication and relapse evaluated 5 year - after the transplant
- Number of Patients Who Require No Device Assistance for Ambulation [ Time Frame: 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant ]No Device Assistance Needed for Ambulation evaluated at 6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant
- Disability Score: Expanded Disability Status Scale (EDSS) [ Time Frame: pretransplant 6 month, 5 year ]
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.
- NMO-IgG Aquaporin- 4 Autoantibody Titer [ Time Frame: Pretransplant and 5 year Post Transplant ]NMO-IgG aquaporin- 4 autoantibody titer will be tested pretransplant and post transplant.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00787722
|United States, Illinois|
|Northwestern University, Feinberg School of Medicine|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||Richard Burt, MD||Northwestern University|